Cover
Cover - USD ($) | 3 Months Ended | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2019 | Feb. 13, 2020 | Jun. 30, 2019 | |
Cover page. | ||||
Document Type | 10-K | |||
Document Annual Report | true | |||
Document Period End Date | Dec. 31, 2019 | |||
Document Transition Report | false | |||
Entity File Number | 001-35966 | |||
Entity Registrant Name | bluebird bio, Inc. | |||
Entity Incorporation, State or Country Code | DE | |||
Entity Tax Identification Number | 13-3680878 | |||
Entity Address, Address Line One | 60 Binney Street | |||
Entity Address, City or Town | Cambridge | |||
Entity Address, State or Province | MA | |||
Entity Address, Postal Zip Code | 02142 | |||
City Area Code | 339 | |||
Local Phone Number | 499-9300 | |||
Title of 12(b) Security | Common Stock, $0.01 par value per share | |||
Trading Symbol | BLUE | |||
Security Exchange Name | NASDAQ | |||
Entity Well-known Seasoned Issuer | Yes | |||
Entity Voluntary Filers | No | |||
Entity Current Reporting Status | Yes | |||
Entity Interactive Data Current | Yes | |||
Entity Filer Category | Large Accelerated Filer | |||
Entity Small Business | false | |||
Entity Emerging Growth Company | false | |||
Entity Shell Company | false | |||
Entity Public Float | $ 7,025,034,290 | |||
Entity Common Stock, Shares Outstanding | 55,611,565 | |||
Documents Incorporated by Reference | Portions of the registrant’s definitive Proxy Statement relating to its 2020 Annual Meeting of Stockholders are incorporated by reference into Part III of this Annual Report on Form 10-K where indicated. Such Proxy Statement will be filed with the U.S. Securities and Exchange Commission within 120 days after the end of the fiscal year to which this report relates. | |||
Amendment Flag | false | |||
Document Fiscal Year Focus | 2019 | |||
Document Fiscal Period Focus | FY | |||
Entity Central Index Key | 0001293971 | |||
Current Fiscal Year End Date | --12-31 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 327,214 | $ 402,579 |
Marketable securities | 779,246 | 982,725 |
Prepaid expenses | 32,888 | 19,762 |
Receivables and other current assets | 12,826 | 13,931 |
Total current assets | 1,152,174 | 1,418,997 |
Marketable securities | 131,506 | 506,123 |
Property, plant and equipment, net | 151,176 | 246,622 |
Intangible assets, net | 14,326 | 13,169 |
Goodwill | 13,128 | 13,128 |
Operating lease right-of-use assets | 185,885 | |
Restricted cash and other non-current assets | 79,229 | 44,805 |
Total assets | 1,727,424 | 2,242,844 |
Current liabilities: | ||
Accounts payable | 42,995 | 17,831 |
Accrued expenses and other current liabilities | 141,556 | 99,393 |
Operating lease liability, current portion | 20,175 | |
Deferred revenue, current portion | 8,474 | 18,602 |
Collaboration research advancement, current portion | 10,380 | 10,605 |
Total current liabilities | 223,580 | 146,431 |
Deferred revenue, net of current portion | 9,791 | 16,338 |
Collaboration research advancement, net of current portion | 27,834 | 33,349 |
Contingent consideration | 7,977 | 5,230 |
Operating lease liability, net of current portion | 170,812 | |
Financing lease obligation, net of current portion | 0 | 153,319 |
Other non-current liabilities | 2,437 | 3,107 |
Total liabilities | 442,431 | 357,774 |
Commitments and contingencies Note 9 | ||
Stockholders' equity: | ||
Preferred stock, $0.01 par value, 5,000 shares authorized; 0 shares issued and outstanding at December 31, 2019 and December 31, 2018 | 0 | 0 |
Common stock, $0.01 par value, 125,000 shares authorized; 55,368 and 54,738 shares issued and outstanding at December 31, 2019 and December 31, 2018, respectively | 554 | 547 |
Additional paid-in capital | 3,568,184 | 3,386,958 |
Accumulated other comprehensive loss | (1,893) | (3,627) |
Accumulated deficit | (2,281,852) | (1,498,808) |
Total stockholders' equity | 1,284,993 | 1,885,070 |
Total liabilities and stockholders' equity | $ 1,727,424 | $ 2,242,844 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value per share (in dollars per share) | $ 10 | $ 10 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 10 | $ 10 |
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, shares issued (in shares) | 55,368,000 | 54,738,000 |
Common stock, shares outstanding (in shares) | 55,368,000 | 54,738,000 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenue: | |||
Revenue | $ 44,674 | $ 54,579 | $ 35,427 |
Operating expenses: | |||
Research and development | 582,413 | 448,589 | 273,040 |
Selling, general and administrative | 271,362 | 174,129 | 93,550 |
Cost of license and royalty revenue | 2,978 | 885 | 1,527 |
Change in fair value of contingent consideration | 2,747 | 2,999 | (525) |
Total operating expenses | 859,500 | 626,602 | 367,592 |
Loss from operations | (814,826) | (572,023) | (332,165) |
Interest income (expense), net | 34,761 | 14,624 | (2,001) |
Other (expense) income, net | (10,088) | 1,961 | (1,267) |
Loss before income taxes | (790,153) | (555,438) | (335,433) |
Income tax benefit (expense) | 545 | (187) | (210) |
Net loss | $ (789,608) | $ (555,625) | $ (335,643) |
Net loss per share - basic and diluted (in dollars per share) | $ (14.31) | $ (10.68) | $ (7.71) |
Weighted-average number of common shares used in computing net loss per share - basic and diluted (in shares) | 55,191 | 52,032 | 43,535 |
Other comprehensive income (loss): | |||
Other comprehensive income | $ 1,734 | $ 578 | $ (3,056) |
Comprehensive loss | (787,874) | (555,047) | (338,699) |
Other comprehensive income (loss) tax expense | 1,200 | 400 | 0 |
Collaboration revenue | |||
Revenue: | |||
Revenue | 36,469 | 52,353 | 22,207 |
License and royalty revenue | |||
Revenue: | |||
Revenue | $ 8,205 | $ 2,226 | $ 13,220 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Statement of Financial Position [Abstract] | ||
Costs from initial public offering | $ 34,588 | $ 53,487 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Cash flows from operating activities: | |||
Net loss | $ (789,608) | $ (555,625) | $ (335,643) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Change in fair value of contingent consideration | 2,747 | 2,999 | (2,189) |
Depreciation and amortization | 17,434 | 17,158 | 13,538 |
Stock-based compensation expense | 160,629 | 110,836 | 53,282 |
Unrealized loss (gain) on equity securities | (9,297) | 2,154 | 0 |
Other non-cash items | (11,000) | (5,880) | 3,153 |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other assets | (13,913) | (24,288) | (20,092) |
Operating lease right-of-use assets | 22,496 | ||
Accounts payable | 23,600 | 3,614 | 526 |
Accrued expenses and other liabilities | 46,291 | 37,832 | 5,848 |
Operating lease liabilities | (9,944) | ||
Deferred revenue | (16,674) | (41,872) | 1,024 |
Collaboration research advancement | (5,739) | 43,954 | 0 |
Net cash used in operating activities | (564,384) | (413,426) | (280,553) |
Cash flows from investing activities: | |||
Purchase of property, plant and equipment | (71,028) | (55,737) | (62,242) |
Purchases of marketable securities | (756,570) | (1,517,982) | (686,204) |
Proceeds from maturities of marketable securities | 1,340,629 | 894,284 | 431,816 |
Purchase of intangible assets | (5,224) | 0 | 0 |
Net cash provided by (used in) investing activities | 507,807 | (679,435) | (316,630) |
Cash flows from financing activities: | |||
Cash paid for contingent purchase price consideration | 0 | 0 | (1,074) |
Reimbursement of assets under financing lease obligation | 0 | 3,098 | 38,021 |
Payments on financing lease obligation | (1,017) | (574) | |
Proceeds from issuance of common stock | 0 | 649,368 | 1,006,570 |
Proceeds from exercise of stock options and ESPP contributions | 21,187 | 31,759 | 33,231 |
Net cash provided by financing activities | 21,187 | 737,692 | 1,076,174 |
(Decrease) increase in cash, cash equivalents and restricted cash | (35,390) | (355,169) | 478,991 |
Cash, cash equivalents and restricted cash at beginning of year | 417,099 | 772,268 | 293,277 |
Cash, cash equivalents and restricted cash at end of year | 381,709 | 417,099 | 772,268 |
Reconciliation of cash, cash equivalents and restricted cash: | |||
Cash and cash equivalents | 327,214 | 402,579 | 758,505 |
Restricted cash included in receivables and other current assets | 0 | 364 | 0 |
Restricted cash included in restricted cash and other non-current assets | 54,495 | 14,156 | 13,763 |
Supplemental cash flow disclosures: | |||
Assets acquired under financing lease obligation | 0 | 0 | 3,271 |
Purchases of property, plant and equipment included in accounts payable and accrued expenses | 5,286 | 7,449 | 2,566 |
Right-of-use assets obtained in exchange for operating lease liabilities | 23,939 | ||
Cash paid during the period for interest | 0 | 15,494 | 11,411 |
Cash paid during the period for income taxes | 637 | 358 | 91 |
Regeneron Collaboration Agreement | |||
Cash flows from financing activities: | |||
Proceeds from issuance of common stock | $ 0 | $ 54,484 | $ 0 |
Consolidated Statements of St_2
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common stock | Additional paid-in capital | Accumulated other comprehensive loss | Accumulated Deficit |
Beginning balance (in shares) at Dec. 31, 2016 | 40,691,000 | ||||
Beginning balance at Dec. 31, 2016 | $ 869,440 | $ 407 | $ 1,447,856 | $ (1,149) | $ (577,674) |
Vesting of restricted stock units (in shares) | 88,000 | ||||
Vesting of restricted stock units | 0 | $ 1 | (1) | ||
Issuance of common stock upon public offering, net of issuance costs (in shares) | 7,625,000 | ||||
Issuance of common stock upon public offering, net of issuance costs of $53,487 | 1,006,570 | $ 76 | 1,006,494 | ||
Exercise of stock options (in shares) | 981,000 | ||||
Exercise of stock options | 31,686 | $ 10 | 31,676 | ||
Purchase of common stock under ESPP (in shares) | 21,000 | ||||
Purchase of common stock under ESPP | 1,153 | 1,153 | |||
Stock-based compensation | 53,282 | 53,282 | |||
Other comprehensive income | (3,056) | (3,056) | |||
Net loss | (335,643) | (335,643) | |||
Ending balance (in shares) at Dec. 31, 2017 | 49,406,000 | ||||
Ending balance at Dec. 31, 2017 | 1,623,432 | $ 494 | 2,540,951 | (4,205) | (913,808) |
Vesting of restricted stock units (in shares) | 152,000 | ||||
Vesting of restricted stock units | $ 2 | (2) | |||
Issuance of common stock upon public offering, net of issuance costs (in shares) | 4,169,000 | ||||
Issuance of common stock upon public offering, net of issuance costs of $53,487 | 649,368 | $ 42 | 649,326 | ||
Issuance of common stock to Regeneron (in shares) | 420,000 | ||||
Issuance of common stock to Regeneron | 54,484 | $ 4 | 54,480 | ||
Exercise of stock options (in shares) | 575,000 | ||||
Exercise of stock options | 29,768 | $ 5 | 29,763 | ||
Purchase of common stock under ESPP (in shares) | 16,000 | ||||
Purchase of common stock under ESPP | 1,604 | 1,604 | |||
Stock-based compensation | 110,836 | 110,836 | |||
Other comprehensive income | 578 | 578 | |||
Net loss | $ (555,625) | (555,625) | |||
Ending balance (in shares) at Dec. 31, 2018 | 54,738,000 | 54,738,000 | |||
Ending balance at Dec. 31, 2018 | $ 1,885,070 | $ 547 | 3,386,958 | (3,627) | (1,498,808) |
Vesting of restricted stock units (in shares) | 251,000 | ||||
Vesting of restricted stock units | $ 3 | (3) | |||
Exercise of stock options (in shares) | 354,000 | 354,000 | |||
Exercise of stock options | $ 17,838 | $ 4 | 17,834 | ||
Purchase of common stock under ESPP (in shares) | 25,000 | ||||
Purchase of common stock under ESPP | 2,766 | 2,766 | |||
Stock-based compensation | 160,629 | 160,629 | |||
Other comprehensive income | 1,734 | 1,734 | |||
Net loss | $ (789,608) | (789,608) | |||
Ending balance (in shares) at Dec. 31, 2019 | 55,368,000 | 55,368,000 | |||
Ending balance at Dec. 31, 2019 | $ 1,284,993 | $ 554 | $ 3,568,184 | $ (1,893) | $ (2,281,852) |
Description of the business
Description of the business | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of the business | Description of the business bluebird bio, Inc. (the “Company” or “bluebird”) was incorporated in Delaware on April 16, 1992, and is headquartered in Cambridge, Massachusetts. The Company is a biotechnology company committed to researching, developing and commercializing potentially transformative gene therapies for severe genetic diseases and cancer. Since its inception, the Company has devoted substantially all of its resources to its research and development efforts relating to its product candidates, including activities to manufacture product candidates, conduct clinical studies of its product candidates, perform preclinical research to identify new product candidates and provide selling, general and administrative support for these operations, including commercial-readiness activities. The Company’s programs in severe genetic diseases include its programs in β-thalassemia (TDT), sickle cell disease (SCD), and cerebral adrenoleukodystrophy (CALD). The Company’s programs in oncology are focused on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR) and T cell receptor (TCR) T cell therapies. Idecabtagene vicleucel, or ide-cel, and bb21217, which are product candidates in oncology under the Company’s collaboration arrangement with Bristol-Myers Squibb ("BMS"), formerly Celgene Corporation (prior to its acquisition by BMS in November 2019), are CAR T cell product candidates for the treatment of multiple myeloma. Please refer to Note 11, Collaborative arrangements, for further discussion of the Company’s collaboration with BMS. In June 2019, the Company received conditional marketing authorization from the European Commission for ZYNTEGLO TM gene therapy (autologous CD34+ cells encoding β A-T87Q -globin gene), also referred to as LentiGlobin for TDT, for the treatment of patients 12 years and older with TDT who do not have a β 0 /β 0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte-matched related HSC donor is not available. Since receiving conditional marketing authorization for ZYNTEGLO, the Company has continued to advance its commercial readiness activities. Through December 31, 2019, the Company had not generated any revenue from product sales. In accordance with Accounting Standards Codification ("ASC") 205-40, Going Concern , the Company evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about its ability to continue as a going concern within one year after the date that the consolidated financial statements are issued. The Company has incurred losses since inception and to date has financed its operations primarily through the sale of equity securities and, to a lesser extent, through collaboration agreements and grants from governmental agencies and charitable foundations. As of December 31, 2019, the Company had an accumulated deficit of $2.28 billion. During the year ended December 31, 2019, the Company incurred a loss of $789.6 million and used $564.4 million of cash in operations. The Company expects to continue to generate operating losses and negative operating cash flows for the next few years and will need additional funding to support its planned operating activities through profitability. The transition to profitability is dependent upon the successful development, approval, and commercialization of the Company’s products and product candidates and the achievement of a level of revenues adequate to support its cost structure. As of December 31, 2019, the Company had cash, cash equivalents and marketable securities of $1.24 billion. The Company expects its cash, cash equivalents and marketable securities will be sufficient to fund current planned operations for at least the next twelve months from the date of issuance of these financial statements, though it may pursue additional cash resources through public or private equity or debt financings or by establishing additional collaborations with other companies. Management's expectations with respect to its ability to fund current planned operations is based on estimates that are subject to risks and uncertainties. If actual results are different from management's estimates, the Company may need to seek additional strategic or financing opportunities sooner than would otherwise be expected. However, there is no guarantee that any of these strategic or financing opportunities will be executed or executed on favorable terms, and some could be dilutive to existing stockholders. If the Company is unable to obtain additional funding on a timely basis, it may be forced to significantly curtail, delay, or discontinue one or more of its planned research or development programs or be unable to expand its operations or otherwise capitalize on its commercialization of its product candidates. |
Summary of significant accounti
Summary of significant accounting policies and basis of presentation | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of significant accounting policies and basis of presentation | Summary of significant accounting policies and basis of presentation Basis of presentation The accompanying consolidated financial statements have been prepared by the Company in accordance with accounting principles generally accepted in the United States (“GAAP”) as found in the ASC and Accounting Standards Updates (“ASUs”) of the Financial Accounting Standards Board (“FASB”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States GAAP as found in the ASC and ASUs of the FASB. Certain items in the prior year’s consolidated financial statements have been reclassified to conform to the current presentation. No subtotals in the prior year consolidated financial statements were impacted by these reclassifications. Amounts reported are computed based on thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. Principles of consolidation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company continually assesses whether it is the primary beneficiary of a variable interest entity as changes to existing relationships or future transactions may result in consolidation or deconsolidation of one or more collaborators or partners. In determining whether it is the primary beneficiary of an entity in which the Company has a variable interest, management applies a qualitative approach that determines whether the Company has both (1) the power to direct the economically significant activities of the entity and (2) the obligation to absorb losses of, or the right to receive benefits from, the entity that could potentially be significant to that entity. Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could materially differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements. Estimates and judgments are used in the following areas, among others: future undiscounted cash flows and subsequent fair value estimates used to assess potential and measure any impairment of long-lived assets, including goodwill and intangible assets, and the measurement of right-of-use assets and lease liabilities, contingent consideration, stock-based compensation expense, accrued expenses, income taxes, and the assessment of the Company's ability to fund its operations for at least the next twelve months from the date of issuance of these financial statements. In addition, estimates and judgments are used in the Company’s accounting for its revenue-generating arrangements, in particular as it relates to determining the standalone selling price of performance obligations, evaluating whether an option to acquire additional goods and services represents a material right, estimating the total transaction price, including estimating variable consideration and the probability of achieving future potential development and regulatory milestones, and the period of performance over which revenue may be recognized. Foreign currency translation The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars using period-end exchange rates for assets and liabilities, historical exchange rates for stockholders’ equity and weighted average exchange rates for operating results. Translation gains and losses are included in accumulated other comprehensive income (loss) in stockholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. Segment information The Company operates in a single segment, focusing on researching, developing and commercializing potentially transformative gene therapies for severe genetic diseases and cancer. Consistent with its operational structure, its chief operating decision maker manages and allocates resources at a global, consolidated level. Therefore, results of the Company's operations are reported on a consolidated basis for purposes of segment reporting. All material long-lived assets of the Company reside in the United States. Cash and cash equivalents The Company considers all highly liquid investments purchased with original final maturities of 90 days or less from the date of purchase to be cash equivalents. Cash and cash equivalents comprise marketable securities with maturities of less than 90 days when purchased. Cash equivalents are reported at fair value. Marketable securities The Company classifies marketable securities with a remaining maturity when purchased of greater than three months as available-for-sale. Marketable securities with a remaining maturity date greater than one year are classified as non-current. The Company’s marketable securities are maintained by investment managers and consist of U.S. Treasury securities, U.S. government agency securities, equity securities, certificates of deposit, corporate bonds, and commercial paper. Debt securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium arising at purchase is amortized to the earliest call date and any discount arising at purchase is accreted to maturity. Amortization and accretion of premiums and discounts are recorded in interest income and/or expense. Equity securities with readily determinable fair values are also carried at fair value with unrealized gains and losses included in other (expense) income, net. Realized gains and losses on both debt and equity securities are determined using the specific identification method and are included in other income (expense), net. The Company classifies equity securities with readily determinable fair values, which would be available for use in its current operations, as current assets even though the Company may not dispose of such marketable securities within the next 12 months. Equity securities are included in the balance of marketable securities on the Company's consolidated balance sheet. If any adjustment to fair value reflects a decline in value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other-than-temporary” and, if so, marks the investment to market through a charge to the Company’s statement of operations and comprehensive loss. Concentrations of credit risk and off-balance sheet risk Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities primarily consist of U.S. Treasury securities, U.S. government agency securities, certificates of deposit, corporate bonds, and commercial paper, and potentially subject the Company to concentrations of credit risk. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure. Fair value of financial instruments The Company has certain financial assets and liabilities recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements Level 1—Fair values are determined utilizing quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2—Fair values are determined utilizing quoted prices for identical or similar assets or liabilities in active markets or other market observable inputs such as interest rates, yield curves and foreign currency spot rates. Level 3—Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. Items measured at fair value on a recurring basis include marketable securities (see Note 3, Marketable securities, and Note 4, Fair value measurements ) and contingent consideration (see Note 4, Fair value measurements ). The carrying amounts of accounts payable and accrued expenses approximate their fair values due to their short-term nature. Business combinations Business combinations are accounted for using the acquisition method of accounting. Using this method, the tangible and intangible assets acquired and the liabilities assumed are recorded as of the acquisition date at their respective fair values. The Company evaluates a business as an integrated set of activities and assets that is capable of being managed for the purpose of providing a return in the form of dividends, lower costs or other economic benefits and consists of inputs and processes that provide or have the ability to provide outputs. In an acquisition of a business, the excess of the fair value of the consideration transferred over the fair value of the net assets acquired is recorded as goodwill. In an acquisition of net assets that does not constitute a business, no goodwill is recognized. The consolidated financial statements include the results of operations of an acquired business after the completion of the acquisition. See Note 4, Fair value measurements, for additional information. Goodwill Goodwill represents the excess of the purchase price over the fair value of the net assets acquired when accounted for using the acquisition method of accounting for business combinations. Goodwill is not amortized but is evaluated for impairment within the Company’s single reporting unit on an annual basis, during the fourth quarter, or more frequently if an event occurs or circumstances change that would more-likely-than-not reduce the fair value of the Company’s reporting unit below its carrying amount. During the fourth quarter of 2019, the Company early adopted ASU 2017-04, which removes the second step of the goodwill impairment test. Under this ASU, the Company will now perform a one-step quantitative test and record the amount of goodwill impairment, if any, as the excess of a reporting unit's carrying amount over its fair value, not to exceed the total amount of goodwill allocated to the reporting unit. The Company has not recognized any impairment charges related to goodwill to date. Intangible assets, net Intangible assets, net consist of acquired core technology and in-licensed rights with finite lives, net of accumulated amortization. The Company amortizes its intangible assets using the straight-line method over their estimated economic lives and periodically reviews for impairment. Contingent consideration Each reporting period, the Company revalues the contingent consideration obligations associated with business combinations to their fair value and records within operating expenses increases in their fair value as contingent consideration expense and decreases in the fair value as contingent consideration income. Changes in contingent consideration result from changes in the assumptions regarding probabilities of successful achievement of related milestones, the estimated timing in which the milestones may be achieved, and the discount rate used to estimate the fair value of the liability. Contingent consideration may change significantly as development of the Company’s programs in certain indications progress and additional data are obtained, impacting the Company’s assumptions. The assumptions used in estimating fair value require significant judgment. The use of different assumptions and judgments could result in a materially different estimate of fair value. See Note 4, Fair value measurements, for additional information. Property, plant and equipment Property, plant and equipment is stated at cost. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Depreciation and amortization is calculated using the straight-line method over the estimated useful lives of the assets, which are as follows: Asset Estimated useful life Building 40 years Computer equipment and software 3 years Furniture and fixtures 2-5 years Laboratory equipment 2-5 years Leasehold improvements Shorter of the useful life or remaining lease term Prior to the adoption of ASU 2016-02, Leases (Topic 842) (“ASU 2016-02” or “ASC 842”), on January 1, 2019 (discussed further below), the Company recorded certain costs incurred and reported by a landlord as a building asset and corresponding financing lease obligation on the consolidated balance sheets. See Note 8, Leases , for additional information. Impairment of long-lived assets The Company reviews long-lived assets when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. Recoverability is measured by comparison of the book values of the assets to future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets. Leases Effective January 1, 2019, the Company adopted ASC 842 using the required modified retrospective approach and utilizing the effective date as its date of initial application. As a result, prior periods are presented in accordance with the previous guidance in ASC 840, Leases (“ASC 840”). At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than one year are recognized on the balance sheet as right-of-use assets and short-term and long-term lease liabilities, as applicable. The Company does not have material financing leases. Operating lease liabilities and their corresponding right-of-use assets are initially recorded based on the present value of lease payments over the expected remaining lease term. Certain adjustments to the right-of-use asset may be required for items such as incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate to discount lease payments, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. To estimate its incremental borrowing rate, a credit rating applicable to the Company is estimated using a synthetic credit rating analysis since the Company does not currently have a rating agency-based credit rating. Prospectively, the Company will adjust the right-of-use assets for straight-line rent expense or any incentives received and remeasure the lease liability at the net present value using the same incremental borrowing rate that was in effect as of the lease commencement or transition date. The Company has elected not to recognize leases with an original term of one year or less on the balance sheet. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. Assumptions made by the Company at the commencement date are re-evaluated upon occurrence of certain events, including a lease modification. A lease modification results in a separate contract when the modification grants the lessee an additional right of use not included in the original lease and when lease payments increase commensurate with the standalone price for the additional right of use. When a lease modification results in a separate contract, it is accounted for in the same manner as a new lease. ASC 842 transition practical expedients and application of transition provisions to leases at the transition date The Company elected the following practical expedients, which must be elected as a package and applied consistently to all of its leases at the transition date (including those for which the entity is a lessee or a lessor): i) the Company did not reassess whether any expired or existing contracts are or contain leases; ii) the Company did not reassess the lease classification for any expired or existing leases (that is, all existing leases that were classified as operating leases in accordance with ASC 840 are classified as operating leases, and all existing leases that were classified as capital leases in accordance with ASC 840 are classified as finance leases); and iii) the Company did not reassess initial direct costs for any existing leases. For leases that existed prior to the date of initial application of ASC 842 (which were previously classified as operating leases), a lessee may elect to use either the total lease term measured at lease inception under ASC 840 or the remaining lease term as of the date of initial application of ASC 842 in determining the period for which to measure its incremental borrowing rate. In transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rates. Application of ASC 842 policy elections to leases post adoption The Company has made certain policy elections to apply to its leases executed post adoption, or subsequent to January 1, 2019, as further described below. In accordance with ASC 842, components of a lease should be split into three categories: lease components, non-lease components, and non-components. The fixed and in-substance fixed contract consideration (including any consideration related to non-components) must be allocated based on the respective relative fair values to the lease components and non-lease components. Entities may elect not to separate lease and non-lease components. Rather, entities would account for each lease component and related non-lease component together as a single lease component. The Company has elected to account for lease and non-lease components together as a single lease component for all underlying assets and allocate all of the contract consideration to the lease component only. ASC 842 allows for the use of judgment in determining whether the assumed lease term is for a major part of the remaining economic life of the underlying asset and whether the present value of lease payments represents substantially all of the fair value of the underlying asset. The Company applies the bright line thresholds referenced in ASC 842-10-55-2 to assist in evaluating leases for appropriate classification. The aforementioned bright lines are applied consistently to the Company’s entire portfolio of leases. Revenue recognition Effective January 1, 2018, the Company adopted ASC Topic 606, Revenue from Contracts with Customers (“Topic 606”), using the modified retrospective transition method. Under this method, the Company has recognized the cumulative effect of the adoption as an adjustment to the opening balance of accumulated deficit in the current period consolidated balance sheet. The Company has not revised its consolidated financial statements for prior periods. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as collaboration arrangements and leases. Under Topic 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration that the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect the consideration to which it is entitled in exchange for the goods or services it transfers to the customer. Once a contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and if so, they are considered performance obligations. The identification of material rights requires judgments related to the determination of the value of the underlying license relative to the option exercise price, including assumptions about technical feasibility and the probability of developing a candidate that would be subject to the option rights. The exercise of a material right is accounted for as a contract modification for accounting purposes. The Company assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract. This assessment involves subjective determinations and requires management to make judgments about the individual promised goods or services and whether such are separable from the other aspects of the contractual relationship. Promised goods and services are considered distinct provided that: (i) the customer can benefit from the good or service either on its own or together with other resources that are readily available to the customer (that is, the good or service is capable of being distinct) and (ii) the entity’s promise to transfer the good or service to the customer is separately identifiable from other promises in the contract (that is, the promise to transfer the good or service is distinct within the context of the contract). In assessing whether a promised good or service is distinct, the Company considers factors such as the research, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. The Company also considers the intended benefit of the contract in assessing whether a promised good or service is separately identifiable from other promises in the contract. If a promised good or service is not distinct, an entity is required to combine that good or service with other promised goods or services until it identifies a bundle of goods or services that is distinct. The transaction price is then determined and allocated to the identified performance obligations in proportion to their standalone selling prices (“SSP”) on a relative SSP basis. SSP is determined at contract inception and is not updated to reflect changes between contract inception and when the performance obligations are satisfied. Determining the SSP for performance obligations requires significant judgment. In developing the SSP for a performance obligation, the Company considers applicable market conditions and relevant entity-specific factors, including factors that were contemplated in negotiating the agreement with the customer and estimated costs. The Company validates the SSP for performance obligations by evaluating whether changes in the key assumptions used to determine the SSP will have a significant effect on the allocation of arrangement consideration between multiple performance obligations. If the consideration promised in a contract includes a variable amount, the Company estimates the amount of consideration to which it will be entitled in exchange for transferring the promised goods or services to a customer. The Company determines the amount of variable consideration by using the expected value method or the most likely amount method. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis in the period of adjustment. If an arrangement includes development and regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the Company’s control or the licensee’s control, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. For arrangements with licenses of intellectual property that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes royalty revenue and sales-based milestones at the later of (i) when the related sales occur, or (ii) when the performance obligation to which the royalty has been allocated has been satisfied. In determining the transaction price, the Company adjusts consideration for the effects of the time value of money if the timing of payments provides the Company with a significant benefit of financing. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. The Company assessed each of its revenue generating arrangements in order to determine whether a significant financing component exists and concluded that a significant financing component does not exist in any of its arrangements. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time, and if over time recognition is based on the use of an output or input method. Collaboration revenue To date, the Company’s collaboration revenue has been generated from its collaboration arrangements with BMS and Regeneron Pharmaceuticals, Inc. (“Regeneron”), as further described in Note 11, Collaborative arrangements . The Company analyzes its collaboration arrangements to assess whether they are within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) to determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For collaboration arrangements within the scope of ASC 808 that contain multiple elements, the Company first determines which elements of the collaboration are deemed to be within the scope of ASC 808 and those that are more reflective of a vendor-customer relationship and therefore within the scope of Topic 606. For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, generally by analogy to Topic 606. Amounts that are owed to collaboration partners are recognized as an offset to collaboration revenues as such amounts are incurred by the collaboration partner. Where amounts owed to a collaboration partner exceed the Company’s collaboration revenues in each quarterly period, such amounts are classified as research and development expense. For those elements of the arrangement that are accounted for pursuant to Topic 606, the Company applies the five-step model described above. License and royalty revenue The Company enters into out-licensing agreements that are within the scope of Topic 606. The Company does not have any material license arrangements that contain more than one performance obligation. The terms of such out-license agreements include the license of functional intellectual property, given the functionality of the intellectual property is not expected to change substantially as a result of the licensor’s ongoing activities, and typically include payment of one or more of the following: non-refundable up-front license fees; development and regulatory milestone payments and milestone payments based on the level of sales; and royalties on net sales of licensed products. Nonrefundable up-front license fees are recognized as revenue at a point in time when the licensed intellectual property is made available for the customer’s use and benefit, which is generally at the inception of the arrangement. Development and regulatory milestone fees, which are a type of variable consideration, are recognized as revenue to the extent that it is probable that a significant reversal will not occur. The Company recognizes royalty revenue and sales-based milestones at the later of (i) when the related sales occur, or (ii) when the performance obligation to which the royalty has been allocated has been satisfied. For a complete discussion of accounting for collaboration and other revenue-generating arrangements, see Note 11, Collaborative arrangements, and Note 12, License and royalty revenue . Research and development expenses Research and development costs are charged to expense as costs are incurred in performing research and development activities, including salaries and benefits, facilities costs, overhead costs, clinical study and related clinical manufacturing costs, license and milestone fees, contract services, manufacturing costs for pre-launch inventory that did not qualify for capitalization, and other related costs. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed as research and development expense as incurred. In circumstances where amounts have been paid in excess of costs incurred, the Company records a prepaid expense. Th |
Marketable securities
Marketable securities | 12 Months Ended |
Dec. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable securities | Marketable securitiesThe following table summarizes the marketable securities held at December 31, 2019 and 2018 (in thousands): Amortized cost / cost Unrealized gains Unrealized losses Fair value December 31, 2019 U.S. government agency securities and treasuries $ 633,970 $ 2,014 $ (48) $ 635,936 Certificates of deposit 960 — — 960 Corporate bonds 185,827 824 (43) 186,608 Commercial paper 74,378 — — 74,378 Equity securities 20,017 — (7,147) 12,870 Total $ 915,152 $ 2,838 $ (7,238) $ 910,752 December 31, 2018 U.S. government agency securities and treasuries $ 1,459,649 $ 963 $ (3,011) $ 1,457,601 Certificates of deposit 9,080 — — 9,080 Equity securities 20,017 2,150 — 22,167 Total $ 1,488,746 $ 3,113 $ (3,011) $ 1,488,848 No available-for-sale debt securities held as of December 31, 2019 or 2018 had remaining maturities greater than three years. |
Fair value measurements
Fair value measurements | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair value measurements | Fair value measurements The following table sets forth the Company’s assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2019 and 2018 (in thousands): Total Quoted Significant Significant December 31, 2019 Assets: Cash and cash equivalents $ 327,214 $ 311,245 $ 15,969 $ — Marketable securities: U.S. government agency securities and treasuries 635,936 — 635,936 — Certificates of deposit 960 — 960 — Commercial paper 74,378 — 74,378 — Corporate bonds 186,608 — 186,608 — Equity securities 12,870 12,870 — — Total assets $ 1,237,966 $ 324,115 $ 913,851 $ — Liabilities: Contingent consideration $ 7,977 $ — $ — $ 7,977 Total liabilities $ 7,977 $ — $ — $ 7,977 December 31, 2018 Assets: Cash and cash equivalents $ 402,579 $ 348,638 $ 53,941 $ — Marketable securities: U.S. government agency securities 1,457,601 — 1,457,601 — Certificates of deposit 9,080 — 9,080 — Equity securities 22,167 22,167 — — Total assets $ 1,891,427 $ 370,805 $ 1,520,622 $ — Liabilities: Contingent consideration $ 5,230 $ — $ — $ 5,230 Total liabilities $ 5,230 $ — $ — $ 5,230 Cash and cash equivalents The Company considers all highly liquid securities with original final maturities of 90 days or less from the date of purchase to be cash equivalents. As of December 31, 2019, cash and cash equivalents comprise funds in cash, commercial paper, and money market accounts. As of December 31, 2018, cash and cash equivalents comprise funds in cash, U.S. Treasury securities, U.S. government agency securities, and money market accounts. Marketable securities Marketable securities classified as Level 2 within the valuation hierarchy generally consist of certificates of deposit, U.S. Treasury securities and government agency securities, corporate bonds, and commercial paper. The Company estimates the fair values of these marketable securities by taking into consideration valuations obtained from third-party pricing sources. These pricing sources utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include market pricing based on real-time trade data for the same or similar securities, issuer credit spreads, benchmark yields, and other observable inputs. The Company validates the prices provided by its third-party pricing sources by understanding the models used, obtaining market values from other pricing sources and analyzing pricing data in certain instances. The amortized cost of available-for-sale debt securities is adjusted for amortization of premiums and accretion of discounts to the earliest call date for premiums or to maturity for discounts. At December 31, 2019 and 2018, the balance in the Company’s accumulated other comprehensive loss was composed primarily of activity related to the Company’s debt securities. There were no material realized gains or losses recognized on the sale or maturity of available-for-sale securities during the year ended December 31, 2019 or 2018, and as a result, the Company did not reclassify any amounts out of accumulated other comprehensive loss for the same periods. The aggregate fair value of debt securities held by the Company in an unrealized loss position for less than twelve months as of December 31, 2019 and 2018 was $67.2 million and $787.5 million, respectively. As of December 31, 2019, and 2018, there were $79.6 million and $315.3 million in securities held by the Company in an unrealized loss position for more than twelve months, respectively. The aggregate unrealized loss on securities held by the Company for less than twelve months as of December 31, 2019 and 2018 was less than $0.1 million and $0.9 million, respectively. The aggregate unrealized loss on securities held by the Company for more than twelve months as of December 31, 2019 and 2018 was less than $0.1 million and $2.1 million, respectively. The Company has the intent to hold such securities until recovery and it is not more likely than not that the Company will be required to sell the securities before recovery of their amortized costs bases, which may be maturity. The Company determined that there was no material change in the credit risk of the above investments. As a result, the Company determined it did not hold any investments with an other-than-temporary impairment as of December 31, 2019 and 2018. The Company holds equity securities with an aggregate fair value of $12.9 million and $22.2 million at December 31, 2019 and 2018, respectively within short-term marketable securities on its consolidated balance sheet. The Company recorded a $9.3 million unrealized loss and a $2.2 million unrealized gain during the year ended December 31, 2019 and 2018, respectively, related to its equity securities, which is included in other (expense) income, net on the consolidated statements of operations and comprehensive loss. Contingent consideration In connection with its prior acquisition of Precision Genome Engineering, Inc. (“Pregenen”), the Company may be required to pay future consideration that is contingent upon the achievement of specified development, regulatory approvals or sales-based milestone events. Contingent consideration is measured at fair value and is based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. The valuation of contingent consideration uses assumptions the Company believes would be made by a market participant. The Company assesses these estimates on an on-going basis as additional data impacting the assumptions is obtained. Future changes in the fair value of contingent consideration related to updated assumptions and estimates are recognized within the consolidated statements of operations and comprehensive loss. Contingent consideration may change significantly as development progresses and additional data are obtained, impacting the Company’s assumptions regarding probabilities of successful achievement of related milestones used to estimate the fair value of the liability and the timing in which they are expected to be achieved. In evaluating the fair value information, considerable judgment is required to interpret the market data used to develop the estimates. The estimates of fair value may not be indicative of the amounts that could be realized in a current market exchange. Accordingly, the use of different market assumptions and/or different valuation techniques could result in materially different fair value estimates. The significant unobservable inputs used in the measurement of fair value of the Company’s contingent consideration are probabilities of successful achievement of clinical and commercial milestones, the period in which these milestones are expected to be achieved ranging from 2021 to 2028 and discount rates ranging from 12.7% to 14.3%. Significant increases or decreases in any of these inputs would result in a significantly higher or lower fair value measurement. The table below provides a roll-forward of fair value of the Company’s contingent consideration obligations which include Level 3 inputs (in thousands): Year ended December 31, 2019 2018 Beginning balance $ 5,230 $ 2,231 Additions — — Changes in fair value 2,747 2,999 Payments — — Ending balance $ 7,977 $ 5,230 Please refer to Note 9, Commitments and contingencies, for further information. |
Property, plant and equipment,
Property, plant and equipment, net | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property, plant and equipment, net | Property, plant and equipment, net Property, plant and equipment, net, consists of the following (in thousands): As of December 31, 2019 2018 Land $ 1,210 $ 1,210 Building 15,664 180,094 Computer equipment and software 6,947 6,365 Office equipment 7,599 5,584 Laboratory equipment 44,560 35,693 Leasehold improvements 33,788 183 Construction-in-progress 77,981 46,669 Total property, plant and equipment 187,749 275,798 Less accumulated depreciation and amortization (36,573) (29,176) Property, plant and equipment, net $ 151,176 $ 246,622 Depreciation and amortization expense related to property, plant and equipment was $13.4 million, $13.4 million, and $9.8 million for the years ended December 31, 2019, 2018, and 2017, respectively. North Carolina manufacturing facility In November 2017, the Company acquired a manufacturing facility in Durham, North Carolina for the future manufacture of lentiviral vector for the Company’s gene therapies. As of December 31, 2019, a portion of the facility has been placed into service and the remainder of the facility is still in process of construction. Construction-in-progress as of December 31, 2019, and 2018, includes $74.2 million and $40.4 million, respectively, related to the North Carolina manufacturing facility. 60 Binney Street Lease As a result of the adoption of ASU 2016-02, the Company de-recognized $156.0 million of the building asset and $6.7 million of accumulated depreciation related to its corporate headquarters at 60 Binney Street. Prior to the adoption of ASU 2016-02, the Company classified leasehold improvements associated with the 60 Binney Street building as building assets. Subsequent to the adoption of ASU 2016-02, the leasehold improvements owned by the Company associated with the 60 Binney Street building are classified as leasehold improvements. Please refer to Note 2, Summary of significant accounting policies and basis of presentation, and Note 8, Leases, for further information. |
Restricted cash
Restricted cash | 12 Months Ended |
Dec. 31, 2019 | |
Other Assets, Noncurrent [Abstract] | |
Restricted cash | Restricted cash As of December 31, 2019, and 2018, the Company maintained letters of credit of $54.5 million and $14.5 million, respectively, which are collateralized with a bank account at a financial institution in accordance with the agreement and consisted of the following (in thousands): As of December 31, 2019 2018 60 Binney Street lease $ 13,763 $ 13,763 50 Binney Street sublease 40,072 — Other leases 660 757 Total restricted cash $ 54,495 $ 14,520 Refer to Note 8, Leases, for further information on the Company's letters of credit. |
Accrued expenses and other curr
Accrued expenses and other current liabilities | 12 Months Ended |
Dec. 31, 2019 | |
Payables and Accruals [Abstract] | |
Accrued expenses and other current liabilities | Accrued expenses and other current liabilities Accrued expenses and other current liabilities consist of the following (in thousands): As of December 31, 2019 2018 Employee compensation $ 44,679 $ 28,567 Manufacturing costs 23,126 21,618 Clinical and contract research organization costs 16,799 11,891 Collaboration research costs 27,142 3,358 Property, plant, and equipment 2,354 5,451 License and milestone fees 300 7,739 Professional fees 1,827 1,830 Financing lease obligation, current portion — 1,424 Other 25,329 17,515 Total accrued expenses and other current liabilities $ 141,556 $ 99,393 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Leases | Leases The Company leases certain office and laboratory space. Additionally, the Company has embedded leases at contract manufacturing organizations. 60 Binney Street lease On September 21, 2015, the Company entered into a lease agreement for office and laboratory space located in a building (the “Building”) at 60 Binney Street, Cambridge, Massachusetts (the “60 Binney Street Lease”), which is now the Company’s corporate headquarters. Under the terms of the 60 Binney Street Lease, starting on October 1, 2016, the Company leases approximately 253,108 square feet of office and laboratory space at $72.50 per square foot per year, or $18.4 million per year in base rent, which is subject to scheduled annual rent increases of 1.75% plus certain operating expenses and taxes. The Company currently maintains a $13.8 million collateralized letter of credit and, subject to the terms of the lease and certain reduction requirements specified therein, including market capitalization requirements, this amount may decrease to $9.2 million over time. Pursuant to a work letter entered into in connection with the 60 Binney Street Lease, the landlord contributed an aggregate of $42.4 million toward the cost of construction and tenant improvements for the Building. The Company occupied the Building beginning on March 27, 2017 and the 60 Binney Street Lease will continue until March 31, 2027. The Company has the option to extend the 60 Binney Street Lease for two successive five Due to the Company’s involvement in the construction project, including having responsibility to pay for a portion of the costs of finish work and mechanical, electrical, and plumbing elements of the Building, among other items, the Company was deemed for accounting purposes to be the owner of the Building during the construction period, per ASC 840. Accordingly, under ASC 840, construction costs that were incurred by the landlord directly or indirectly through reimbursement to the Company as part of its tenant improvement allowance were recorded as an asset in property, plant and equipment, net on the Company’s consolidated balance sheets. The Company evaluated the 60 Binney Street Lease upon occupancy on March 27, 2017 and determined that the 60 Binney Street Lease did not meet the criteria for “sale-leaseback” treatment under ASC 840. This determination was based on, among other things, the Company's continuing involvement with the property in the form of non-recourse financing to the lessor. Accordingly, upon occupancy, the Company commenced depreciating the portion of the building in service over a useful life of 40 years and incurred interest expense related to the financing obligation. As part of its adoption of ASC 842, the Company de-recognized the building asset and corresponding financing obligation recorded on the Company’s consolidated balance sheets as of December 31, 2018, in accordance with the ASC 842 transition guidance. In applying the ASC 842 transition guidance, the Company classified this lease as an operating lease and recorded a right-of-use asset of $127.3 million and lease liability of $125.8 million on the effective date. The Company is recognizing rent expense on a straight-line basis throughout the remaining term of the lease. 50 Binney Street sublease In April 2019, the Company entered into a sublease agreement for office space located at 50 Binney Street in Cambridge, Massachusetts (the “50 Binney Street Sublease”) to supplement the Company’s corporate headquarters located at 60 Binney Street in Cambridge, Massachusetts. Under the terms of the 50 Binney Street Sublease, the Company will lease 267,278 square feet of office space for $99.95 per square foot, or $26.7 million per year in base rent subject to certain operating expenses, taxes and annual rent increases of approximately 3%. The lease will commence when the space is available for use by the Company, which is anticipated to be in the second half of 2021, and end on December 31, 2030, unless the Company earlier occupies the premises or other conditions specified in the 50 Binney Street Sublease occur. The sublessor has the right to postpone the commencement date until January 1, 2022 by providing not less than nine months’ prior written notice to the Company. Upon signing the 50 Binney Street Sublease, the Company executed a $40.1 million cash-collateralized letter of credit, which may be reduced in the future subject to the terms of the 50 Binney Street Sublease and certain reduction requirements specified therein. The $40.1 million of cash collateralizing the letter of credit is classified as restricted cash and other non-current assets on the Company’s consolidated balance sheets. Payments will commence at the earlier of (i) the date which is 90 days following the commencement date and (ii) the date the Company takes occupancy of all or any portion of the premises. In connection with the execution of the 50 Binney Street Sublease, the Company also entered into a Purchase Agreement for furniture and equipment (the “Furniture Purchase Agreement”) located on the premises upon lease commencement. Upon execution of the Furniture Purchase Agreement, the Company made an upfront payment of $7.5 million, all of which was recorded within restricted cash and other non-current assets on the Company’s consolidated balance sheets as of December 31, 2019. The Company will assess the lease classification of the 50 Binney Street Sublease and commence recognition of the associated rent expense upon lease commencement. Seattle, Washington leases In July 2018, the Company entered into a lease agreement for office and laboratory space located in a portion of a building in Seattle, Washington. The lease was amended in October 2018 to increase the total rentable space to approximately 36,126 square feet at $54.00 per square foot in base rent per year, which is subject to scheduled annual rent increases of 2.5% plus certain operating expenses and taxes. The lease commenced on January 1, 2019 and the lease term will continue through January 31, 2027 ("the Initial Term"). The Company moved into the facility in June 2019. The lease allowed for a tenant improvement (“TI”) allowance of up to $215.00 per square foot, or approximately $8.0 million. The Company utilized the $8.0 million TI allowance and it has been fully reimbursed by the landlord as of December 31, 2019. The Company determined the classification of this lease to be an operating lease and recorded a right-of-use asset and lease liability at lease commencement. The Company was determined to be the owner of the tenant improvements and as such recorded the building improvements as property, plant, and equipment, net, and began to depreciate the building improvements over the remaining lease term at such time the assets were placed into service. In September 2019, the Company entered into a second amendment to the lease (the “Second Amendment”). The Second Amendment added approximately 22,188 square feet to the existing space and extended the lease term of the entire premises by 16 months, or until April 2028. Fixed monthly rent for the expanded space will be incurred at a rate of $62.80 per square foot per year beginning in January 2021, subject to annual increases of 2.5%. The Second Amendment includes a five extend the term. The Company is recognizing rent expense on a straight-line basis through the remaining extended term of the lease. Upon the execution of the Second Amendment, which was deemed to be a lease modification, the Company re-evaluated the assumptions made at the original lease commencement date. The Company determined the Second Amendment consists of two separate contracts under ASC 842. One contract is related to a new right-of-use for the expanded 22,188 square feet of space, which is to be accounted for as a new lease, and the other is related to the modification of term for the original 36,126 square feet of space. The Company recorded an additional right-of-use asset and lease liability upon lease commencement of the expanded space. Embedded leases On June 3, 2016, the Company entered into a manufacturing agreement for the future commercial production of the Company’s LentiGlobin and Lenti-D drug products with a contract manufacturing organization. Under this 12-year agreement, the contract manufacturing organization will complete the design, construction, validation and process validation of the leased suites prior to anticipated commercial launch of the product candidates. During construction, the Company paid $12.0 million upon the achievement of certain contractual milestones, and may pay up to $8.0 million in additional contractual milestones if the Company elects its option to lease additional suites. Construction was completed in March 2018 and beginning in April 2018 the Company pays $5.1 million per year in fixed suite fees, as well as certain fixed labor, raw materials, testing and shipping costs for manufacturing services, and may pay additional suite fees if it elects its option to reserve or lease additional suites. The Company may terminate this agreement at any time upon payment of a one-time termination fee and up to 24 months of fixed suite and labor fees. The Company concluded in prior periods that this agreement contained an embedded lease under ASC 840 as the suites are designated for the Company’s exclusive use during the term of the agreement. The Company concluded that it was not the deemed owner during construction nor was it a capital lease under ASC 840. As a result, in prior periods the Company accounted for the agreement as an operating lease under ASC 840 and recognized straight-line rent expense over the non-cancellable term of the embedded lease. As part of its adoption of ASC 842, effective January 1, 2019, the Company carried forward its existing lease classification under ASC 840. Additionally, the Company recorded a right-of-use asset and lease liability for this operating lease on the effective date and is recognizing rent expense on a straight-line basis throughout the remaining term of the embedded lease. The Company’s other embedded leases are not material to the consolidated financial statements. Summary of all lease costs recognized under ASC 842 The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the year ended December 31, 2019 (in thousands): For the year ended December 31, 2019 Lease cost (1) Operating lease cost $ 35,346 Total lease cost $ 35,346 Other information Operating cash flows used for operating leases $ 31,026 Weighted average remaining lease term 7.4 years Weighted average discount rate 6.70 % (1) Short-term lease costs and variable lease costs incurred by the Company for the twelve months ended December 31, 2019 were immaterial. Rent expense is calculated on a straight-line basis over the term of the lease. Rent expense recognized under all leases, including additional charges for utilities, parking, maintenance, and real estate taxes was $45.3 million, $9.8 million, and $9.0 million for the years ended December 31, 2019, 2018 and 2017, respectively. Note that the Company adopted ASC 842 effective January 1, 2019 using the required modified retrospective approach and utilizing the effective date as its date of initial application. Therefore, amounts disclosed pertaining to the years ended December 31, 2018 and 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under ASC 842. As of December 31, 2019, future minimum commitments under ASC 842 under the Company’s operating leases were as follows (in thousands): As of Maturity of lease liabilities December 31, 2019 2020 $ 33,257 2021 35,816 2022 31,052 2023 31,540 2024 31,552 2025 and thereafter 88,336 Total lease payments 251,553 Less: imputed interest (60,566) Total operating lease liabilities $ 190,987 The above table excludes legally binding minimum lease payments for leases executed but not yet commenced as of December 31, 2019. |
Commitments and contingencies
Commitments and contingencies | 12 Months Ended |
Dec. 31, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and contingencies | Commitments and contingencies Lease commitments The Company leases certain office and laboratory space and has embedded leases at contract manufacturing organizations. Refer to Note 8, Leases, for further information on the terms of these lease agreements. Contingent consideration related to business combinations On June 30, 2014, the Company acquired Pregenen. The Company may be required to make up to an additional $120.0 million in remaining future contingent cash payments to the former equityholders of Pregenen upon the achievement of certain clinical and commercial milestones related to the Pregenen technology, of which $20.1 million relates to clinical milestones and $99.9 million relates to commercial milestones. In accordance with accounting guidance for business combinations, contingent consideration liabilities are required to be recognized on the consolidated balance sheets at fair value. Estimating the fair value of contingent consideration requires the use of significant assumptions primarily relating to probabilities of successful achievement of certain clinical and commercial milestones, the expected timing in which these milestones will be achieved and discount rates. The use of different assumptions could result in materially different estimates of fair value. See Note 4, Fair value measurements, for additional information. Other funding commitments The Company is party to various agreements, principally relating to licensed technology, that require future payments relating to milestones that may be met in subsequent periods or royalties on future sales of specified products, which includes the collaboration agreement entered into with Regeneron in August 2018. Additionally, the Company is party to various contracts with contract research organizations and contract manufacturers that generally provide for termination on notice, with the exact amounts in the event of termination to be based on the timing of the termination and the terms of the agreement. Please refer to Note 11, Collaborative arrangements, for further information on the collaboration agreement with Regeneron. Based on our development plans as of December 31, 2019, we may be obligated to make future development, regulatory and commercial milestone payments and royalty payments on future sales of specified products associated with the Company's collaboration and license agreements. Payments under these agreements generally become due and payable upon achievement of such milestones or sales. When the achievement of these milestones or sales have not occurred, such contingencies are not recorded in the Company’s financial statements and are excluded from the table below. The Company has various manufacturing development and license agreements to support clinical and commercial product needs. The following table presents non-cancelable contractual obligations arising from these arrangements: Years ended December 31, Purchase 2020 $ 129,950 2021 22,597 2022 24,310 2023 25,040 2024 46,050 2025 and thereafter — Total purchase commitments $ 247,947 The Company enters into agreements containing standard indemnification provisions in the ordinary course of business. Pursuant to such terms, the Company indemnifies, holds harmless, and agrees to reimburse the indemnified party for losses suffered or incurred by the indemnified party, generally the Company’s business partners. The term of these indemnification agreements is generally perpetual any time after execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is unlimited. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements. In January 2020, the Company executed an amendment to an existing contract manufacturing arrangement, which extended the term of the contract by one year, through December 31, 2021, which increases the Company's contractual obligations by an additional $24.7 million to the table above. |
Common stock and preferred stoc
Common stock and preferred stock | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
Common stock and preferred stock | Common stock and preferred stock The Company is authorized to issue 125.0 million shares of common stock. Holders of common stock are entitled to one vote per share. Holders of common stock are entitled to receive dividends, if and when declared by the Company’s board of directors, and to share ratably in the Company’s assets legally available for distribution to the Company’s shareholders in the event of liquidation. Holders of common stock have no preemptive, subscription, redemption or conversion rights. As of December 31, 2019, and 2018, the Company had 55.4 million and 54.7 million shares of common stock issued and outstanding, respectively. In January 2018, the Company sold 0.3 million shares of common stock pursuant to the partial exercise of an overallotment option granted to the underwriters in connection with the December 2017 underwritten public offering at a price of $185.00 per share for aggregate net proceeds of $48.7 million. In July 2018, the Company sold 3.9 million shares of common stock (excluding any shares sold pursuant to an overallotment option granted to the underwriters in connection with the offering) through an underwritten public offering at a price of $162.50 per share for aggregate net proceeds of $600.6 million. In August 2018, the Company sold 0.4 million shares of common stock to Regeneron in connection with a collaboration arrangement at a price of $238.10 per share for aggregate net proceeds of $100.0 million, of which $45.5 million was attributed to a prepayment of joint research activities. The Company is authorized to issue 5.0 million shares of preferred stock in one or more series and to fix the powers, designations, preferences and relative participating option or other rights thereof, including dividend rights, conversion rights, voting rights, redemption terms, liquidation preferences and the number of shares constituting any series, without any further vote or action by the Company’s shareholders. As of December 31, 2019 and 2018, the Company had no shares of preferred stock issued or outstanding. Reserved for future issuance The Company has reserved for future issuance the following number of shares of common stock (in thousands): As of December 31, 2019 2018 Options to purchase common stock 5,483 4,643 Restricted stock units 1,127 931 2013 Stock Option and Incentive Plan 2,007 1,458 2013 Employee Stock Purchase Plan 147 172 8,764 7,204 |
Collaborative arrangements
Collaborative arrangements | 3 Months Ended | 12 Months Ended |
Jun. 30, 2019 | Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Collaborative arrangements | Collaborative arrangements To date, the Company’s collaboration revenue has been primarily generated from its collaboration arrangements with Bristol-Myers Squibb ("BMS"), formerly Celgene Corporation ("Celgene") prior to its acquisition by BMS in November 2019, and Regeneron, each as further described below. Bristol-Myers Squibb BMS Original Collaboration Agreement On March 19, 2013, the Company entered into a Master Collaboration Agreement (the "BMS Collaboration Agreement”) with Celgene (now BMS following its acquisition of Celgene in November 2019) to discover, develop and commercialize potentially disease-altering gene therapies in oncology. The collaboration is focused on applying gene therapy technology to genetically modify a patient’s own T cells, known as chimeric antigen receptor, or CAR T cells, to target and destroy cancer cells. Additionally, on March 19, 2013, the Company entered into a Platform Technology Sublicense Agreement (the “Sublicense Agreement”) with BMS pursuant to which the Company obtained a sublicense to certain intellectual property from BMS, originating under BMS’s license from Baylor College of Medicine, for use in the collaboration. Under the terms of the BMS Collaboration Agreement, the Company received a $75.0 million up-front, non-refundable cash payment. The Company was responsible for conducting discovery, research and development activities through completion of phase 1 clinical studies, if any, during the initial term of the BMS Collaboration Agreement, or three years. The collaboration is governed by a joint steering committee (“JSC”) formed by an equal number of representatives from the Company and BMS. The JSC, among other activities, reviews the collaboration program, reviews and evaluates product candidates and approves regulatory plans. In addition to the JSC, the BMS Collaboration Agreement provides that the Company and BMS each appoint representatives to a patent committee, which is responsible for managing the intellectual property developed and used during the collaboration. BMS Amended Collaboration Agreement On June 3, 2015, the Company and BMS amended and restated the BMS Collaboration Agreement (the “Amended BMS Collaboration Agreement”). Under the Amended BMS Collaboration Agreement, the parties narrowed the focus of the collaboration to exclusively work on anti- B-cell maturation antigen (“BCMA”) product candidates for a new three On a product candidate-by-product candidate basis, up through a specified period following enrollment of the first patient in an initial phase 1 clinical study for such product candidate (the “Option Period”), the Company had granted BMS an option to obtain an exclusive worldwide license to develop and commercialize such product. Following BMS’s license of each product candidate, the Company is entitled to elect to co-develop and co-promote each product candidate in the U.S. BMS Ide-cel License Agreement On February 10, 2016, BMS exercised its option to obtain an exclusive worldwide license to develop and commercialize ide-cel, the first product candidate under the Amended BMS Collaboration Agreement, pursuant to an executed license agreement (“Ide-cel License Agreement”) entered into by the parties on February 16, 2016 and paid to the Company the associated $10.0 million option fee. Pursuant to the Ide-cel License Agreement, BMS was responsible for development and related funding of ide-cel after the substantial completion of the phase 1 clinical trial. The Company was responsible for the manufacture of vector and associated payload throughout development and upon BMS’s request, throughout commercialization, the costs of which were reimbursable by BMS in accordance with the terms of the Amended and Restated Co-Development, Co-Promote and Profit Share Agreement, as further described below. BMS was responsible for the manufacture of drug product throughout development and commercialization. BMS Ide-cel Co-Development, Co-Promote and Profit Share Agreement On March 28, 2018, the Company elected to co-develop and co-promote ide-cel within the U.S. pursuant to the execution of the Amended and Restated Co-Development, Co-Promote and Profit Share Agreement (“Ide-cel CCPS”), which replaced the Ide-cel License Agreement. The responsibilities of the parties remain unchanged from those under the Ide-cel License Agreement, however, the Company will share equally in all profits and losses relating to developing, commercializing and manufacturing ide-cel within the U.S. and has the right to participate in the development and promotion of ide-cel in the U.S. BMS is responsible for the costs incurred to manufacture vector and associated payload for use outside of the U.S., plus a markup. Under the Ide-cel CCPS, the Company may receive up to a total of $70.0 million in development milestone payments for the first indication to be addressed by ide-cel, with the ability to obtain additional milestone payments for a second indication and modified licensed products. In the second quarter of 2019, a $10.0 million development milestone was achieved such that as of December 31, 2019, the total remaining potential development milestones on the first indication of ide-cel is $60.0 million. In addition, to the extent ide-cel is commercialized, the Company is entitled to receive tiered royalty payments ranging from the mid-single digits to low-teens based on a percentage of net sales generated outside of the U.S., subject to certain reductions. BMS bb21217 License Agreement On September 22, 2017, BMS exercised its option to obtain an exclusive worldwide license to develop and commercialize bb21217, the second product candidate under the Amended BMS Collaboration Agreement, pursuant to an executed license agreement (“bb21217 License Agreement”) entered into by the parties on September 28, 2017 and paid the Company an option fee of $15.0 million. Pursuant to the bb21217 License Agreement, BMS is responsible for development and related funding of bb21217 after the substantial completion of the ongoing phase 1 clinical trial. In 2019, the parties amended the protocol for the ongoing phase 1 clinical trial to enroll additional patients. The Company is responsible for the manufacture of vector and associated payload throughout development and upon BMS’s request, throughout commercialization. Expenses incurred by the Company associated with these activities are fully reimbursable by BMS at cost plus a mark-up. Throughout both development and commercialization, BMS is responsible for the manufacture of drug product. The Company currently expects it will exercise its option to co-develop and co-promote bb21217 within the U.S. The Company’s election to co-develop and co-promote bb21217 must be made by the substantial completion of the on-going phase 1 clinical trial of bb21217. If elected, the Company expects the responsibilities of the parties to remain largely unchanged, however, the Company expects it will share equally in all profits and losses relating to developing, commercializing and manufacturing bb21217 within the U.S. and to have the right to participate in the development and promotion of bb21217 in the U.S. BMS would be responsible for the costs incurred to manufacture vector and associated payload for use outside of the U.S., plus a markup. Under this scenario, the Company expects to receive, per product, up to $70.0 million in development milestone payments for the first indication to be addressed by the bb21217 product candidate, with the ability to obtain additional milestone payments for a second indication and modified licensed products. In addition, to the extent bb21217 is commercialized, the Company would be entitled to receive tiered royalty payments ranging from the mid-single digits to low-teens based on a percentage of net sales generated outside of the U.S., subject to certain reductions. In the event the Company does not exercise its option to co-develop and co-promote bb21217, the Company will receive an additional fee in the amount of $10.0 million. Under this scenario, the Company may be eligible to receive up to $10.0 million in clinical milestone payments, up to $117.0 million in regulatory milestone payments, and up to $78.0 million in commercial milestone payments. In addition, to the extent bb21217 is commercialized, the Company would be entitled to receive tiered royalty payments ranging from the mid-single digits to low-teens based on a percentage of net sales, subject to certain reductions. Accounting Analysis – Ide-cel ASC Topic 606, Revenue from Contracts With Customers ("Topic 606"), allows entities to reflect the aggregate effect of all contract modifications when identifying the satisfied and unsatisfied performance obligations for contracts that were modified prior to Topic 606 adoption. BMS’s option to in-license the first product candidate, ide-cel, under the arrangement was considered a material right at the time the Amended BMS Collaboration Agreement was executed in June 2015 given the product candidate had been formally nominated by the JSC and that substantially all investigational new drug application, or IND, enabling activities had been completed by that time. In making this determination, the Company also considered the option price relative to the value of the underlying license. BMS’s exercise of this material right in February 2016 was determined to represent a contract modification and represents the last contract modification prior to the adoption of Topic 606. As a result, the BMS Collaboration Agreement, Amended BMS Collaboration Agreement, and Ide-cel CCPS are combined for accounting purposes and treated as a single arrangement. As of February 2016, BMS’s option to license an additional product candidate under the collaboration did not represent a material right due primarily to the significant uncertainty regarding whether any additional product candidates would be identified under the Amended BMS Collaboration Agreement. Therefore, the license to the Company’s second product candidate, bb21217, which was executed in September 2017, is accounted for as a separate contract. Refer below for discussion of the bb21217 accounting analysis. As of the February 2016 contract modification date, the Company concluded the arrangement contained the following promised goods and services: (i) research and development services, (ii) a license to ide-cel, and (iii) manufacture of vectors and associated payload for incorporation into ide-cel through development. The Company determined that the manufacture of commercial vector represented an option to acquire additional goods and services that is not representative of a material right. In addition, as of the February 2016 contract modification date, BMS had not exercised its option to purchase any commercial vector. Accordingly, the manufacture of commercial vector was not considered to be a performance obligation at that time. The Company concluded that the research and development services are distinct from the other promised goods and services under the arrangement given that BMS can benefit from the research and development services on their own and such services are distinct within the context of the contract. Thus, such services are considered to be a separate performance obligation. The Company concluded that the license to ide-cel is not distinct from the vector manufacturing services because the manufacturing is essential to the use of the license. Accordingly, these two promised goods and services are considered a single combined performance obligation. Ide-cel transaction price The following tables summarize the total transaction price, the allocation of the total transaction price to the identified performance obligations under the arrangement, and the amount of the transaction price unsatisfied as of December 31, 2019 (in thousands): Ide-cel transaction price as of December 31, 2019 Up-front non-refundable payment - BMS Collaboration Agreement $ 75,000 Up-front non-refundable payment - Amended BMS Collaboration Agreement 25,000 Ide-cel license fee - Ide-cel License Agreement 10,000 Ide-cel development milestone 10,000 Estimated variable consideration 87,189 $ 207,189 Allocation of Transaction price unsatisfied as of December 31, 2019 Ide-cel research and development services $ 40,912 $ — Ide-cel license and manufacturing services 166,277 17,815 $ 207,189 $ 17,815 The estimated variable consideration of $87.2 million relates to the estimated reimbursement from BMS for the manufacture of vectors and associated payload through development. The total transaction price has been allocated to the performance obligations identified based on a relative standalone selling price ("SSP") basis. The Company estimated the SSP of the license after considering potential future cash flows under the license. The Company then discounted these probability-weighted cash flows to their present value. The Company estimated the SSP of each of the research and development services and manufacturing services to be provided based on the Company’s estimated cost of providing the services plus an applicable profit margin commensurate with observable market data for similar services. All of the clinical and regulatory milestones which have not been met as of period end are fully constrained and are excluded from the transaction price. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones is outside the control of the Company and contingent upon the future success of clinical trials, the licensee’s efforts, or the receipt of regulatory approval. Any consideration related to sales-based milestones (including royalties) will be recognized when the related sales occur as these amounts have been determined to relate predominantly to the license granted to BMS and therefore are recognized at the later of when the performance obligation is satisfied, or the related sales occur. The Company re-evaluates the transaction price, including its estimated variable consideration included in the transaction price and all constrained amounts, at each reporting period and as uncertain events are resolved or other changes in circumstances occur. Ide-cel research and development services The Company allocated $40.9 million of the transaction price to the research and development services. The Company satisfied this performance obligation as the research and development services were performed. The Company determined that the period of performance of the research and development services was three years through projected initial phase 1 clinical study substantial completion, or through May 2018. The Company recognized revenue related to research and development services performed using an input method by calculating costs incurred at each period end relative to total costs expected to be incurred. Although the Company fully satisfied this performance obligation during the second quarter of 2018, any changes to the total transaction price following the completion of this performance obligation in May 2018 will be allocated to the performance obligations under the arrangement based on a relative SSP basis and therefore the allocation of any changes to the total transaction price may impact the revenue recognized for this performance obligation in the period of change. The following table summarizes the revenue recognized, or revenue adjustment recorded, related to the ide-cel research and development services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (1) Ide-cel research and development services revenue $ 2,264 $ 5,751 $ 6,208 $ 2,264 $ 5,751 $ 6,208 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. Ide-cel license and manufacturing services The Company allocated $166.3 million of the transaction price to the combined unit of accounting which consists of the license and manufacture of vectors and associated payload for incorporation into ide-cel. The Company accounts for its vector manufacturing services for development in the U.S. and BMS’s U.S. development efforts within the scope of ASC 808 given that both parties are active participants in the activities and both parties are exposed to significant risks and rewards dependent on the commercial success of the activities. The Company recognizes collaboration revenue for its U.S. manufacturing services by analogy to Topic 606. The portion of BMS’s U.S. development costs that the Company is responsible for are recognized as a reduction to its collaboration revenues, or, if in excess of such revenues in a given quarter, the excess is recorded as research and development expense. Revenue recognition for the combined unit of accounting commenced during the first quarter of 2017. The Company recognizes revenue associated with the combined unit of accounting using the proportional performance method, as the Company will satisfy this performance obligation as the manufacturing services are performed through development. In using this method, the Company estimated its development plan for ide-cel, including expected demand from BMS, and the costs associated with the manufacture of vectors and associated payload for incorporation into ide-cel. On a quarterly basis, the Company determines the proportion of effort incurred as a percentage of total effort it expects to expend. This ratio is applied to the transaction price, which includes variable consideration, allocated to the combined performance obligation consisting of the ide-cel license and manufacturing services. Management has applied significant judgment in the process of developing its budget estimates and any changes to these estimates will be recognized in the period in which they change as a cumulative catch up. The following table summarizes the net collaboration revenue recognized or expense incurred for the joint ide-cel development efforts in the U.S. under ASC 808, including revenue or expense related to the combined performance obligation for the license and vector manufacturing of ide-cel in the U.S. for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (2) ASC 808 ide-cel revenue - U.S. (1) $ — $ 6,255 $ 4,905 ASC 808 ide-cel research and development expense - U.S. (1) $ 32,415 $ 8,689 $ 3,037 (1) As noted above, the calculation of collaboration revenue or research and development expense to be recognized for joint ide-cel development efforts in the U.S. is performed on a quarterly basis. The calculation is independent of previous activity, which may result in fluctuations between revenue and expense recognition period over period, depending on the varying extent of effort performed by each party during the period. (2) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. Revenue related to the combined unit of accounting for the non-US license and vector manufacturing services is accounted for in accordance with Topic 606. The following table summarizes the revenue recognized related to the combined unit of accounting for the ide-cel non-US license and vector manufacturing services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31 2019 2018 2017 (1) ASC 606 ide-cel license and manufacturing revenue - outside of U.S. $ 25,522 $ 35,900 $ 10,372 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. As of December 31, 2019, the aggregate amount of the transaction price allocated to the combined performance obligation, which consists of the ide-cel license and manufacturing services, that is unsatisfied, or partially unsatisfied, is $17.8 million, which the Company expects to recognize as revenue as manufacturing services are provided through the remaining development period which is estimated to be through 2020. As of December 31, 2019 and 2018, the Company had $8.5 million and $23.0 million, respectively, of deferred revenue associated with the combined performance obligation consisting of the ide-cel license and manufacturing services. Accounting Analysis – bb21217 On September 22, 2017, BMS exercised its option to obtain an exclusive worldwide license to develop and commercialize bb21217, the second optioned product candidate, pursuant to the bb21217 License Agreement entered into by the parties on September 28, 2017. The bb21217 License Agreement is considered a separate contract for accounting purposes as the option to obtain an exclusive worldwide license to develop and commercialize bb21217, or any other product candidate, was not considered a material right to BMS at the time the practical expedient was applied. The Company made this evaluation after considering the significant uncertainty at that time regarding whether any additional product candidates would be identified under the Amended BMS Collaboration Agreement. In particular, the Company considered that bb21217 had not been formally nominated as a product candidate under the collaboration at that time, primarily due to a lack of preclinical data as well as uncertainty surrounding the ability to successfully complete various IND-enabling activities. At contract inception, the Company concluded that the arrangement contained the following promised goods and services: (i) research and development services, (ii) a license to the second product candidate, bb21217, and (iii) manufacture of vectors and associated payload for incorporation into bb21217 through development. The Company determined that the manufacture of commercial vector represents an option to acquire additional goods and services that is not representative of a material right. In addition, at this time BMS has not exercised its option to purchase any commercial vector. Accordingly, the manufacture of commercial vector is not considered to be a performance obligation at this time. The Company concluded that the research and development services for the phase 1 clinical trial as originally contemplated by the parties are distinct from the other promised goods and services under the arrangement given that BMS can benefit from the research and development services on their own and such services are distinct within the context of the contract. Thus, such services are considered to be a separate performance obligation. Similar to ide-cel, the Company concluded that the license to bb21217 is not distinct from the vector manufacturing services because the manufacturing is essential to the use of the license. Accordingly, these two promised goods and services are considered a single combined performance obligation. The agreement to expand the bb21217 phase 1 trial is treated as a contract modification for accounting purposes because the trial expansion is the addition of a promised good or service that is distinct and the associated consideration reflects the standalone selling price of the additional promised good or service. It will be accounted for prospectively as a separate contract from the bb21217 License Agreement. The transaction price associated with these additional patients consists of variable consideration and is based upon an agreed-upon amount per patient which will be recognized as revenue as the patients are treated. The related performance obligation remained partially unsatisfied as of December 31, 2019. bb21217 License Agreement transaction price The following tables summarize the total transaction price, the allocation of the total transaction price to the identified performance obligations under the arrangement, and the amount of the transaction price unsatisfied as of December 31, 2019 (in thousands): bb21217 transaction price as of December 31, 2019 bb21217 license fee - bb21217 License Agreement $ 15,000 Estimated variable consideration 26,687 $ 41,687 Allocation of transaction Transaction price unsatisfied as of December 31, 2019 bb21217 research and development services $ 5,444 $ — bb21217 license and manufacturing services 36,243 36,243 $ 41,687 $ 36,243 The estimated variable consideration of $26.7 million relates to reimbursement from BMS for the manufacturing services during development. The total transaction price has been allocated to the performance obligations identified based on a relative SSP basis. The Company estimated the SSP of the license after considering potential future cash flows under the license. The Company then discounted these probability-weighted cash flows to their present value. The Company estimated the SSP of each of the research and development services and manufacturing services to be provided based on the Company’s estimated cost of providing the services plus an applicable profit margin commensurate with observable market data for similar services. All of the clinical and regulatory milestones are fully constrained and are excluded from the transaction price. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones is outside the control of the Company and contingent upon the future success of its clinical trials, the licensee’s efforts, or the receipt of regulatory approval. Any consideration related to sales-based milestones (including royalties) will be recognized when the related sales occur as these amounts have been determined to relate predominantly to the license granted to BMS and therefore are recognized at the later of when the performance obligation is satisfied, or the related sales occur. The Company re-evaluates the transaction price, including its estimated variable consideration included in the transaction price and all constrained amounts, each reporting period and as uncertain events are resolved or other changes in circumstances occur. bb21217 research and development services The Company allocated $5.4 million of the transaction price to the research and development services under the phase 1 trial as originally contemplated. The Company satisfied this performance obligation as the research and development services were performed. The Company determined that the period of performance of the research and development services was two years through projected initial phase 1 clinical study substantial completion for these patients, or through September 2019. The Company recognized revenue related to research and development services performed using an input method by calculating costs incurred at each period end relative to total costs expected to be incurred. Although the Company fully satisfied this performance obligation during the third quarter of 2019, any changes to the total transaction price following the completion of this performance obligation in September 2019 will be allocated to the performance obligations under the arrangement based on a relative SSP basis and therefore the allocation of any changes to the total transaction price may impact the revenue recognized for this performance obligation in the period of change. The following table summarizes the revenue recognized related to the bb21217 research and development services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (1) bb21217 research and development services revenue $ 2,163 $ 2,884 $ 721 $ 2,163 $ 2,884 $ 721 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. bb21217 license and manufacturing services The Company will satisfy its performance obligation related to the manufacture of vectors and associated payload for incorporation into bb21217 through development as the bb21217 manufacturing services are performed. As of December 31, 2019, the manufacturing services for bb21217 had not yet commenced. Therefore, no amounts have been recognized for the combined performance obligation in the consolidated statements of operations for the years ended December 31, 2019, 2018, and 2017. The aggregate amount of the transaction price allocated to the combined performance obligation, which consists of the bb21217 license and manufacturing services, is $36.2 million. The Company does not expect that recognition will begin in the next twelve months and has therefore classified deferred revenue associated with the combined performance obligation as deferred revenue, net of current portion on its consolidated balance sheet. The Company had $9.8 million of remaining deferred revenue as of December 31, 2019 and 2018 associated with the combined performance obligation consisting of the bb21217 license and manufacturing services. Contract assets and liabilities – ide-cel and bb21217 The Company receives payments from its collaborative partners based on billing schedules established in each contract. Up-front payments and fees are recorded as deferred revenue upon receipt or when due until such time as the Company satisfies its performance obligations under these arrangements. A contract asset is a conditional right to consideration in exchange for goods or services that the Company has transferred to a customer. Amounts are recorded as accounts receivable when the Company’s right to consideration is unconditional. The following table presents changes in the balances of the Company’s BMS receivables and contract liabilities during the twelve months ended December 31, 2019 (in thousands): Balance at December 31, 2018 Additions Deductions Balance at December 31, 2019 Receivables $ 6,528 $ 11,250 $ (17,378) $ 400 Contract liabilities: Deferred revenue $ 34,939 $ 10,000 $ (26,674) $ 18,265 The change in the receivables balance for the year ended December 31, 2019 is primarily driven by amounts collected from BMS in the period, offset by amounts owed to the Company for a development milestone that was achieved in the second quarter of 2019. The decrease in deferred revenue during the year ended December 31, 2019 is primarily driven by amounts recognized for the combined performance obligation consisting of the ide-cel license and manufacturing services. During the year ended December 31, 2019, $17.6 million of the deferred revenue balance at the beginning of the period was released from deferred revenue. Regeneron Regeneron Collaboration Agreement On August 3, 2018, the Company entered into a Collaboration Agreement (the “Regeneron Collaboration Agreement”) with Regeneron pursuant to which the parties will apply their respective technology platforms to the discovery, development, and commercialization of novel immune cell therapies for cancer. On August 24, 2018, following the completion of required regulatory reviews, the Regeneron Collaboration Agreement became effective. Under the terms of the agreement, the parties will leverage Regeneron’s proprietary platform technologies for the discovery and characterization of fully human antibodies, as well as T cell receptors directed against tumor-specific proteins and peptides and the Company will contribute its field-leading expertise in gene therapy. In accordance with the Regeneron Collaboration Agreement, the parties jointly selected six initial targets and intend to equally share the costs of research up to the point of submitting an IND application for a potential gene therapy product directed to a particular target. Additional targets may be selected to add to or replace any of the initial targets during the five Regeneron will accrue a certain number of option rights exercisable against targets as the parties reach certain mile | The following table presents changes in the balances of the Company’s BMS receivables and contract liabilities during the twelve months ended December 31, 2019 (in thousands): Balance at December 31, 2018 Additions Deductions Balance at December 31, 2019 Receivables $ 6,528 $ 11,250 $ (17,378) $ 400 Contract liabilities: Deferred revenue $ 34,939 $ 10,000 $ (26,674) $ 18,265 |
License and royalty revenue
License and royalty revenue | 12 Months Ended |
Dec. 31, 2019 | |
License And Royalty Revenue [Abstract] | |
License and royalty revenue | License and royalty revenue Novartis Pharma AG On April 26, 2017, the Company entered into a worldwide license agreement with Novartis. Under the terms of the agreement, Novartis non-exclusively licensed certain patent rights related to lentiviral vector technology to develop and commercialize CAR T cell therapies for oncology, including Kymriah (formerly known as CTL19), Novartis’s anti-CD19 CAR T therapy. At contract inception, financial terms of the agreement included a $7.5 million payment upon execution, $7.5 million of potential future milestone payments associated with regulatory approvals, and $1.1 million of payments for each subsequently licensed product, as well as low single digit royalty payments on net sales of covered products. In August 2017, Novartis received FDA approval for Kymriah and paid the Company $2.5 million as a result of the achievement of a related milestone. Under Topic 606, the Company identified only one performance obligation, consisting of the license, which was satisfied at contract inception. Accordingly, the nonrefundable license fee of $7.5 million was recognized as revenue upon contract execution in the second quarter of 2017 and a $2.5 million regulatory milestone was recognized as revenue upon milestone achievement, also in the second quarter of 2017, given there were no other unsatisfied performance obligations in the arrangement. Regulatory approvals are not within the Company’s control or the licensee’s control and are generally not considered probable of being achieved until those approvals are received. As such, these milestones are constrained until such time as regulatory approvals are received. Because the single performance obligation was previously satisfied, all regulatory milestones will be recognized as revenue in full in the period in which the associated milestone is achieved. The Company began recognizing royalty revenue from sales of Kymriah in the fourth quarter of 2017. As the license was deemed to be the predominant item to which the royalties relate, the Company recognizes royalties from the sales of Kymriah when the related sales occur. For the year ended December 31, 2019, the Company recognized royalty revenue of $8.2 million and the cost of license and royalty revenue was $3.0 million. During the year ended December 31, 2018, the Company recognized royalty revenue of $2.2 million and the cost of license and royalty revenue was $0.9 million. Orchard Therapeutics Limited (assigned by GlaxoSmithKline Intellectual Property Development Limited) On April 28, 2017, the Company entered into a worldwide license agreement with GlaxoSmithKline Intellectual Property Development Limited (“GSK”). Under the terms of the agreement, GSK non-exclusively licensed certain patent rights related to lentiviral vector technology to develop and commercialize gene therapies for Wiscott-Aldrich syndrome and metachromatic leukodystrophy, two rare genetic diseases. Financial terms of the agreement include a nonrefundable upfront payment of $3.0 million as well as $1.3 million of potential milestone payments for each marketing authorization for each indication in any country as well as low single digit royalties on net sales of covered products. This license agreement was assigned by GSK to Orchard Therapeutics Limited, effective as of April 11, 2018. Under Topic 606, the Company identified only one performance obligation, consisting of the license, which was satisfied at contract inception. Accordingly, the entire nonrefundable license fee of $3.0 million was recognized as revenue upon contract execution in the second quarter of 2017 given there were no other unsatisfied performance obligations in the arrangement. Regulatory approvals are not within the Company’s control or the licensee’s control and are generally not considered probable of being achieved until those approvals are received. As such, these milestones are constrained until such time as regulatory approvals are received. There was no revenue recognized under this arrangement in the years ended December 31, 2019 and 2018. Because the single performance obligation was previously satisfied, all regulatory milestones will be recognized as revenue in full in the period in which the associated milestone is achieved. Given there was no revenue recognized under this arrangement in the years ended December 31, 2019 and 2018, there was no associated cost of license and royalty revenue. The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed pertaining to prior periods are presented under previous accounting guidance. |
Intangible assets
Intangible assets | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible assets | Intangible assets Intangible assets, net of accumulated amortization, are summarized as follows (in thousands): As of December 31, As of December 31, 2019 2018 Cost Accumulated amortization Net Cost Accumulated amortization Net Developed technology $ 30,100 $ (20,694) $ 9,406 $ 30,100 $ (16,931) $ 13,169 In-licensed rights 5,224 (304) 4,920 — — — Total $ 35,324 $ (20,998) $ 14,326 $ 30,100 $ (16,931) $ 13,169 Amortization expense for intangible assets was $4.1 million, $3.8 million, and $3.8 million for each of the years ended December 31, 2019, 2018 and 2017, respectively. Developed technology The Company's developed technology was obtained through its acquisition of Pregenen, a privately-held biotechnology company. As a result, the Company obtained gene editing and cell signaling technology with a broad range of potential therapeutic applications. The Company considered the intangible asset acquired to be developed technology, as at the date of the acquisition it could be used the way it was intended to be used in certain ongoing research and development activities. The gene editing platform intangible asset is being amortized on a straight-line basis over its expected useful life of approximately eight years from the date of the acquisition. Please refer to Note 4, Fair value measurements , and Note 9, Commitments and contingencies , for further information. In-licensed rights In-licensed rights consist of capitalized milestone payments made to third parties upon receiving regulatory approval of ZYNTEGLO in the EU. The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents of approximately ten years, as the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights. The following table summarizes the estimated future amortization for intangible assets for the next five years (in thousands): As of December 31, 2019 2020 $ 4,285 2021 4,285 2022 2,404 2023 522 2024 522 Total $ 12,018 |
Stock-based compensation
Stock-based compensation | 12 Months Ended |
Dec. 31, 2019 | |
Share-based Payment Arrangement [Abstract] | |
Stock-based compensation | Stock-based compensation On June 3, 2013, the Company’s board of directors adopted its 2013 Stock Option and Incentive Plan (“2013 Plan”), which was subsequently approved by its stockholders and became effective upon the closing of the Company’s IPO on June 24, 2013. The 2013 Plan replaces the 2010 Stock Option and Grant Plan (“2010 Plan”). The 2013 Plan allows for the granting of incentive stock options, non-qualified stock options, restricted stock units and restricted stock awards to the Company’s employees, members of the board of directors, and consultants of the Company. The Company initially reserved 1.0 million shares of its common stock for the issuance of awards under the 2013 Plan. The 2013 Plan provides that the number of shares reserved and available for issuance under the 2013 Plan will automatically increase each January 1, beginning on January 1, 2014, by four percent of the outstanding number of shares of common stock on the immediately preceding December 31 or such lesser number of shares as determined by the Company’s compensation committee. In January 2019 and January 2020, the number of common stock available for issuance under the 2013 Plan was increased by approximately 2.2 million and 2.2 million shares, respectively, as a result of this automatic increase provision. Any options or awards outstanding under the Company’s previous stock option plans, including both the 2010 Plan and the Second Amended and Restated 2002 Employee, Director and Consultant Stock Plan (“2002 Plan”), at the time of adoption of the 2013 Plan remain outstanding and effective. The shares of common stock underlying any awards that are forfeited, canceled, repurchased, expired or are otherwise terminated (other than by exercise) under the 2002 Plan and 2010 Plan are added to the shares of common stock available for issuance under the 2013 Plan. As of December 31, 2019, the total number of common stock that may be issued under all plans is 2.2 million. The Company does not currently hold any treasury shares. Upon stock option exercise, the Company issues new shares and delivers them to the participant. Stock-based compensation expense The Company recognized stock-based compensation expense totaling $160.6 million, $110.8 million, and $53.3 million during the years ended December 31, 2019, 2018 and 2017, respectively. Stock-based compensation expense recognized by award type is as follows (in thousands): Year Ended December 31, 2019 2018 2017 Stock options $ 95,668 $ 83,449 $ 42,262 Restricted stock units 63,580 26,628 10,495 Employee stock purchase plan 1,381 759 525 $ 160,629 $ 110,836 $ 53,282 Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 80,139 $ 54,422 $ 26,633 Selling, general and administrative 80,490 56,414 26,649 $ 160,629 $ 110,836 $ 53,282 In February 2018, the Company issued restricted stock units with service and performance conditions to employees, approximately 0.2 million of which are outstanding as of December 31, 2019. Vesting of these awards is contingent on the occurrence of a certain regulatory milestone event which was achieved in June 2019 and fulfillment of any remaining service condition. The Company began recognizing expense for these awards in the second quarter of 2019 when achievement of the regulatory milestone was deemed probable. The Company recognized $20.1 million of expense related to these awards in the second quarter of 2019 and will continue to recognize stock-based compensation expense related to these awards through June 2021 when the final tranche of the awards vest. As of December 31, 2019, the Company had $208.1 million, $105.8 million and less than $0.1 million of unrecognized compensation expense related to unvested stock options, restricted stock units, and the employee stock purchase plan, respectively, that is expected to be recognized over a weighted-average period of 2.5 years, 2.6 years, and 0.1 years, respectively. Stock options The fair value of each option issued to employees was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions: Year Ended December 31, 2019 2018 2017 Expected volatility 70.7 % 75.5 % 78.1 % Expected term (in years) 6.0 6.0 6.0 Risk-free interest rate 2.3 % 2.7 % 2.1 % Expected dividend yield 0.0 % 0.0 % 0.0 % The following table summarizes the stock option activity under the Company’s equity awards plans: Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2018 4,643 $ 108.56 Granted 1,594 $ 129.76 Exercised (354) $ 50.36 Canceled or forfeited (400) $ 138.61 Outstanding at December 31, 2019 5,483 $ 116.30 7.3 $ 68,511 Exercisable at December 31, 2019 2,853 $ 94.77 6.0 $ 64,899 Vested and expected to vest at December 31, 2019 5,483 $ 116.30 7.3 $ 68,511 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at December 31, 2019. The weighted-average fair values of options granted during 2019, 2018 and 2017 was $83.44, $125.12, and $62.03, respectively. The intrinsic value of options exercised during the years ended December 31, 2019, 2018, and 2017, was $29.0 million, $73.1 million and $91.0 million, respectively. Restricted stock units The following table summarizes the restricted stock unit activity under the Company’s equity award plans: Shares Weighted-average Unvested balance at December 31, 2018 931 $ 155.99 Granted 570 128.87 Vested (251) 138.93 Forfeited (123) 155.68 Unvested balance at December 31, 2019 1,127 $ 146.10 The intrinsic value of restricted stock units vested during the years ended December 31, 2019, 2018, and 2017 was $28.4 million, $25.5 million and $8.1 million, respectively. Employee Stock Purchase Plan On June 3, 2013, the Company’s board of directors adopted its 2013 Employee Stock Purchase Plan (“2013 ESPP”), which was subsequently approved by its stockholders and became effective upon the closing of the Company’s IPO on June 24, 2013. The 2013 ESPP authorizes the initial issuance of up to a total of 0.2 million shares of the Company’s common stock to participating employees. During each of the years ended December 31, 2019 and 2018, less than 0.1 million shares of common stock were issued under the 2013 ESPP, respectively. |
401(k) savings plan
401(k) savings plan | 12 Months Ended |
Dec. 31, 2019 | |
Retirement Benefits [Abstract] | |
401(k) savings plan | 401(k) Savings planIn 1997, the Company established a defined-contribution savings plan under Section 401(k) of the Internal Revenue Code (“the 401(k) Plan”). The 401(k) Plan covers all employees who meet defined minimum age and service requirements, and allows participants to defer a portion of their annual compensation on a pretax basis. In March 2020, the Company expects to make matching contributions of approximately $4.6 million related to employee contributions made during 2019. In February 2019, the Company made $2.4 million of matching contributions related to employee contributions made during 2018. The match contribution is included in accrued expenses and other current liabilities as of December 31, 2019 and 2018. Expense related to the 401(k) Plan totaled $4.6 million, $2.4 million, $1.5 million for the years ended December 31, 2019, 2018, and 2017, respectively. |
Income taxes
Income taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income taxes | Income taxes The components of loss before income taxes were as follows (in thousands): Year ended December 31, 2019 2018 2017 U.S. $ (649,172) $ (440,473) $ (266,236) Foreign (140,981) (114,965) (69,197) Total $ (790,153) $ (555,438) $ (335,433) The provision for (benefit from) income taxes were as follows (in thousands): Year ended December 31, 2019 2018 2017 Current: Federal $ — $ — $ — State 7 324 115 Foreign 612 222 95 Deferred: Federal (966) (307) — State (198) (52) — Foreign — — — Total income tax (benefit) expense $ (545) $ 187 $ 210 A reconciliation of income tax provision (benefit) computed at the statutory federal income tax rate to the Company’s effective income tax rate (provision) benefit as reflected in the financial statements is as follows: Year ended December 31, 2019 2018 2017 Federal income tax expense at statutory rate 21.0 % 21.0 % 34.0 % State income tax, net of federal benefit 3.7 % 5.1 % 4.3 % Permanent differences (1.1) % 0.9 % 2.7 % Research and development credit 5.4 % 6.5 % 12.8 % Foreign differential (3.7) % (4.4) % (6.9) % Federal tax rate change — % 0.1 % (31.6) % Other 0.4 % (0.1) % (0.8) % Change in valuation allowance (25.6) % (29.1) % (14.6) % Effective income tax rate benefit (expense) 0.1 % — % (0.1) % For the years ended December 31, 2019, 2018 and 2017, the Company recognized an income tax (benefit) expense of $(0.5) million or 0.1%, $0.2 million or 0.0%, and $0.2 million or (0.1)%, respectively. The Company did not recognize any significant tax expense for the years ended December 31, 2019, 2018, or 2017 as the Company was subject to a full valuation allowance. Deferred taxes are recognized for temporary differences between the basis of assets and liabilities for financial statement and income tax purposes. The significant components of the Company’s deferred tax assets and liabilities are composed of the following (in thousands): Year ended December 31, 2019 2018 Deferred tax assets: U.S. net operating loss carryforwards (federal and state) $ 439,839 $ 298,701 Tax credit carryforwards (federal and state) 213,810 167,517 Capitalized license fees and research and development expenses 16,295 18,083 60 Binney Street lease — 42,059 Deferred revenue 15,119 21,442 Stock-based compensation 46,111 31,858 Lease liabilities 52,631 — Accruals and other 15,432 8,886 Total deferred tax assets 799,237 588,546 Intangible assets (2,518) (3,579) Right-of-use assets (49,480) — Fixed assets (2,670) (41,472) Less valuation allowance (744,569) (543,495) Net deferred taxes $ — $ — A valuation allowance is recorded against deferred tax assets if it is more likely than not that some or all of the deferred tax assets will not be realized. Due to the uncertainty surrounding the realization of the favorable tax attributes in future tax returns, the Company has recorded a full valuation allowance against the Company’s otherwise recognizable net deferred tax assets. The valuation allowance increased on a net basis by approximately $201.1 million during the year ended December 31, 2019 due primarily to net operating losses, tax credit carryforwards, and stock-based compensation. Effective January 1, 2019, the Company adopted ASU 2016-02, which resulted in the de-recognition of the 60 Binney Street lease and related fixed assets and the recognition of lease liabilities and right-of-use assets. The Company adjusted its deferred tax balances as a result of the adoption. Please refer to Note 2, Summary of significant accounting policies and basis of presentation , and Note 8, Leases , for further information. As of December 31, 2019, 2018 and 2017, the Company had U.S. federal net operating loss carryforwards of approximately $1.62 billion, $1.10 billion, and $716.1 million, respectively, which may be available to offset future income tax liabilities. Of the amount as of December 31, 2019, $913.8 million will carryforward indefinitely while $711.0 million will expire at various dates through 2037. As of December 31, 2019, 2018 and 2017, the Company also had U.S. state net operating loss carryforwards of approximately $1.56 billion, $1.08 billion, and $692.9 million, respectively, which may be available to offset future income tax liabilities and expire at various dates through 2039. At December 31, 2016, $195.4 million and $195.4 million of federal and state net operating losses, respectively, related to excess equity-based compensation tax deductions, the benefits for which will be recorded to additional paid-in capital when recognized through a reduction of cash taxes paid. As a result of adopting FASB ASU 2016-09 in the first quarter of 2017, the Company recorded a cumulative-effect adjustment to retained earnings of $76.7 million to record a net deferred tax asset relative to these tax attribute carryforwards. The deferred tax asset was offset by a corresponding adjustment to the valuation allowance. As of December 31, 2019, 2018 and 2017, the Company had federal research and development and orphan drug tax credit carryforwards of approximately $203.1 million, $156.2 million, and $124.1 million, respectively, available to reduce future tax liabilities which expire at various dates through 2039. As of December 31, 2019, 2018 and 2017, the Company had state credit carryforwards of approximately $13.6 million, $14.3 million, and $9.1 million, respectively, available to reduce future tax liabilities which expire at various dates through 2034. During the fourth quarter of 2018, the Company completed an analysis of prior year estimates of U.S. research and development and orphan drug tax credits for the years 2013 through 2017. The analysis resulted in an immaterial adjustment to the Company's income tax benefit, which was offset by an adjustment to the valuation allowance. An analysis of the U.S. research and development and orphan drug credits has not been completed for 2018 and 2019. Under the provisions of the Internal Revenue Code, the net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three The Company or one of its subsidiaries files income tax returns in the United States, and various state and foreign jurisdictions. The federal, state and foreign income tax returns are generally subject to tax examinations for the tax years ended December 31, 2016 through December 31, 2018. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, or state or foreign tax authorities to the extent utilized in a future period. A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): Unrecognized tax benefits Balance as of December 31, 2017 $ — Increases for tax positions related to current period 3,370 Increases for tax positions related to prior periods 8,725 Balance as of December 31, 2018 12,095 Increases for tax positions related to current period 3,554 Increases for tax positions related to prior periods 296 Balance as of December 31, 2019 15,945 The unrecognized tax benefits at December 31, 2019, if recognized, would not affect the Company’s effective tax rate due to its full valuation allowance position. The Company does not anticipate that the amount of existing unrecognized tax benefits will significantly increase or decrease within the next 12 months. The Company has elected to include interest and penalties related to uncertain tax positions as a component of its provision for income taxes. For the years ended December 31, 2019, 2018 and 2017, the Company’s accrued interest and penalties related to uncertain tax positions were not material. |
Net loss per share
Net loss per share | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Net loss per share | Net loss per shareThe following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect (in thousands): Year ended December 31, 2019 2019 2018 2017 Outstanding stock options 5,483 4,643 3,755 Restricted stock units 1,127 931 477 ESPP shares 19 10 9 6,629 5,584 4,241 |
Selected quarterly financial da
Selected quarterly financial data (unaudited) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
Selected quarterly financial data (Unaudited) | Selected quarterly financial data (unaudited) The following table contains quarterly financial information for 2019 and 2018. The Company believes that the following information reflects all normal recurring adjustments necessary for a fair presentation of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2019 First Second Third Fourth Total (in thousands, except per share data) Total revenues $ 12,471 $ 13,296 $ 8,910 $ 9,997 $ 44,674 Total operating expenses 183,645 215,998 219,326 240,531 859,500 Loss from operations (171,174) (202,702) (210,416) (230,534) (814,826) Net loss (164,446) (195,782) (206,033) (223,347) (789,608) Net loss per share applicable to common $ (2.99) $ (3.55) $ (3.73) $ (4.04) $ (14.31) 2018 First Second Third Fourth Total (in thousands, except per share data) Total revenues $ 15,957 $ 7,851 $ 11,528 $ 19,243 $ 54,579 Total operating expenses 132,586 156,465 161,347 176,204 626,602 Loss from operations (116,629) (148,614) (149,819) (156,961) (572,023) Net loss (115,126) (145,996) (145,480) (149,023) (555,625) Net loss per share applicable to common $ (2.31) $ (2.91) $ (2.73) $ (2.72) $ (10.68) |
Summary of significant accoun_2
Summary of significant accounting policies and basis of presentation (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation The accompanying consolidated financial statements have been prepared by the Company in accordance with accounting principles generally accepted in the United States (“GAAP”) as found in the ASC and Accounting Standards Updates (“ASUs”) of the Financial Accounting Standards Board (“FASB”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States GAAP as found in the ASC and ASUs of the FASB. Certain items in the prior year’s consolidated financial statements have been reclassified to conform to the current presentation. No subtotals in the prior year consolidated financial statements were impacted by these reclassifications. Amounts reported are computed based on thousands, except percentages, per share amounts or as otherwise noted. As a result, certain totals may not sum due to rounding. |
Principles of consolidation | Principles of consolidation The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company continually assesses whether it is the primary beneficiary of a variable interest entity as changes to existing relationships or future transactions may result in consolidation or deconsolidation of one or more collaborators or partners. In determining whether it is the primary beneficiary of an entity in which the Company has a variable interest, management applies a qualitative approach that determines whether the Company has both (1) the power to direct the economically significant activities of the entity and (2) the obligation to absorb losses of, or the right to receive benefits from, the entity that could potentially be significant to that entity. |
Use of estimates | Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could materially differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the financial statements. Estimates and judgments are used in the following areas, among others: future undiscounted cash flows and subsequent fair value estimates used to assess potential and measure any impairment of long-lived assets, including goodwill and intangible assets, and the measurement of right-of-use assets and lease liabilities, contingent consideration, stock-based compensation expense, accrued expenses, income taxes, and the assessment of the Company's ability to fund its operations for at least the next twelve months from the date of issuance of these financial statements. In addition, estimates and judgments are used in the Company’s accounting for its revenue-generating arrangements, in particular as it relates to determining the standalone selling price of performance obligations, evaluating whether an option to acquire additional goods and services represents a material right, estimating the total transaction price, including estimating variable consideration and the probability of achieving future potential development and regulatory milestones, and the period of performance over which revenue may be recognized. |
Foreign currency transaction | Foreign currency translation The financial statements of the Company’s subsidiaries with functional currencies other than the U.S. dollar are translated into U.S. dollars using period-end exchange rates for assets and liabilities, historical exchange rates for stockholders’ equity and weighted average exchange rates for operating results. Translation gains and losses are included in accumulated other comprehensive income (loss) in stockholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the results of operations. |
Segment information | Segment information The Company operates in a single segment, focusing on researching, developing and commercializing potentially transformative gene therapies for severe genetic diseases and cancer. Consistent with its operational structure, its chief operating decision maker manages and allocates resources at a global, consolidated level. Therefore, results of the Company's operations are reported on a consolidated basis for purposes of segment reporting. All material long-lived assets of the Company reside in the United States. |
Cash and cash equivalents | Cash and cash equivalents The Company considers all highly liquid investments purchased with original final maturities of 90 days or less from the date of purchase to be cash equivalents. Cash and cash equivalents comprise marketable securities with maturities of less than 90 days when purchased. Cash equivalents are reported at fair value. |
Marketable securities | Marketable securities The Company classifies marketable securities with a remaining maturity when purchased of greater than three months as available-for-sale. Marketable securities with a remaining maturity date greater than one year are classified as non-current. The Company’s marketable securities are maintained by investment managers and consist of U.S. Treasury securities, U.S. government agency securities, equity securities, certificates of deposit, corporate bonds, and commercial paper. Debt securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium arising at purchase is amortized to the earliest call date and any discount arising at purchase is accreted to maturity. Amortization and accretion of premiums and discounts are recorded in interest income and/or expense. Equity securities with readily determinable fair values are also carried at fair value with unrealized gains and losses included in other (expense) income, net. Realized gains and losses on both debt and equity securities are determined using the specific identification method and are included in other income (expense), net. The Company classifies equity securities with readily determinable fair values, which would be available for use in its current operations, as current assets even though the Company may not dispose of such marketable securities within the next 12 months. Equity securities are included in the balance of marketable securities on the Company's consolidated balance sheet. If any adjustment to fair value reflects a decline in value of the investment, the Company considers all available evidence to evaluate the extent to which the decline is “other-than-temporary” and, if so, marks the investment to market through a charge to the Company’s statement of operations and comprehensive loss. |
Concentrations of credit risk and off-balance sheet risk | Concentrations of credit risk and off-balance sheet risk Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities primarily consist of U.S. Treasury securities, U.S. government agency securities, certificates of deposit, corporate bonds, and commercial paper, and potentially subject the Company to concentrations of credit risk. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure. |
Fair value of financial instruments | Fair value of financial instruments The Company has certain financial assets and liabilities recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements Level 1—Fair values are determined utilizing quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2—Fair values are determined utilizing quoted prices for identical or similar assets or liabilities in active markets or other market observable inputs such as interest rates, yield curves and foreign currency spot rates. Level 3—Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. Items measured at fair value on a recurring basis include marketable securities (see Note 3, Marketable securities, and Note 4, Fair value measurements ) and contingent consideration (see Note 4, Fair value measurements ). The carrying amounts of accounts payable and accrued expenses approximate their fair values due to their short-term nature. |
Business combinations | Business combinations Business combinations are accounted for using the acquisition method of accounting. Using this method, the tangible and intangible assets acquired and the liabilities assumed are recorded as of the acquisition date at their respective fair values. The Company evaluates a business as an integrated set of activities and assets that is capable of being managed for the purpose of providing a return in the form of dividends, lower costs or other economic benefits and consists of inputs and processes that provide or have the ability to provide outputs. In an acquisition of a business, the excess of the fair value of the consideration transferred over the fair value of the net assets acquired is recorded as goodwill. In an acquisition of net assets that does not constitute a business, no goodwill is recognized. |
Goodwill | GoodwillGoodwill represents the excess of the purchase price over the fair value of the net assets acquired when accounted for using the acquisition method of accounting for business combinations. Goodwill is not amortized but is evaluated for impairment within the Company’s single reporting unit on an annual basis, during the fourth quarter, or more frequently if an event occurs or circumstances change that would more-likely-than-not reduce the fair value of the Company’s reporting unit below its carrying amount. During the fourth quarter of 2019, the Company early adopted ASU 2017-04, which removes the second step of the goodwill impairment test. Under this ASU, the Company will now perform a one-step quantitative test and record the amount of goodwill impairment, if any, as the excess of a reporting unit's carrying amount over its fair value, not to exceed the total amount of goodwill allocated to the reporting unit. |
Intangible assets | Intangible assets, net Intangible assets, net consist of acquired core technology and in-licensed rights with finite lives, net of accumulated amortization. The Company amortizes its intangible assets using the straight-line method over their estimated economic lives and periodically reviews for impairment. |
Contingent consideration | Contingent consideration Each reporting period, the Company revalues the contingent consideration obligations associated with business combinations to their fair value and records within operating expenses increases in their fair value as contingent consideration expense and decreases in the fair value as contingent consideration income. Changes in contingent consideration result from changes in the assumptions regarding probabilities of successful achievement of related milestones, the estimated timing in which the milestones may be achieved, and the discount rate used to estimate the fair value of the liability. Contingent consideration may change significantly as development of the Company’s programs in certain indications progress and additional data are obtained, impacting the Company’s assumptions. The assumptions used in estimating fair value require significant judgment. The use of different assumptions and judgments could result in a materially different estimate of fair value. See Note 4, Fair value measurements, for additional information. |
Property, plant and equipment | Property, plant and equipment Property, plant and equipment is stated at cost. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Depreciation and amortization is calculated using the straight-line method over the estimated useful lives of the assets, which are as follows: Asset Estimated useful life Building 40 years Computer equipment and software 3 years Furniture and fixtures 2-5 years Laboratory equipment 2-5 years Leasehold improvements Shorter of the useful life or remaining lease term |
Impairment of long-lived assets | Impairment of long-lived assets The Company reviews long-lived assets when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. Recoverability is measured by comparison of the book values of the assets to future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets. |
Leases | Leases Effective January 1, 2019, the Company adopted ASC 842 using the required modified retrospective approach and utilizing the effective date as its date of initial application. As a result, prior periods are presented in accordance with the previous guidance in ASC 840, Leases (“ASC 840”). At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than one year are recognized on the balance sheet as right-of-use assets and short-term and long-term lease liabilities, as applicable. The Company does not have material financing leases. Operating lease liabilities and their corresponding right-of-use assets are initially recorded based on the present value of lease payments over the expected remaining lease term. Certain adjustments to the right-of-use asset may be required for items such as incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate to discount lease payments, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. To estimate its incremental borrowing rate, a credit rating applicable to the Company is estimated using a synthetic credit rating analysis since the Company does not currently have a rating agency-based credit rating. Prospectively, the Company will adjust the right-of-use assets for straight-line rent expense or any incentives received and remeasure the lease liability at the net present value using the same incremental borrowing rate that was in effect as of the lease commencement or transition date. The Company has elected not to recognize leases with an original term of one year or less on the balance sheet. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. Assumptions made by the Company at the commencement date are re-evaluated upon occurrence of certain events, including a lease modification. A lease modification results in a separate contract when the modification grants the lessee an additional right of use not included in the original lease and when lease payments increase commensurate with the standalone price for the additional right of use. When a lease modification results in a separate contract, it is accounted for in the same manner as a new lease. ASC 842 transition practical expedients and application of transition provisions to leases at the transition date The Company elected the following practical expedients, which must be elected as a package and applied consistently to all of its leases at the transition date (including those for which the entity is a lessee or a lessor): i) the Company did not reassess whether any expired or existing contracts are or contain leases; ii) the Company did not reassess the lease classification for any expired or existing leases (that is, all existing leases that were classified as operating leases in accordance with ASC 840 are classified as operating leases, and all existing leases that were classified as capital leases in accordance with ASC 840 are classified as finance leases); and iii) the Company did not reassess initial direct costs for any existing leases. For leases that existed prior to the date of initial application of ASC 842 (which were previously classified as operating leases), a lessee may elect to use either the total lease term measured at lease inception under ASC 840 or the remaining lease term as of the date of initial application of ASC 842 in determining the period for which to measure its incremental borrowing rate. In transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rates. Application of ASC 842 policy elections to leases post adoption The Company has made certain policy elections to apply to its leases executed post adoption, or subsequent to January 1, 2019, as further described below. In accordance with ASC 842, components of a lease should be split into three categories: lease components, non-lease components, and non-components. The fixed and in-substance fixed contract consideration (including any consideration related to non-components) must be allocated based on the respective relative fair values to the lease components and non-lease components. Entities may elect not to separate lease and non-lease components. Rather, entities would account for each lease component and related non-lease component together as a single lease component. The Company has elected to account for lease and non-lease components together as a single lease component for all underlying assets and allocate all of the contract consideration to the lease component only. ASC 842 allows for the use of judgment in determining whether the assumed lease term is for a major part of the remaining economic life of the underlying asset and whether the present value of lease payments represents substantially all of the fair value of the underlying asset. The Company applies the bright line thresholds referenced in ASC 842-10-55-2 to assist in evaluating leases for appropriate classification. The aforementioned bright lines are applied consistently to the Company’s entire portfolio of leases. |
Revenue recognition and research and development expenses | Revenue recognition Effective January 1, 2018, the Company adopted ASC Topic 606, Revenue from Contracts with Customers (“Topic 606”), using the modified retrospective transition method. Under this method, the Company has recognized the cumulative effect of the adoption as an adjustment to the opening balance of accumulated deficit in the current period consolidated balance sheet. The Company has not revised its consolidated financial statements for prior periods. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as collaboration arrangements and leases. Under Topic 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration that the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price, including variable consideration, if any; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect the consideration to which it is entitled in exchange for the goods or services it transfers to the customer. Once a contract is determined to be within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations. Arrangements that include rights to additional goods or services that are exercisable at a customer’s discretion are generally considered options. The Company assesses if these options provide a material right to the customer and if so, they are considered performance obligations. The identification of material rights requires judgments related to the determination of the value of the underlying license relative to the option exercise price, including assumptions about technical feasibility and the probability of developing a candidate that would be subject to the option rights. The exercise of a material right is accounted for as a contract modification for accounting purposes. The Company assesses whether each promised good or service is distinct for the purpose of identifying the performance obligations in the contract. This assessment involves subjective determinations and requires management to make judgments about the individual promised goods or services and whether such are separable from the other aspects of the contractual relationship. Promised goods and services are considered distinct provided that: (i) the customer can benefit from the good or service either on its own or together with other resources that are readily available to the customer (that is, the good or service is capable of being distinct) and (ii) the entity’s promise to transfer the good or service to the customer is separately identifiable from other promises in the contract (that is, the promise to transfer the good or service is distinct within the context of the contract). In assessing whether a promised good or service is distinct, the Company considers factors such as the research, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. The Company also considers the intended benefit of the contract in assessing whether a promised good or service is separately identifiable from other promises in the contract. If a promised good or service is not distinct, an entity is required to combine that good or service with other promised goods or services until it identifies a bundle of goods or services that is distinct. The transaction price is then determined and allocated to the identified performance obligations in proportion to their standalone selling prices (“SSP”) on a relative SSP basis. SSP is determined at contract inception and is not updated to reflect changes between contract inception and when the performance obligations are satisfied. Determining the SSP for performance obligations requires significant judgment. In developing the SSP for a performance obligation, the Company considers applicable market conditions and relevant entity-specific factors, including factors that were contemplated in negotiating the agreement with the customer and estimated costs. The Company validates the SSP for performance obligations by evaluating whether changes in the key assumptions used to determine the SSP will have a significant effect on the allocation of arrangement consideration between multiple performance obligations. If the consideration promised in a contract includes a variable amount, the Company estimates the amount of consideration to which it will be entitled in exchange for transferring the promised goods or services to a customer. The Company determines the amount of variable consideration by using the expected value method or the most likely amount method. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis in the period of adjustment. If an arrangement includes development and regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the Company’s control or the licensee’s control, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. For arrangements with licenses of intellectual property that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes royalty revenue and sales-based milestones at the later of (i) when the related sales occur, or (ii) when the performance obligation to which the royalty has been allocated has been satisfied. In determining the transaction price, the Company adjusts consideration for the effects of the time value of money if the timing of payments provides the Company with a significant benefit of financing. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. The Company assessed each of its revenue generating arrangements in order to determine whether a significant financing component exists and concluded that a significant financing component does not exist in any of its arrangements. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time, and if over time recognition is based on the use of an output or input method. Collaboration revenue To date, the Company’s collaboration revenue has been generated from its collaboration arrangements with BMS and Regeneron Pharmaceuticals, Inc. (“Regeneron”), as further described in Note 11, Collaborative arrangements . The Company analyzes its collaboration arrangements to assess whether they are within the scope of ASC 808, Collaborative Arrangements (“ASC 808”) to determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities. This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For collaboration arrangements within the scope of ASC 808 that contain multiple elements, the Company first determines which elements of the collaboration are deemed to be within the scope of ASC 808 and those that are more reflective of a vendor-customer relationship and therefore within the scope of Topic 606. For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, generally by analogy to Topic 606. Amounts that are owed to collaboration partners are recognized as an offset to collaboration revenues as such amounts are incurred by the collaboration partner. Where amounts owed to a collaboration partner exceed the Company’s collaboration revenues in each quarterly period, such amounts are classified as research and development expense. For those elements of the arrangement that are accounted for pursuant to Topic 606, the Company applies the five-step model described above. License and royalty revenue The Company enters into out-licensing agreements that are within the scope of Topic 606. The Company does not have any material license arrangements that contain more than one performance obligation. The terms of such out-license agreements include the license of functional intellectual property, given the functionality of the intellectual property is not expected to change substantially as a result of the licensor’s ongoing activities, and typically include payment of one or more of the following: non-refundable up-front license fees; development and regulatory milestone payments and milestone payments based on the level of sales; and royalties on net sales of licensed products. Nonrefundable up-front license fees are recognized as revenue at a point in time when the licensed intellectual property is made available for the customer’s use and benefit, which is generally at the inception of the arrangement. Development and regulatory milestone fees, which are a type of variable consideration, are recognized as revenue to the extent that it is probable that a significant reversal will not occur. The Company recognizes royalty revenue and sales-based milestones at the later of (i) when the related sales occur, or (ii) when the performance obligation to which the royalty has been allocated has been satisfied. |
Research and development expenses | Research and development expenses Research and development costs are charged to expense as costs are incurred in performing research and development activities, including salaries and benefits, facilities costs, overhead costs, clinical study and related clinical manufacturing costs, license and milestone fees, contract services, manufacturing costs for pre-launch inventory that did not qualify for capitalization, and other related costs. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed as research and development expense as incurred. In circumstances where amounts have been paid in excess of costs incurred, the Company records a prepaid expense. The Company accrues costs for clinical trial activities based upon estimates of the services received and related expenses incurred that have yet to be invoiced by the contract research organizations, clinical study sites, laboratories, consultants, or other clinical trial vendors that perform the activities. The Company recognizes the reimbursement associated with collaborative activities to its collaborative partners as research and development expense in the period the services are provided. |
Cost of license and royalty revenue | Cost of license and royalty revenue Cost of license and royalty revenue represents expense associated with amounts owed to third parties as a result of revenue recognized under the Company’s out-license arrangements. |
Stock-based compensation | Stock-based compensation The Company’s share-based compensation programs grant awards that have included stock options, restricted stock units, restricted stock awards, and shares issued under its employee stock purchase plan. Grants are awarded to employees and non-employees, including the Company's board of directors. The Company accounts for its stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock-based payments, including grants of stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive loss based on their fair values. The Company’s stock-based awards are subject to either service or performance-based vesting conditions. Compensation expense related to awards with service-based vesting conditions is recognized on a straight-line basis based on the grant date fair value over the associated service period of the award, which is generally the vesting term. Compensation expense related to awards with performance-based vesting conditions is recognized based on the grant date fair value over the requisite service period using the accelerated attribution method to the extent achievement of the performance condition is probable. The Company estimates the fair value of its option awards using the Black-Scholes option pricing model, which requires the input of subjective assumptions, including (i) the expected stock price volatility, (ii) the calculation of expected term of the award, (iii) the risk-free interest rate, and (iv) expected dividends. Due to the lack of complete company-specific historical and implied volatility data for the full expected term of the stock-based awards, the Company bases its estimate of expected volatility on a representative group of publicly traded companies in addition to its own volatility data. For these analyses, the Company selected companies with comparable characteristics to its own, including enterprise value, risk profiles, position within the industry, and with historical share price information sufficient to meet the expected life of the stock-based awards. The Company computes historical volatility data using the daily closing prices for the selected companies’ shares during the equivalent period of the calculated expected term of the stock-based awards. The Company will continue to apply this process until a sufficient amount of historical information regarding the volatility of its own stock price becomes available. The Company has estimated the expected term of its employee stock options using the “simplified” method, whereby, the expected term equals the arithmetic average of the vesting term and the original contractual term of the option due to its lack of sufficient historical data. The risk-free interest rates for periods within the expected term of the option are based on the U.S. Treasury securities with a maturity date commensurate with the expected term of the associated award. The Company has never paid, and does not expect to pay, dividends in the foreseeable future. The Company accounts for forfeitures as they occur. Stock-based compensation expense recognized in the financial statements is based on awards for which performance or service conditions are expected to be satisfied. Prior to the adoption of ASU No. 2018-07, Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”), the measurement date for non-employee awards was generally the date the services are completed, resulting in financial reporting period adjustments to stock-based compensation during the vesting terms for changes in the fair value of the awards. After adoption of ASU 2018-07, the measurement date for non-employee awards is the date of grant without changes in the fair value of the award. |
Interest income (expense), net | Interest income (expense), netInterest income (expense), net consists primarily of interest income earned on investments, net of amortization of premium and accretion of discount. |
Other income (expense), net | Other (expense) income, net Other (expense) income, net consists primarily of unrealized gains and losses on equity securities, gains and losses on the disposal of fixed assets, realized gains and losses on debt securities, and gains and losses on foreign currency transactions. |
Net loss per share | Net loss per share Basic net loss per share is calculated by dividing net loss attributable to common stockholders by the weighted average number of common shares outstanding during the period. Diluted net income per share is calculated by dividing the net income attributable to common stockholders by the weighted-average number of common equivalent shares outstanding for the period, including any dilutive effect from outstanding stock options, unvested restricted stock, restricted stock units, and employee stock purchase plan stock using the treasury stock method. Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. The Company follows the two-class method when computing net loss per share in periods when issued shares that meet the definition of participating securities are outstanding. The two-class method determines net loss per share for each class of common and participating securities according to dividends declared or accumulated and participation rights in undistributed earnings. The two-class method requires income available to common stockholders for the period to be allocated between common and participating securities based upon their respective rights to received dividends as if all income for the period had |
Income taxes | Income taxes Income taxes are recorded in accordance with FASB ASC Topic 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. The Company accrues for potential interest and penalties related to unrecognized tax benefits in income tax expense. |
Comprehensive loss | Comprehensive loss Comprehensive loss is composed of net loss and other comprehensive income (loss). Other comprehensive income (loss) consists of unrealized gains and losses on debt securities, foreign currency translation adjustments and other items. |
Recent accounting pronouncements | Recent accounting pronouncements Recently adopted ASU No. 2016-02, Leases (Topic 842), ASU No. 2018-10 Codification Improvements to Topic 842, Leases, ASU No. 2018-11, Leases (Topic 842): Targeted Improvements, and ASU No. 2019-01 Leases (Topic 842): Codification Improvements In February 2016, the FASB issued ASU 2016-02, as amended, which superseded the lease accounting requirements in ASC 840 and created ASC 842. ASC 842 requires a lessee to recognize assets and liabilities on the balance sheet for most leases and changes many key definitions, including the definition of a lease. The new standard includes a short-term lease exception for leases with a term of one year or less, as part of which a lessee can make an accounting policy election not to recognize lease assets and lease liabilities for those leases. Lessees will continue to differentiate between finance leases (previously referred to as capital leases) and operating leases using classification criteria that are substantially similar to the previous guidance. Effective January 1, 2019, the Company adopted ASU 2016-02, using the required modified retrospective approach and utilizing the effective date as its date of initial application. As a result, prior periods are presented in accordance with the previous guidance in ASC 840. The adoption of this standard resulted in the recognition of operating lease right-of-use assets and operating lease liabilities of $184.4 million and $177.0 million, respectively, on the Company’s consolidated balance sheet at adoption relating to its leases for its corporate headquarters at 60 Binney Street in Cambridge, Massachusetts (the “60 Binney Street Lease”), its office and laboratory space in Seattle, Washington, its office space in Zug, Switzerland, and its embedded leases associated with certain of the Company’s contract manufacturing agreements. The application of the standard’s transition guidance required the de-recognition of the 60 Binney Street Lease building asset, financing lease obligation, current portion, and financing lease obligation, net of current portion in the amounts of $149.3 million, $1.4 million, and $153.3 million, respectively, as well as certain other adjustments to related account balances. In adopting ASU 2016-02, the Company recorded a total one-time adjustment of $6.6 million to the opening balance of accumulated deficit as of January 1, 2019 primarily relating to the de-recognition of the 60 Binney Street Lease building asset and related finance lease obligation. As a result of adopting ASU 2016-02, the Company recorded an increase to deferred tax assets and deferred tax liabilities of $5.3 million and $7.1 million, respectively. The $1.8 million net increase to deferred tax liabilities and an offsetting valuation allowance adjustment was recorded through the accumulated deficit as of January 1, 2019, such that there was no tax impact on the Company’s consolidated financial statements as a result of adoption. ASU No. 2017-04, Intangibles – Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment In January 2017, the FASB issued ASU 2017-04, Intangibles – Goodwill and Other (Topic 350): Simplifying the Test for Goodwill Impairment ("ASU 2017-04"). To address concerns over the cost and complexity of the two-step goodwill impairment test, the amendments in this ASU remove the second step of the test. An entity will instead apply a one-step quantitative test and record the amount of goodwill impairment as the excess of a reporting unit's carrying amount over its fair value, not to exceed the total amount of goodwill allocated to the reporting unit. The new guidance does not amend the optional qualitative assessment of goodwill impairment. The new standard will be effective beginning January 1, 2020 and the Company early adopted the provisions of this ASU for purposes of performing its annual goodwill impairment test for 2019 during the fourth quarter of 2019. The adoption of this standard did not have a material impact on the Company’s financial position or results of operations upon adoption. ASU No. 2017-08, Receivables – Nonrefundable Fees and Other Costs (Topic 310-20): Premium Amortization on Purchased Callable Debt Securities In April 2017, the FASB issued ASU 2017-08, Receivables – Nonrefundable Fees and Other Costs (Topic 310-20): Premium Amortization on Purchased Callable Debt Securities (“Subtopic 310-20”). The new standard amends the amortization period for certain purchased callable debt securities held at a premium by shortening the amortization period for the premium to the earliest call date. Subtopic 310-20 calls for a modified retrospective application under which a cumulative-effect adjustment will be made to retained earnings as of the beginning of the first reporting period in which the guidance is adopted. The Company adopted this standard on January 1, 2019 and it did not have a material impact on the Company’s financial position or results of operations upon adoption. ASU No. 2018-02, Income Statement - Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income In February 2018, the FASB issued ASU 2018-02, Income Statement – Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income . The new standard allows for a reclassification from accumulated other comprehensive income to retained earnings for stranded tax effects resulting from the Tax Cuts and Jobs Act. The Company adopted this standard on January 1, 2019 and it did not have a material impact on the Company’s financial position and results of operations upon adoption. ASU No. 2019-07, Codification Updates to SEC Sections – Amendments to SEC Paragraphs Pursuant to SEC Final Rule Releases In July 2019, the FASB issued ASU 2019-07, Codification Updates to SEC Sections , which clarifies or improves the disclosure and presentation requirements of a variety of codification topics by aligning them with the SEC’s regulations, thereby eliminating redundancies and making the codification easier to apply. The Company prospectively adopted this update upon issuance, and it did not have a material impact on its financial position and results of operations. Not yet adopted ASU No. 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements, ASU No. 2019-05 Financial Instruments – Credit Losses (Topic 326): Targeted Transition Relief, ASU No. 2019-11, Codification Improvements to Topic 326, Financial Instruments - Credit Losses In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements ("ASU 2016-13"). The new standard requires that expected credit losses relating to financial assets measured on an amortized cost basis and available-for-sale debt securities be recorded through an allowance for credit losses. It also limits the amount of credit losses to be recognized for available-for-sale debt securities to the amount by which carrying value exceeds fair value and also requires the reversal of previously recognized credit losses if fair value increases. The targeted transition relief standard allows filers an option to irrevocably elect the fair value option of ASC 825-10, Financial Instruments-Overall, applied on an instrument-by-instrument basis for eligible instruments. The new standard will be effective beginning January 1, 2020. The Company is currently evaluating the potential impact ASU 2016-13, and related updates, will have on its financial position and results of operations upon adoption. ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement (“ASU 2018-13”) . The new standard removes certain disclosures, modifies certain disclosures and adds additional disclosures related to fair value measurement. The new standard will be effective beginning January 1, 2020. The adoption of ASU 2018-13 is not expected to have a material impact on the Company's financial position or results of operations upon adoption. ASU No. 2018-15, Intangibles-Goodwill and Other - Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract In August 2018, the FASB issued ASU 2018-15, Intangibles-Goodwill and Other-Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract (“ASU 2018-15”). The amendments in this update align the requirements for capitalizing implementation costs incurred in a hosting arrangement that is a service contract with the requirements for capitalizing implementation costs incurred to develop or obtain internal-use software (and hosting arrangements that include an internal-use software license). The accounting for the service element of a hosting arrangement that is a service contract is not affected by the amendments in this update. The new standard will be effective beginning January 1, 2020. The amendments in this update should be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. The Company is currently evaluating the potential impact ASU 2018-15 may have on its financial position and results of operations upon adoption. ASU No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic 808 and Topic 606 In November 2018, the FASB issued ASU 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic 808 and Topic 606 (“ASU 2018-18”). The amendments in this update clarify that certain transactions between collaborative arrangement participants should be accounted for as revenue when the collaborative arrangement participant is a customer in the context of a unit of account and precludes recognizing as revenue consideration received from a collaborative arrangement participant if the participant is not a customer. The new standard will be effective beginning January 1, 2020. The Company is currently evaluating the potential impact ASU 2018-18 may have on its financial position and results of operations upon adoption. ASU No. 2019-04, Codification Improvements to Topic 326, Financial Instruments – Credit Losses, Topic 815, Derivatives and Hedging, and Topic 825, Financial Instruments In April 2019, the FASB issued ASU 2019-04, Codification Improvements to Topic 326, Financial Instruments – Credit Losses, Topic 815, Derivatives and Hedging, and Topic 825, Financial Instruments (“ASU 2019-04”) . This update provides clarifications for three topics related to financial instruments accounting, some of which apply to the Company. The amendments in this update will be effective beginning January 1, 2020. The adoption of ASU 2019-04 is not expected to have a material impact on the Company’s financial position or results of operations upon adoption. ASU No. 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes ("ASU 2019-12"), which is intended to simplify the accounting for income taxes. ASU 2019-12 removes certain exceptions to the general principles in Topic 740 and also clarifies and amends existing guidance to improve consistent application. The new standard will be effective beginning January 1, 2021. The Company is currently evaluating the potential impact ASU 2019-12 may have on its financial position and results of operations upon adoption. |
Summary of significant accoun_3
Summary of significant accounting policies and basis of presentation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Estimated Useful Lives of Assets | Depreciation and amortization is calculated using the straight-line method over the estimated useful lives of the assets, which are as follows: Asset Estimated useful life Building 40 years Computer equipment and software 3 years Furniture and fixtures 2-5 years Laboratory equipment 2-5 years Leasehold improvements Shorter of the useful life or remaining lease term |
Marketable securities (Tables)
Marketable securities (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Marketable Securities Held | The following table summarizes the marketable securities held at December 31, 2019 and 2018 (in thousands): Amortized cost / cost Unrealized gains Unrealized losses Fair value December 31, 2019 U.S. government agency securities and treasuries $ 633,970 $ 2,014 $ (48) $ 635,936 Certificates of deposit 960 — — 960 Corporate bonds 185,827 824 (43) 186,608 Commercial paper 74,378 — — 74,378 Equity securities 20,017 — (7,147) 12,870 Total $ 915,152 $ 2,838 $ (7,238) $ 910,752 December 31, 2018 U.S. government agency securities and treasuries $ 1,459,649 $ 963 $ (3,011) $ 1,457,601 Certificates of deposit 9,080 — — 9,080 Equity securities 20,017 2,150 — 22,167 Total $ 1,488,746 $ 3,113 $ (3,011) $ 1,488,848 |
Fair value measurements (Tables
Fair value measurements (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Recorded Amount of Assets and Liabilities Measured at Fair Value on Recurring Basis | The following table sets forth the Company’s assets and liabilities that are measured at fair value on a recurring basis as of December 31, 2019 and 2018 (in thousands): Total Quoted Significant Significant December 31, 2019 Assets: Cash and cash equivalents $ 327,214 $ 311,245 $ 15,969 $ — Marketable securities: U.S. government agency securities and treasuries 635,936 — 635,936 — Certificates of deposit 960 — 960 — Commercial paper 74,378 — 74,378 — Corporate bonds 186,608 — 186,608 — Equity securities 12,870 12,870 — — Total assets $ 1,237,966 $ 324,115 $ 913,851 $ — Liabilities: Contingent consideration $ 7,977 $ — $ — $ 7,977 Total liabilities $ 7,977 $ — $ — $ 7,977 December 31, 2018 Assets: Cash and cash equivalents $ 402,579 $ 348,638 $ 53,941 $ — Marketable securities: U.S. government agency securities 1,457,601 — 1,457,601 — Certificates of deposit 9,080 — 9,080 — Equity securities 22,167 22,167 — — Total assets $ 1,891,427 $ 370,805 $ 1,520,622 $ — Liabilities: Contingent consideration $ 5,230 $ — $ — $ 5,230 Total liabilities $ 5,230 $ — $ — $ 5,230 |
Roll-Forward of Fair Value of the Company's Contingent Consideration Obligations | The table below provides a roll-forward of fair value of the Company’s contingent consideration obligations which include Level 3 inputs (in thousands): Year ended December 31, 2019 2018 Beginning balance $ 5,230 $ 2,231 Additions — — Changes in fair value 2,747 2,999 Payments — — Ending balance $ 7,977 $ 5,230 |
Property, plant and equipment_2
Property, plant and equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property, Plant and Equipment Net | Property, plant and equipment, net, consists of the following (in thousands): As of December 31, 2019 2018 Land $ 1,210 $ 1,210 Building 15,664 180,094 Computer equipment and software 6,947 6,365 Office equipment 7,599 5,584 Laboratory equipment 44,560 35,693 Leasehold improvements 33,788 183 Construction-in-progress 77,981 46,669 Total property, plant and equipment 187,749 275,798 Less accumulated depreciation and amortization (36,573) (29,176) Property, plant and equipment, net $ 151,176 $ 246,622 |
Restricted cash (Tables)
Restricted cash (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Other Assets, Noncurrent [Abstract] | |
Schedule of collateralized bank account of financial institution | As of December 31, 2019, and 2018, the Company maintained letters of credit of $54.5 million and $14.5 million, respectively, which are collateralized with a bank account at a financial institution in accordance with the agreement and consisted of the following (in thousands): As of December 31, 2019 2018 60 Binney Street lease $ 13,763 $ 13,763 50 Binney Street sublease 40,072 — Other leases 660 757 Total restricted cash $ 54,495 $ 14,520 |
Accrued expenses and other cu_2
Accrued expenses and other current liabilities (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Payables and Accruals [Abstract] | |
Summary of accrued expenses and other current liabilities | Accrued expenses and other current liabilities consist of the following (in thousands): As of December 31, 2019 2018 Employee compensation $ 44,679 $ 28,567 Manufacturing costs 23,126 21,618 Clinical and contract research organization costs 16,799 11,891 Collaboration research costs 27,142 3,358 Property, plant, and equipment 2,354 5,451 License and milestone fees 300 7,739 Professional fees 1,827 1,830 Financing lease obligation, current portion — 1,424 Other 25,329 17,515 Total accrued expenses and other current liabilities $ 141,556 $ 99,393 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Lease, Cost | The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the year ended December 31, 2019 (in thousands): For the year ended December 31, 2019 Lease cost (1) Operating lease cost $ 35,346 Total lease cost $ 35,346 Other information Operating cash flows used for operating leases $ 31,026 Weighted average remaining lease term 7.4 years Weighted average discount rate 6.70 % (1) Short-term lease costs and variable lease costs incurred by the Company for the twelve months ended December 31, 2019 were immaterial. |
Summary of Future Minimum Commitments | As of December 31, 2019, future minimum commitments under ASC 842 under the Company’s operating leases were as follows (in thousands): As of Maturity of lease liabilities December 31, 2019 2020 $ 33,257 2021 35,816 2022 31,052 2023 31,540 2024 31,552 2025 and thereafter 88,336 Total lease payments 251,553 Less: imputed interest (60,566) Total operating lease liabilities $ 190,987 |
Commitments and contingencies (
Commitments and contingencies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Schedule of Non-cancelable Contractual Obligations | The following table presents non-cancelable contractual obligations arising from these arrangements: Years ended December 31, Purchase 2020 $ 129,950 2021 22,597 2022 24,310 2023 25,040 2024 46,050 2025 and thereafter — Total purchase commitments $ 247,947 |
Common stock and preferred st_2
Common stock and preferred stock (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
Summary of Future Issuance of Common Stock Shares | The Company has reserved for future issuance the following number of shares of common stock (in thousands): As of December 31, 2019 2018 Options to purchase common stock 5,483 4,643 Restricted stock units 1,127 931 2013 Stock Option and Incentive Plan 2,007 1,458 2013 Employee Stock Purchase Plan 147 172 8,764 7,204 |
Collaborative arrangements (Tab
Collaborative arrangements (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Total Transaction Price, Allocation of Total Transaction Price to Identified Performance Obligations Under Arrangement and Amount of Transaction Price Unsatisfied | The following tables summarize the total transaction price, the allocation of the total transaction price to the identified performance obligations under the arrangement, and the amount of the transaction price unsatisfied as of December 31, 2019 (in thousands): Ide-cel transaction price as of December 31, 2019 Up-front non-refundable payment - BMS Collaboration Agreement $ 75,000 Up-front non-refundable payment - Amended BMS Collaboration Agreement 25,000 Ide-cel license fee - Ide-cel License Agreement 10,000 Ide-cel development milestone 10,000 Estimated variable consideration 87,189 $ 207,189 Allocation of Transaction price unsatisfied as of December 31, 2019 Ide-cel research and development services $ 40,912 $ — Ide-cel license and manufacturing services 166,277 17,815 $ 207,189 $ 17,815 The following table summarizes the net collaboration revenue recognized or expense incurred for the joint ide-cel development efforts in the U.S. under ASC 808, including revenue or expense related to the combined performance obligation for the license and vector manufacturing of ide-cel in the U.S. for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (2) ASC 808 ide-cel revenue - U.S. (1) $ — $ 6,255 $ 4,905 ASC 808 ide-cel research and development expense - U.S. (1) $ 32,415 $ 8,689 $ 3,037 (1) As noted above, the calculation of collaboration revenue or research and development expense to be recognized for joint ide-cel development efforts in the U.S. is performed on a quarterly basis. The calculation is independent of previous activity, which may result in fluctuations between revenue and expense recognition period over period, depending on the varying extent of effort performed by each party during the period. (2) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. Revenue related to the combined unit of accounting for the non-US license and vector manufacturing services is accounted for in accordance with Topic 606. The following table summarizes the revenue recognized related to the combined unit of accounting for the ide-cel non-US license and vector manufacturing services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31 2019 2018 2017 (1) ASC 606 ide-cel license and manufacturing revenue - outside of U.S. $ 25,522 $ 35,900 $ 10,372 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. The following tables summarize the total transaction price, the allocation of the total transaction price to the identified performance obligations under the arrangement, and the amount of the transaction price unsatisfied as of December 31, 2019 (in thousands): bb21217 transaction price as of December 31, 2019 bb21217 license fee - bb21217 License Agreement $ 15,000 Estimated variable consideration 26,687 $ 41,687 Allocation of transaction Transaction price unsatisfied as of December 31, 2019 bb21217 research and development services $ 5,444 $ — bb21217 license and manufacturing services 36,243 36,243 $ 41,687 $ 36,243 |
Summary of Revenue Recognized Related to Research and Development Services | The following table summarizes the revenue recognized, or revenue adjustment recorded, related to the ide-cel research and development services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (1) Ide-cel research and development services revenue $ 2,264 $ 5,751 $ 6,208 $ 2,264 $ 5,751 $ 6,208 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. The following table summarizes the revenue recognized related to the bb21217 research and development services for the years ended December 31, 2019, 2018, and 2017 (in thousands): For the years ended December 31, 2019 2018 2017 (1) bb21217 research and development services revenue $ 2,163 $ 2,884 $ 721 $ 2,163 $ 2,884 $ 721 (1) The Company adopted Topic 606 effective January 1, 2018 using the modified retrospective transition method. Therefore, amounts disclosed in the table above pertaining to the year ended December 31, 2017 are presented under previous accounting guidance and are therefore not comparable to the amounts recorded in the current period under Topic 606. |
Changes in Balances of Company's Receivables and Contract Liabilities | Balance at December 31, 2018 Additions Deductions Balance at December 31, 2019 Receivables $ 6,528 $ 11,250 $ (17,378) $ 400 Contract liabilities: Deferred revenue $ 34,939 $ 10,000 $ (26,674) $ 18,265 |
Intangible assets (Tables)
Intangible assets (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of Finite-Lived Intangible Assets | Intangible assets, net of accumulated amortization, are summarized as follows (in thousands): As of December 31, As of December 31, 2019 2018 Cost Accumulated amortization Net Cost Accumulated amortization Net Developed technology $ 30,100 $ (20,694) $ 9,406 $ 30,100 $ (16,931) $ 13,169 In-licensed rights 5,224 (304) 4,920 — — — Total $ 35,324 $ (20,998) $ 14,326 $ 30,100 $ (16,931) $ 13,169 |
Schedule of Finite-Lived Intangible Assets, Future Amortization Expense | The following table summarizes the estimated future amortization for intangible assets for the next five years (in thousands): As of December 31, 2019 2020 $ 4,285 2021 4,285 2022 2,404 2023 522 2024 522 Total $ 12,018 |
Stock-based compensation (Table
Stock-based compensation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Share-based Payment Arrangement [Abstract] | |
Summary of Stock-Based Compensation Expense by Award Type | Stock-based compensation expense recognized by award type is as follows (in thousands): Year Ended December 31, 2019 2018 2017 Stock options $ 95,668 $ 83,449 $ 42,262 Restricted stock units 63,580 26,628 10,495 Employee stock purchase plan 1,381 759 525 $ 160,629 $ 110,836 $ 53,282 |
Schedule of Stock-Based Compensation Expense by Classification | Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 80,139 $ 54,422 $ 26,633 Selling, general and administrative 80,490 56,414 26,649 $ 160,629 $ 110,836 $ 53,282 |
Assumptions Used for the Black-Scholes Option-Pricing Model to Determine the Per Share Weighted Average Fair Value for Options Granted | The fair value of each option issued to employees was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions: Year Ended December 31, 2019 2018 2017 Expected volatility 70.7 % 75.5 % 78.1 % Expected term (in years) 6.0 6.0 6.0 Risk-free interest rate 2.3 % 2.7 % 2.1 % Expected dividend yield 0.0 % 0.0 % 0.0 % |
Summary of Stock Option Activity Under Plan | The following table summarizes the stock option activity under the Company’s equity awards plans: Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2018 4,643 $ 108.56 Granted 1,594 $ 129.76 Exercised (354) $ 50.36 Canceled or forfeited (400) $ 138.61 Outstanding at December 31, 2019 5,483 $ 116.30 7.3 $ 68,511 Exercisable at December 31, 2019 2,853 $ 94.77 6.0 $ 64,899 Vested and expected to vest at December 31, 2019 5,483 $ 116.30 7.3 $ 68,511 |
Summary of Restricted Common Stock Awards | The following table summarizes the restricted stock unit activity under the Company’s equity award plans: Shares Weighted-average Unvested balance at December 31, 2018 931 $ 155.99 Granted 570 128.87 Vested (251) 138.93 Forfeited (123) 155.68 Unvested balance at December 31, 2019 1,127 $ 146.10 |
Income taxes (Tables)
Income taxes (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Schedule Components of Loss Before Income Taxes | The components of loss before income taxes were as follows (in thousands): Year ended December 31, 2019 2018 2017 U.S. $ (649,172) $ (440,473) $ (266,236) Foreign (140,981) (114,965) (69,197) Total $ (790,153) $ (555,438) $ (335,433) |
Summary of Provision for (Benefit from) Income Taxes | The provision for (benefit from) income taxes were as follows (in thousands): Year ended December 31, 2019 2018 2017 Current: Federal $ — $ — $ — State 7 324 115 Foreign 612 222 95 Deferred: Federal (966) (307) — State (198) (52) — Foreign — — — Total income tax (benefit) expense $ (545) $ 187 $ 210 |
Reconciliation of Income Tax Provision (Benefit) Computed at the Statutory Federal Income Tax Rate to Effective Income Tax Rate (Provision) Benefit as Reflected in the Financial Statements | A reconciliation of income tax provision (benefit) computed at the statutory federal income tax rate to the Company’s effective income tax rate (provision) benefit as reflected in the financial statements is as follows: Year ended December 31, 2019 2018 2017 Federal income tax expense at statutory rate 21.0 % 21.0 % 34.0 % State income tax, net of federal benefit 3.7 % 5.1 % 4.3 % Permanent differences (1.1) % 0.9 % 2.7 % Research and development credit 5.4 % 6.5 % 12.8 % Foreign differential (3.7) % (4.4) % (6.9) % Federal tax rate change — % 0.1 % (31.6) % Other 0.4 % (0.1) % (0.8) % Change in valuation allowance (25.6) % (29.1) % (14.6) % Effective income tax rate benefit (expense) 0.1 % — % (0.1) % |
Components of Deferred Tax Assets and Liabilities | The significant components of the Company’s deferred tax assets and liabilities are composed of the following (in thousands): Year ended December 31, 2019 2018 Deferred tax assets: U.S. net operating loss carryforwards (federal and state) $ 439,839 $ 298,701 Tax credit carryforwards (federal and state) 213,810 167,517 Capitalized license fees and research and development expenses 16,295 18,083 60 Binney Street lease — 42,059 Deferred revenue 15,119 21,442 Stock-based compensation 46,111 31,858 Lease liabilities 52,631 — Accruals and other 15,432 8,886 Total deferred tax assets 799,237 588,546 Intangible assets (2,518) (3,579) Right-of-use assets (49,480) — Fixed assets (2,670) (41,472) Less valuation allowance (744,569) (543,495) Net deferred taxes $ — $ — |
Reconciliation of Unrecognized Tax Benefits | A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands): Unrecognized tax benefits Balance as of December 31, 2017 $ — Increases for tax positions related to current period 3,370 Increases for tax positions related to prior periods 8,725 Balance as of December 31, 2018 12,095 Increases for tax positions related to current period 3,554 Increases for tax positions related to prior periods 296 Balance as of December 31, 2019 15,945 |
Net loss per share (Tables)
Net loss per share (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share | The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect (in thousands): Year ended December 31, 2019 2019 2018 2017 Outstanding stock options 5,483 4,643 3,755 Restricted stock units 1,127 931 477 ESPP shares 19 10 9 6,629 5,584 4,241 |
Selected quarterly financial _2
Selected quarterly financial data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Information | The following table contains quarterly financial information for 2019 and 2018. The Company believes that the following information reflects all normal recurring adjustments necessary for a fair presentation of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2019 First Second Third Fourth Total (in thousands, except per share data) Total revenues $ 12,471 $ 13,296 $ 8,910 $ 9,997 $ 44,674 Total operating expenses 183,645 215,998 219,326 240,531 859,500 Loss from operations (171,174) (202,702) (210,416) (230,534) (814,826) Net loss (164,446) (195,782) (206,033) (223,347) (789,608) Net loss per share applicable to common $ (2.99) $ (3.55) $ (3.73) $ (4.04) $ (14.31) 2018 First Second Third Fourth Total (in thousands, except per share data) Total revenues $ 15,957 $ 7,851 $ 11,528 $ 19,243 $ 54,579 Total operating expenses 132,586 156,465 161,347 176,204 626,602 Loss from operations (116,629) (148,614) (149,819) (156,961) (572,023) Net loss (115,126) (145,996) (145,480) (149,023) (555,625) Net loss per share applicable to common $ (2.31) $ (2.91) $ (2.73) $ (2.72) $ (10.68) |
Description of the business - A
Description of the business - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||||||||||
Accumulated deficit | $ (2,281,852) | $ (1,498,808) | $ (2,281,852) | $ (1,498,808) | |||||||
Net loss | (223,347) | $ (206,033) | $ (195,782) | $ (164,446) | $ (149,023) | $ (145,480) | $ (145,996) | $ (115,126) | (789,608) | (555,625) | $ (335,643) |
Net Cash Provided by (Used in) Operating Activities | (564,384) | $ (413,426) | $ (280,553) | ||||||||
Cash, cash equivalents and marketable securities | $ 1,240,000 | $ 1,240,000 |
Summary of significant accoun_4
Summary of significant accounting policies and basis of presentation - Estimated Useful Lives of Assets (Detail) | 12 Months Ended |
Dec. 31, 2019 | |
Building | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 40 years |
Computer equipment and software | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 3 years |
Furniture and fixtures | Minimum | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 2 years |
Furniture and fixtures | Maximum | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 5 years |
Laboratory equipment | Minimum | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 2 years |
Laboratory equipment | Maximum | |
Property, Plant and Equipment [Line Items] | |
Estimated useful lives of assets | 5 years |
Summary of significant accoun_5
Summary of significant accounting policies and basis of presentation - Additional Information (Detail) | 12 Months Ended | |||||
Dec. 31, 2019USD ($)target | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Jan. 01, 2019USD ($) | Jan. 01, 2018USD ($) | Jan. 01, 2017USD ($) | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Number of operating segments | target | 1 | |||||
Goodwill, impaired, accumulated impairment loss | $ 0 | |||||
Interest income net of amortization of premium and accretion of discount | 34,800,000 | $ 30,100,000 | $ 9,500,000 | |||
Interest expenses | 0 | 15,500,000 | $ 11,400,000 | |||
Operating lease right-of-use assets | 185,885,000 | |||||
Total operating lease liabilities | 190,987,000 | |||||
Property, plant and equipment, net | 151,176,000 | 246,622,000 | ||||
Financing lease obligation, current portion | 0 | 1,424,000 | ||||
Cumulative effect of new accounting principle in period of adoption | $ 0 | |||||
Deferred tax assets, gross | 799,237,000 | 588,546,000 | $ 5,300,000 | |||
Deferred tax liabilities, gross | 7,100,000 | |||||
Deferred tax liabilities, net | $ 0 | $ 0 | 1,800,000 | |||
Accumulated Deficit | ||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Cumulative effect of new accounting principle in period of adoption | 6,564,000 | $ (29,375,000) | $ (491,000) | |||
Accounting Standards Update 2016-02 | ||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Operating lease right-of-use assets | 184,400,000 | |||||
Total operating lease liabilities | 177,000,000 | |||||
Financing lease obligation, current portion | 1,400,000 | |||||
Finance lease, liability, noncurrent | 153,300,000 | |||||
60 Binney Street lease | Accounting Standards Update 2016-02 | ||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||||||
Operating lease right-of-use assets | 127,300,000 | |||||
Total operating lease liabilities | 125,800,000 | |||||
Property, plant and equipment, net | $ 149,300,000 |
Marketable securities - Additio
Marketable securities - Additional Information (Detail) - USD ($) | Dec. 31, 2019 | Dec. 31, 2018 |
Investments, Debt and Equity Securities [Abstract] | ||
Debt securities, available-for-sale, noncurrent | $ 0 | $ 0 |
Marketable securities - Summary
Marketable securities - Summary of Marketable Securities Held (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Debt Securities, Available-for-sale [Line Items] | ||
Equity securities, amortized cost / cost | $ 20,017 | $ 20,017 |
Marketable securities, amortized cost / cost | 915,152 | 1,488,746 |
Equity securities, unrealized gains | 0 | 2,150 |
Marketable securities, unrealized gains | 2,838 | 3,113 |
Equity securities, unrealized losses | (7,147) | 0 |
Marketable securities, unrealized losses | (7,238) | (3,011) |
Equity securities | 12,870 | 22,167 |
Marketable securities, fair value | 910,752 | 1,488,848 |
U.S. government agency securities and treasuries | ||
Debt Securities, Available-for-sale [Line Items] | ||
Debt securities, amortized cost / cost | 633,970 | 1,459,649 |
Debt securities, unrealized gains | 2,014 | 963 |
Debt securities, unrealized losses | (48) | (3,011) |
Debt securities, fair value | 635,936 | 1,457,601 |
Certificates of deposit | ||
Debt Securities, Available-for-sale [Line Items] | ||
Debt securities, amortized cost / cost | 960 | 9,080 |
Debt securities, unrealized gains | 0 | 0 |
Debt securities, unrealized losses | 0 | 0 |
Debt securities, fair value | 960 | $ 9,080 |
Corporate bonds | ||
Debt Securities, Available-for-sale [Line Items] | ||
Debt securities, amortized cost / cost | 185,827 | |
Debt securities, unrealized gains | 824 | |
Debt securities, unrealized losses | (43) | |
Debt securities, fair value | 186,608 | |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Debt securities, amortized cost / cost | 74,378 | |
Debt securities, unrealized gains | 0 | |
Debt securities, unrealized losses | 0 | |
Debt securities, fair value | $ 74,378 |
Fair value measurements - Recor
Fair value measurements - Recorded Amount of Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Assets: | ||
Equity securities | $ 12,870 | $ 22,167 |
Liabilities: | ||
Contingent consideration | 7,977 | 5,230 |
U.S. government agency securities and treasuries | ||
Assets: | ||
Marketable securities | 635,936 | 1,457,601 |
Certificates of deposit | ||
Assets: | ||
Marketable securities | 960 | 9,080 |
Commercial paper | ||
Assets: | ||
Marketable securities | 74,378 | |
Corporate bonds | ||
Assets: | ||
Marketable securities | 186,608 | |
Fair value, measurements, recurring | ||
Assets: | ||
Cash and cash equivalents | 327,214 | 402,579 |
Equity securities | 12,870 | 22,167 |
Total assets | 1,237,966 | 1,891,427 |
Liabilities: | ||
Contingent consideration | 7,977 | 5,230 |
Total liabilities | 7,977 | 5,230 |
Fair value, measurements, recurring | U.S. government agency securities and treasuries | ||
Assets: | ||
Marketable securities | 635,936 | |
Fair value, measurements, recurring | U.S. government agency securities | ||
Assets: | ||
Marketable securities | 1,457,601 | |
Fair value, measurements, recurring | Certificates of deposit | ||
Assets: | ||
Marketable securities | 960 | |
Fair value, measurements, recurring | Commercial paper | ||
Assets: | ||
Marketable securities | 74,378 | |
Fair value, measurements, recurring | Corporate bonds | ||
Assets: | ||
Marketable securities | 186,608 | |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | ||
Assets: | ||
Cash and cash equivalents | 311,245 | 348,638 |
Equity securities | 12,870 | 22,167 |
Total assets | 324,115 | 370,805 |
Liabilities: | ||
Contingent consideration | 0 | 0 |
Total liabilities | 0 | 0 |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | U.S. government agency securities and treasuries | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | U.S. government agency securities | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | Certificates of deposit | ||
Assets: | ||
Marketable securities | 0 | 0 |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | Commercial paper | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Quoted prices in active markets (Level 1) | Corporate bonds | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | ||
Assets: | ||
Cash and cash equivalents | 15,969 | 53,941 |
Equity securities | 0 | 0 |
Total assets | 913,851 | 1,520,622 |
Liabilities: | ||
Contingent consideration | 0 | 0 |
Total liabilities | 0 | 0 |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | U.S. government agency securities and treasuries | ||
Assets: | ||
Marketable securities | 635,936 | |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | U.S. government agency securities | ||
Assets: | ||
Marketable securities | 1,457,601 | |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | Certificates of deposit | ||
Assets: | ||
Marketable securities | 960 | 9,080 |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | Commercial paper | ||
Assets: | ||
Marketable securities | 74,378 | |
Fair value, measurements, recurring | Significant other observable inputs (Level 2) | Corporate bonds | ||
Assets: | ||
Marketable securities | 186,608 | |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | ||
Assets: | ||
Cash and cash equivalents | 0 | 0 |
Equity securities | 0 | 0 |
Total assets | 0 | 0 |
Liabilities: | ||
Contingent consideration | 7,977 | 5,230 |
Total liabilities | 7,977 | 5,230 |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | U.S. government agency securities and treasuries | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | U.S. government agency securities | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | Certificates of deposit | ||
Assets: | ||
Marketable securities | 0 | $ 0 |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | Commercial paper | ||
Assets: | ||
Marketable securities | 0 | |
Fair value, measurements, recurring | Significant unobservable inputs (Level 3) | Corporate bonds | ||
Assets: | ||
Marketable securities | $ 0 |
Fair value measurements - Addit
Fair value measurements - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Unrealized loss on debt securities, less than twelve months | $ 67,200,000 | $ 787,500,000 |
Unrealized loss on debt securities, more than twelve months | 79,600,000 | 315,300,000 |
Debt securities, available-for-sale, continuous unrealized loss position, less than 12 Months, accumulated loss | 100,000 | 900,000 |
Debt securities, available-for-sale, continuous unrealized loss position, 12 months or longer, accumulated loss | 100,000 | 2,100,000 |
Investments with other-than-temporary impairment | 0 | 0 |
Investment at fair value | $ 12,870,000 | 22,167,000 |
Minimum | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Milestone discount rates | 12.70% | |
Maximum | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Milestone discount rates | 14.30% | |
Other Income (Expense), Net | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Unrealized gain on equity securities | $ 9,300,000 | $ 2,200,000 |
Fair value measurements - Roll-
Fair value measurements - Roll-Forward of Fair Value of the Company's Contingent Consideration Obligations (Detail) - Significant unobservable inputs (Level 3) - Contingent consideration obligations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward] | ||
Beginning balance | $ 5,230 | $ 2,231 |
Additions | 0 | 0 |
Changes in fair value | 2,747 | 2,999 |
Payments | 0 | 0 |
Ending balance | $ 7,977 | $ 5,230 |
Property, plant and equipment_3
Property, plant and equipment, net - Summary of Property, Plant and Equipment Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | $ 187,749 | $ 275,798 |
Less accumulated depreciation and amortization | (36,573) | (29,176) |
Property, plant and equipment, net | 151,176 | 246,622 |
Land | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 1,210 | 1,210 |
Building | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 15,664 | 180,094 |
Computer equipment and software | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 6,947 | 6,365 |
Office equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 7,599 | 5,584 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 44,560 | 35,693 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | 33,788 | 183 |
Construction-in-progress | ||
Property, Plant and Equipment [Line Items] | ||
Total property, plant and equipment | $ 77,981 | $ 46,669 |
Property, plant and equipment_4
Property, plant and equipment, net - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Property, Plant and Equipment [Line Items] | |||
Depreciation and amortization expense | $ 13,400 | $ 13,400 | $ 9,800 |
Property and equipment, gross | 187,749 | 275,798 | |
Accumulated depreciation, depletion and amortization, property, plant, and equipment | 36,573 | 29,176 | |
Construction In Progress North Carolina Manufacturing Facility | |||
Property, Plant and Equipment [Line Items] | |||
Property and equipment, gross | 74,200 | 40,400 | |
Building | |||
Property, Plant and Equipment [Line Items] | |||
Property and equipment, gross | 15,664 | $ 180,094 | |
Building | Accounting Standards Update 2016-02 | |||
Property, Plant and Equipment [Line Items] | |||
Property and equipment, gross | (156,000) | ||
Accumulated depreciation, depletion and amortization, property, plant, and equipment | $ (6,700) |
Restricted cash - Additional In
Restricted cash - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Debt and Equity Securities, FV-NI [Line Items] | ||
Restricted cash balance | $ 54,495 | $ 14,520 |
Letter of Credit | ||
Debt and Equity Securities, FV-NI [Line Items] | ||
Restricted cash balance | $ 54,500 | $ 14,500 |
Restricted cash - Schedule of C
Restricted cash - Schedule of Collateralized Bank Account of Financial Institution (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Debt and Equity Securities, FV-NI [Line Items] | ||
Total restricted cash | $ 54,495 | $ 14,520 |
60 Binney Street lease | ||
Debt and Equity Securities, FV-NI [Line Items] | ||
Total restricted cash | 13,763 | 13,763 |
50 Binney Street sublease | ||
Debt and Equity Securities, FV-NI [Line Items] | ||
Total restricted cash | 40,072 | 0 |
Other leases | ||
Debt and Equity Securities, FV-NI [Line Items] | ||
Total restricted cash | $ 660 | $ 757 |
Accrued expenses and other cu_3
Accrued expenses and other current liabilities - Summary of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Payables and Accruals [Abstract] | ||
Employee compensation | $ 44,679 | $ 28,567 |
Manufacturing costs | 23,126 | 21,618 |
Clinical and contract research organization costs | 16,799 | 11,891 |
Collaboration research costs | 27,142 | 3,358 |
Property, plant, and equipment | 2,354 | 5,451 |
License and milestone fees | 300 | 7,739 |
Professional fees | 1,827 | 1,830 |
Financing lease obligation, current portion | 0 | 1,424 |
Other | 25,329 | 17,515 |
Total accrued expenses and other current liabilities | $ 141,556 | $ 99,393 |
Leases - Additional Information
Leases - Additional Information (Details) | Mar. 27, 2017 | Sep. 21, 2015USD ($)ft²$ / ft² | Sep. 30, 2019ft²lease_term$ / ft² | Apr. 30, 2019USD ($)ft²$ / ft² | Jul. 31, 2018USD ($)ft²$ / ft² | Dec. 31, 2019USD ($)lease_term | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Mar. 31, 2018USD ($) | Jan. 01, 2019USD ($) | Jun. 03, 2016 |
Lessee, Lease, Description [Line Items] | |||||||||||
Operating lease right-of-use assets | $ 185,885,000 | ||||||||||
Total operating lease liabilities | 190,987,000 | ||||||||||
lease arrangements annual rent increase percentage | 3.00% | ||||||||||
Operating lease, expense | 45,300,000 | ||||||||||
Rent expense | $ 9,800,000 | $ 9,000,000 | |||||||||
Accounting Standards Update 2016-02 | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Operating lease right-of-use assets | $ 184,400,000 | ||||||||||
Total operating lease liabilities | 177,000,000 | ||||||||||
Seattle, Washington | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lease building space (in sq ft) | ft² | 22,188 | 36,126 | |||||||||
Annual lease rent per square foot (in dollars per sq ft) | $ / ft² | 62.80 | 54 | |||||||||
Lease rent, annual increase percentage | 2.50% | 2.50% | |||||||||
Lease extension terms | 5 years | ||||||||||
Tenant improvement allowance per square foot | $ 215 | ||||||||||
Tenant improvement allowance | $ 8,000,000 | ||||||||||
Proceeds from tenant allowance | $ 8,000,000 | ||||||||||
Lessee, operating lease, term of contract extension | 16 months | ||||||||||
Lessee, operating lease, number of contracts | lease_term | 2 | ||||||||||
60 Binney Street lease | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lessee, operating lease, number of terms | lease_term | 2 | ||||||||||
Lease extension terms | 5 years | ||||||||||
Estimated useful lives of assets | 40 years | ||||||||||
60 Binney Street lease | Accounting Standards Update 2016-02 | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Operating lease right-of-use assets | 127,300,000 | ||||||||||
Total operating lease liabilities | $ 125,800,000 | ||||||||||
50 Binney Street sublease | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lease building space (in sq ft) | ft² | 267,278 | ||||||||||
Collateralized letter of credit | $ 40,100,000 | ||||||||||
Rate of area for sublease per square feet (in dollars per sq ft) | $ / ft² | 99.95 | ||||||||||
Lessee, operating lease, annual expense | $ 26,700,000 | ||||||||||
Lessee, operating sublease, payment commencement, term | 90 days | ||||||||||
Upfront payment for purchase of furniture and equipment | $ 7,500,000 | ||||||||||
50 Binney Street sublease | Minimum | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lessee, operating sublease, option to postpone commencement, term | 9 months | ||||||||||
Manufacturing Facility | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lessee, operating lease, annual expense | $ 5,100,000 | ||||||||||
Lease period | 12 years | ||||||||||
Milestone paid | $ 12,000,000 | ||||||||||
Lessee, operating lease, lease not yet commenced, amount | $ 8,000,000 | ||||||||||
Lessee, operating lease, termination fees, term | 24 months | ||||||||||
Lease starting on October 1, 2016 | 60 Binney Street lease | |||||||||||
Lessee, Lease, Description [Line Items] | |||||||||||
Lease building space (in sq ft) | ft² | 253,108 | ||||||||||
Annual lease rent per square foot (in dollars per sq ft) | $ / ft² | 72.50 | ||||||||||
Lease payments base annual rent | $ 18,400,000 | ||||||||||
Lease rent, annual increase percentage | 1.75% | ||||||||||
Collateralized letter of credit | $ 13,800,000 | ||||||||||
Decrease In letter of credit under the terms of the lease | 9,200,000 | ||||||||||
Landlord contribution for cost of construction and tenant improvements | $ 42,400,000 |
Leases - Summary of Lease Costs
Leases - Summary of Lease Costs and Other Information Pertaining to Operating Leases (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Leases [Abstract] | |
Operating lease cost | $ 35,346 |
Total lease cost | 35,346 |
Operating cash flows used for operating leases | $ 31,026 |
Weighted average remaining lease term | 7 years 4 months 24 days |
Weighted average discount rate | 6.70% |
Short-term lease, cost | $ 0 |
Variable lease, cost | $ 0 |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Commitments Under Operating Leases (Details) $ in Thousands | Dec. 31, 2019USD ($) |
Leases [Abstract] | |
2020 | $ 33,257 |
2021 | 35,816 |
2022 | 31,052 |
2023 | 31,540 |
2024 | 31,552 |
2025 and thereafter | 88,336 |
Total lease payments | 251,553 |
Less: imputed interest | (60,566) |
Total operating lease liabilities | $ 190,987 |
Commitments and contingencies -
Commitments and contingencies - Additional Information (Detail) - USD ($) $ in Millions | 1 Months Ended | |
Jan. 31, 2020 | Jun. 30, 2014 | |
Subsequent Event | ||
Commitments And Contingencies Disclosure [Line Items] | ||
Contractual obligation, amendment term increase | 1 year | |
Contractual obligation, amendment increase | $ 24.7 | |
Pregenen | ||
Commitments And Contingencies Disclosure [Line Items] | ||
Contingent future cash payments | $ 120 | |
Pregenen | Clinical Milestone Payments | ||
Commitments And Contingencies Disclosure [Line Items] | ||
Contingent future cash payments | 20.1 | |
Pregenen | Commercial Milestones Payments | ||
Commitments And Contingencies Disclosure [Line Items] | ||
Contingent future cash payments | $ 99.9 |
Commitments and contingencies_2
Commitments and contingencies - Schedule of Non-cancelable Contractual Obligations (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Commitments and Contingencies Disclosure [Abstract] | |
2020 | $ 129,950 |
2021 | 22,597 |
2022 | 24,310 |
2023 | 25,040 |
2024 | 46,050 |
Purchase Obligation, Due after Fifth Year | 0 |
Total purchase commitments | $ 247,947 |
Common stock and preferred st_3
Common stock and preferred stock - Additional Information (Detail) $ / shares in Units, $ in Millions | 1 Months Ended | ||||
Aug. 31, 2018USD ($)$ / sharesshares | Jul. 31, 2018USD ($)$ / sharesshares | Jan. 31, 2018USD ($)$ / sharesshares | Dec. 31, 2019voteshares | Dec. 31, 2018shares | |
Equity [Abstract] | |||||
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 | |||
Common stock, number of votes (in votes per share) | vote | 1 | ||||
Common stock, shares issued (in shares) | 55,368,000 | 54,738,000 | |||
Common stock, shares outstanding (in shares) | 55,368,000 | 54,738,000 | |||
Number of shares issued in public offering (in shares) | 400,000 | 3,900,000 | 300,000 | ||
Shares issued (in dollars per share) | $ / shares | $ 238.10 | $ 162.50 | $ 185 | ||
Proceeds from public offering of common stock, net of issuance costs | $ | $ 100 | $ 600.6 | $ 48.7 | ||
Common stock amount attributable to prepayment of joint research activities | $ | $ 45.5 | ||||
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 | |||
Preferred Stock, shares issued (in shares) | 0 | 0 | |||
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock and preferred st_4
Common stock and preferred stock - Summary of Future Issuance of Common Stock Shares (Detail) - shares shares in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Schedule of Capitalization [Line Items] | ||
Common shares reserved for future issuance (in shares) | 8,764 | 7,204 |
2013 Stock Option and Incentive Plan | ||
Schedule of Capitalization [Line Items] | ||
Common shares reserved for future issuance (in shares) | 2,007 | 1,458 |
Options to purchase common stock | ||
Schedule of Capitalization [Line Items] | ||
Common shares reserved for future issuance (in shares) | 5,483 | 4,643 |
Restricted stock units | ||
Schedule of Capitalization [Line Items] | ||
Common shares reserved for future issuance (in shares) | 1,127 | 931 |
2013 Employee Stock Purchase Plan | ||
Schedule of Capitalization [Line Items] | ||
Common shares reserved for future issuance (in shares) | 147 | 172 |
Collaborative arrangements - Ad
Collaborative arrangements - Additional Information (Detail) | Aug. 24, 2018USD ($)target$ / sharesshares | Sep. 28, 2017USD ($) | Feb. 16, 2016USD ($) | Mar. 19, 2013USD ($) | Jun. 30, 2019USD ($) | Dec. 31, 2019USD ($)shares | Dec. 31, 2018USD ($)shares | Dec. 31, 2017USD ($) | Aug. 03, 2018USD ($) | Mar. 28, 2018USD ($) | Jun. 03, 2015USD ($)product |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Investment in common stock | $ 0 | $ 649,368,000 | $ 1,006,570,000 | ||||||||
Joint research activities remaining to be recognized | 38,200,000 | $ 44,000,000 | |||||||||
Common shares | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Common stock shares issued (in shares) | shares | 420,000 | ||||||||||
Bristol-Myers Squibb | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Contract with customer liability | 18,265,000 | $ 34,939,000 | |||||||||
Term of collaboration agreement | 3 years | ||||||||||
Deferred revenue balance recognized as gross revenues | 17,600,000 | ||||||||||
Bristol-Myers Squibb | BMS collaborative arrangement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Contract with customer liability | $ 75,000,000 | ||||||||||
Bristol-Myers Squibb | Amended collaborative arrangement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Contract with customer liability | $ 25,000,000 | ||||||||||
Term of collaboration agreement | 3 years | ||||||||||
Products, number of candidates | product | 2 | ||||||||||
Estimated variable consideration | $ 87,200,000 | ||||||||||
Deferred revenue recognition period | 3 years | ||||||||||
Bristol-Myers Squibb | Amended collaborative arrangement | Research and development services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, transaction price | $ 40,900,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel license and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, transaction price | 166,300,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel license and manufacturing services | License and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Contract with customer liability | 8,500,000 | 23,000,000 | |||||||||
Remaining performance obligation revenue | 17,800,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel license agreement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, option fee received | 10,000,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel license agreement | First product candidates | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, option fee received | $ 10,000,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel co-development, co-promote and profit share agreement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Development milestone payments receivable | 10,000,000 | ||||||||||
Contract with customer development milestone payment achieved | $ 10,000,000 | ||||||||||
Contract with customer remaining potential development milestone payment receivable | 60,000,000 | ||||||||||
Bristol-Myers Squibb | Ide-cel co-development, co-promote and profit share agreement | Maximum | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Development milestone payments receivable | $ 70,000,000 | ||||||||||
Bristol-Myers Squibb | bb21217 license agreement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, option fee received | $ 15,000,000 | 15,000,000 | |||||||||
Development milestone payments receivable | 70,000,000 | ||||||||||
Additional fee receivable if option to co-develop and co-promote is not exercised | 10,000,000 | ||||||||||
Estimated variable consideration | 26,700,000 | ||||||||||
Bristol-Myers Squibb | bb21217 license agreement | Maximum | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Clinical milestone payments receivable | 10,000,000 | ||||||||||
Regulatory milestone payments receivable | 117,000,000 | ||||||||||
Commercial milestone payments receivable | $ 78,000,000 | ||||||||||
Bristol-Myers Squibb | bb21217 license agreement | Research and development services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, transaction price | 5,400,000 | ||||||||||
Bristol-Myers Squibb | bb21217 license and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, transaction price | 36,243,000 | ||||||||||
Remaining performance obligation revenue | 36,243,000 | ||||||||||
Bristol-Myers Squibb | bb21217 license and manufacturing services | License and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Contract with customer liability | 9,800,000 | 9,800,000 | |||||||||
Remaining performance obligation revenue | 36,200,000 | ||||||||||
Deferred revenue balance recognized as gross revenues | 0 | 0 | 0 | ||||||||
Regeneron Collaboration Agreement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Number of initial collaboration targets | target | 6 | ||||||||||
Research collaboration term | 5 years | ||||||||||
Investment in common stock | 0 | 54,484,000 | $ 0 | ||||||||
Regeneron Collaboration Agreement | Collaboration revenue | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Deferred revenue balance recognized as gross revenues | 5,700,000 | $ 1,600,000 | |||||||||
Regeneron Collaboration Agreement | Maximum | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Milestone payments receivable | $ 130,000,000 | ||||||||||
Regeneron Collaboration Agreement | Research and development services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Collaboration agreement, transaction price | 100,000,000 | ||||||||||
Purchase price premium | $ 37,000,000 | ||||||||||
Collaborative arrangement amortization period | 5 years | ||||||||||
Collaborative arrangement amount attributed to equity sold | $ 54,500,000 | ||||||||||
Collaborative arrangement amount attributed to joint research activities | $ 45,500,000 | ||||||||||
Regeneron Collaboration Agreement | Share purchase agreement | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Common stock shares issued (in shares) | shares | 400,000 | ||||||||||
Common stock price per share (in dollars per share) | $ / shares | $ 238.10 | ||||||||||
Investment in common stock | $ 100,000,000 | ||||||||||
Purchase price premium | $ 37,000,000 | ||||||||||
Collaborative arrangement research initial funding obligation, percentage | 50.00% | ||||||||||
Regeneron Collaboration Agreement | Share purchase agreement | Common shares | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Common stock shares issued (in shares) | shares | 400,000 | ||||||||||
Investment in common stock | $ 63,000,000 |
Collaborative arrangements - Su
Collaborative arrangements - Summary of Total Transaction Price (Detail) - Bristol-Myers Squibb - USD ($) $ in Thousands | Sep. 28, 2017 | Dec. 31, 2019 |
BMS collaborative arrangement | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Up-front non-refundable payment | $ 75,000 | |
Amended Celgene collaboration agreement | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Up-front non-refundable payment | 25,000 | |
Ide-cel license agreement | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, license fee | 10,000 | |
Ide-cel co-development, co-promote and profit share agreement | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Development milestone payments receivable | 10,000 | |
Vectors and associated payload | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Estimated variable consideration | 87,189 | |
Ide-cel Research And Development Services | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 40,912 | |
Transaction price unsatisfied | 0 | |
Ide-cel Manufacturing Services | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 166,277 | |
Transaction price unsatisfied | 17,815 | |
Ide-cel Transaction Price | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 207,189 | |
Transaction price unsatisfied | 17,815 | |
bb21217 transaction price | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 41,687 | |
Transaction price unsatisfied | 36,243 | |
bb21217 license agreement | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, license fee | $ 15,000 | 15,000 |
Development milestone payments receivable | $ 70,000 | |
Estimated variable consideration | 26,700 | |
Manufacturing services | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Estimated variable consideration | 26,687 | |
bb21217 research and development services | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 5,444 | |
Transaction price unsatisfied | 0 | |
bb21217 license and manufacturing services | ||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | ||
Collaboration agreement, transaction price | 36,243 | |
Transaction price unsatisfied | $ 36,243 |
Collaborative arrangements - _2
Collaborative arrangements - Summary of Revenue Recognized Related to Research and Development Services (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | $ 9,997 | $ 8,910 | $ 13,296 | $ 12,471 | $ 19,243 | $ 11,528 | $ 7,851 | $ 15,957 | $ 44,674 | $ 54,579 | $ 35,427 |
Ide-cel Research and Development | Balances Without Adoption of Topic 606 | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | 6,208 | ||||||||||
Bristol-Myers Squibb | Ide-cel Research and Development | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | 2,264 | 5,751 | |||||||||
Bristol-Myers Squibb | bb21217 research and development services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | $ 2,163 | $ 2,884 | |||||||||
Bristol-Myers Squibb | bb21217 research and development services | Balances Without Adoption of Topic 606 | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | $ 721 |
Collaborative arrangements - _3
Collaborative arrangements - Summary of Revenue Recognized or Expense Incurred (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Research and development | $ 582,413 | $ 448,589 | $ 273,040 | ||||||||
Revenue | $ 9,997 | $ 8,910 | $ 13,296 | $ 12,471 | $ 19,243 | $ 11,528 | $ 7,851 | $ 15,957 | 44,674 | 54,579 | 35,427 |
Bristol-Myers Squibb | U.S. | License and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue from Collaborative Arrangement, Excluding Revenue from Contract with Customer | 0 | 6,255 | |||||||||
Bristol-Myers Squibb | U.S. | License and manufacturing services | Balances Without Adoption of Topic 606 | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue from Collaborative Arrangement, Excluding Revenue from Contract with Customer | 4,905 | ||||||||||
Bristol-Myers Squibb | U.S. | Ide-cel license and manufacturing services | License and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Research and development | 32,415 | 8,689 | |||||||||
Bristol-Myers Squibb | U.S. | Ide-cel license and manufacturing services | License and manufacturing services | Balances Without Adoption of Topic 606 | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Research and development | 3,037 | ||||||||||
Bristol-Myers Squibb | Outside of U.S. | Ide-cel license and manufacturing services | License and manufacturing services | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | $ 25,522 | $ 35,900 | |||||||||
Bristol-Myers Squibb | Outside of U.S. | Ide-cel license and manufacturing services | License and manufacturing services | Balances Without Adoption of Topic 606 | |||||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||||
Revenue | $ 10,372 |
Collaborative arrangements - Ch
Collaborative arrangements - Changes in Balances of Company's Receivables and Contract Liabilities (Detail) - Bristol-Myers Squibb $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Receivables | |
Balance at beginning of period | $ 6,528 |
Additions | 11,250 |
Deductions | (17,378) |
Balance at end of period | 400 |
Contract liabilities: | |
Balance at beginning of period | 34,939 |
Additions | 10,000 |
Deductions | (26,674) |
Balance at end of period | $ 18,265 |
License and royalty revenue - A
License and royalty revenue - Additional Information (Detail) | Apr. 28, 2017USD ($)performance_obligation | Apr. 26, 2017USD ($)performance_obligation | Aug. 31, 2017USD ($) | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Jun. 30, 2017USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) |
License And Royalty Revenue [Line Items] | |||||||||||||||
Revenue | $ 9,997,000 | $ 8,910,000 | $ 13,296,000 | $ 12,471,000 | $ 19,243,000 | $ 11,528,000 | $ 7,851,000 | $ 15,957,000 | $ 44,674,000 | $ 54,579,000 | $ 35,427,000 | ||||
Cost of license and royalty revenue | 2,978,000 | 885,000 | 1,527,000 | ||||||||||||
License and royalty revenue | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Revenue | 8,205,000 | 2,226,000 | $ 13,220,000 | ||||||||||||
Novartis Pharma AG | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
License agreement upfront payment | $ 7,500,000 | ||||||||||||||
Revenue recognized upon achievement of related milestone | $ 2,500,000 | ||||||||||||||
Novartis Pharma AG | Royalty | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Revenue | 8,200,000 | 2,200,000 | |||||||||||||
Novartis Pharma AG | License and royalty revenue | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Cost of license and royalty revenue | 3,000,000 | 900,000 | |||||||||||||
Novartis Pharma AG | Topic 606 | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Number of performance obligation identified at the date of contract inception | performance_obligation | 1 | ||||||||||||||
Revenue | $ 7,500,000 | ||||||||||||||
Novartis Pharma AG | Topic 606 | Regulatory Milestones Payments | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Revenue | 2,500,000 | ||||||||||||||
Orchard Therapeutics Limited | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
License agreement upfront payment | $ 3,000,000 | ||||||||||||||
Orchard Therapeutics Limited | License and royalty revenue | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Cost of license and royalty revenue | 0 | 0 | |||||||||||||
Orchard Therapeutics Limited | Topic 606 | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Number of performance obligation identified at the date of contract inception | performance_obligation | 1 | ||||||||||||||
Revenue | $ 3,000,000 | ||||||||||||||
Number of unsatisfied performance obligation identified at date of contract inception | performance_obligation | 0 | ||||||||||||||
Orchard Therapeutics Limited | Topic 606 | License | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Revenue | $ 0 | $ 0 | |||||||||||||
Regulatory Milestones Payments | Novartis Pharma AG | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Amount per product eligible to be received upon achievement of specified event | $ 7,500,000 | ||||||||||||||
Each Subsequently Licensed Product | Novartis Pharma AG | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Amount per product eligible to be received upon achievement of specified event | $ 1,100,000 | ||||||||||||||
Potential Milestones Payments | Orchard Therapeutics Limited | |||||||||||||||
License And Royalty Revenue [Line Items] | |||||||||||||||
Amount per product eligible to be received upon achievement of specified event | $ 1,300,000 |
Intangible assets - Additional
Intangible assets - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Finite-Lived Intangible Assets [Line Items] | |||
Amortization expense | $ 4.1 | $ 3.8 | $ 3.8 |
Developed technology | |||
Finite-Lived Intangible Assets [Line Items] | |||
Gene editing platform expected useful life | 8 years | ||
In-licensed rights | |||
Finite-Lived Intangible Assets [Line Items] | |||
Gene editing platform expected useful life | 10 years |
Intangible assets - Schedule of
Intangible assets - Schedule of Finite-Lived Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Finite-Lived Intangible Assets [Line Items] | ||
Cost | $ 35,324 | $ 30,100 |
Accumulated amortization | (20,998) | (16,931) |
Net | 14,326 | 13,169 |
Developed technology | ||
Finite-Lived Intangible Assets [Line Items] | ||
Cost | 30,100 | 30,100 |
Accumulated amortization | (20,694) | (16,931) |
Net | 9,406 | 13,169 |
In-licensed rights | ||
Finite-Lived Intangible Assets [Line Items] | ||
Cost | 5,224 | 0 |
Accumulated amortization | (304) | 0 |
Net | $ 4,920 | $ 0 |
Intangible assets - Schedule _2
Intangible assets - Schedule of Finite-Lived Intangible Assets, Future Amortization Expense (Details) $ in Thousands | Dec. 31, 2019USD ($) |
Goodwill and Intangible Assets Disclosure [Abstract] | |
2020 | $ 4,285 |
2021 | 4,285 |
2022 | 2,404 |
2023 | 522 |
2024 | 522 |
Total | $ 12,018 |
Stock-based compensation - Addi
Stock-based compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||||
Jan. 31, 2020 | Jan. 31, 2019 | Jun. 30, 2019 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jun. 24, 2013 | Jun. 03, 2013 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Increased number of issuance of awards under the 2013 Plan (in shares) | 2,200,000 | |||||||
Number of shares available for issuance (in shares) | 2,200,000 | |||||||
Stock-based compensation expense | $ 160,629 | $ 110,836 | $ 53,282 | |||||
Weighted average grant date fair value of options granted (in dollars per share) | $ 83.44 | $ 125.12 | $ 62.03 | |||||
Intrinsic value of stock options exercised | $ 29,000 | $ 73,100 | $ 91,000 | |||||
Subsequent Event | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Increased number of issuance of awards under the 2013 Plan (in shares) | 2,200,000 | |||||||
Performance and service condition based restricted stock unit | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Stock-based compensation expense | $ 20,100 | |||||||
Unvested restricted stock awards outstanding (in shares) | 200,000 | |||||||
Stock options | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Stock-based compensation expense | $ 95,668 | 83,449 | 42,262 | |||||
Unrecognized stock- based compensation expense related to unvested stock options, restricted stock awards and employee stock purchase plan | $ 208,100 | |||||||
Expected weighted-average period related to unvested stock options, restricted stock awards and employee stock purchase plan | 2 years 6 months | |||||||
Restricted stock units | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Stock-based compensation expense | $ 63,580 | $ 26,628 | 10,495 | |||||
Unvested restricted stock awards outstanding (in shares) | 1,127,000 | 931,000 | ||||||
Unrecognized stock- based compensation expense related to unvested stock options, restricted stock awards and employee stock purchase plan | $ 105,800 | |||||||
Expected weighted-average period related to unvested stock options, restricted stock awards and employee stock purchase plan | 2 years 7 months 6 days | |||||||
Intrinsic value of restricted stock units vested | $ 28,400 | $ 25,500 | 8,100 | |||||
Employee stock purchase plan | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Common shares reserved for future issuance (in shares) | 200,000 | |||||||
Stock-based compensation expense | 1,381 | $ 759 | $ 525 | |||||
Unrecognized stock- based compensation expense related to unvested stock options, restricted stock awards and employee stock purchase plan | $ 100 | |||||||
Expected weighted-average period related to unvested stock options, restricted stock awards and employee stock purchase plan | 1 month 6 days | |||||||
Share-based compensation arrangement by share-based payment award, shares issued in period (less than) (in shares) | 100,000 | 100,000 | ||||||
2013 Stock Option and Incentive Plan | ||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||
Common shares reserved for future issuance (in shares) | 1,000,000 | |||||||
Percentage of automatic increase in shares reserved and available for issuance determined based on shares outstanding | 4.00% |
Stock-based compensation - Summ
Stock-based compensation - Summary of Stock-Based Compensation Expense by Award Type (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 160,629 | $ 110,836 | $ 53,282 |
Stock options | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | 95,668 | 83,449 | 42,262 |
Restricted stock units | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | 63,580 | 26,628 | 10,495 |
Employee stock purchase plan | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 1,381 | $ 759 | $ 525 |
Stock-based compensation - Sche
Stock-based compensation - Schedule of Stock-Based Compensation Expense by Classification (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 160,629 | $ 110,836 | $ 53,282 |
Research and development | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | 80,139 | 54,422 | 26,633 |
Selling, general and administrative | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 80,490 | $ 56,414 | $ 26,649 |
Stock-based compensation - Assu
Stock-based compensation - Assumptions Used for the Black-Scholes Option-Pricing Model to Determine the Per Share Weighted Average Fair Value for Options Granted (Detail) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Payment Arrangement [Abstract] | |||
Expected volatility | 70.70% | 75.50% | 78.10% |
Expected term (in years) | 6 years | 6 years | 6 years |
Risk-free interest rate | 2.30% | 2.70% | 2.10% |
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Stock-based compensation - Su_2
Stock-based compensation - Summary of Stock Option Activity Under Plan (Detail) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($)$ / sharesshares | |
Shares (in thousands) | |
Outstanding at beginning of period (in shares) | shares | 4,643 |
Granted (in shares) | shares | 1,594 |
Exercised (in shares) | shares | (354) |
Canceled or forfeited (in shares) | shares | (400) |
Outstanding at end of period (in shares) | shares | 5,483 |
Exercisable at end of period (in shares) | shares | 2,853 |
Vested and expected to vest at end of period (in shares) | shares | 5,483 |
Weighted- average exercise price per share | |
Outstanding at beginning of period (in dollars per share) | $ / shares | $ 108.56 |
Granted (in dollars per share) | $ / shares | 129.76 |
Exercised (in dollars per share) | $ / shares | 50.36 |
Canceled or forfeited (in dollars per share) | $ / shares | 138.61 |
Outstanding at end of period (in dollars per share) | $ / shares | 116.30 |
Exercisable at end of period (in dollars per share) | $ / shares | 94.77 |
Vested and expected to vest at end of period (in dollars per share) | $ / shares | $ 116.30 |
Weighted- average contractual life (in years) | |
Outstanding at December 31, 2019 | 7 years 3 months 18 days |
Exercisable at December 31, 2019 | 6 years |
Vested and expected to vest at December 31, 2019 | 7 years 3 months 18 days |
Aggregate intrinsic value (in thousands) | |
Outstanding at end of period | $ | $ 68,511 |
Exercisable at end of period | $ | 64,899 |
Vested and expected to vest at end of period | $ | $ 68,511 |
Stock-based compensation - Su_3
Stock-based compensation - Summary of Restricted Stock Units (Detail) - Restricted stock units shares in Thousands | 12 Months Ended |
Dec. 31, 2019$ / sharesshares | |
Shares (in thousands) | |
Unvested balance at beginning of period (in shares) | shares | 931 |
Granted (in shares) | shares | 570 |
Vested (in shares) | shares | (251) |
Forfeited (in shares) | shares | (123) |
Unvested balance at end of period (in shares) | shares | 1,127 |
Weighted-average grant date fair value | |
Unvested balance at beginning of period (in dollars per share) | $ / shares | $ 155.99 |
Granted (in dollars per share) | $ / shares | 128.87 |
Vested (in dollars per share) | $ / shares | 138.93 |
Forfeited (in dollars per share) | $ / shares | 155.68 |
Unvested balance at end of period (in dollars per share) | $ / shares | $ 146.10 |
401(k) savings plan - Additiona
401(k) savings plan - Additional Information (Detail) - 401 (k) - USD ($) $ in Millions | 1 Months Ended | 12 Months Ended | |||
Mar. 31, 2020 | Feb. 28, 2019 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Defined Contribution Plan Disclosure [Line Items] | |||||
Contributions to the 401(k) Plan | $ 2.4 | ||||
Expenses related to 401(k) Plan | $ 4.6 | $ 2.4 | $ 1.5 | ||
Scenario, Forecast | |||||
Defined Contribution Plan Disclosure [Line Items] | |||||
Contributions to the 401(k) Plan | $ 4.6 |
Income taxes - Schedule of Comp
Income taxes - Schedule of Components of Loss Before Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
U.S. | $ (649,172) | $ (440,473) | $ (266,236) |
Foreign | (140,981) | (114,965) | (69,197) |
Loss before income taxes | $ (790,153) | $ (555,438) | $ (335,433) |
Income taxes - Summary of Provi
Income taxes - Summary of Provision for (Benefit from) Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Current: | |||
Federal | $ 0 | $ 0 | $ 0 |
State | 7 | 324 | 115 |
Foreign | 612 | 222 | 95 |
Deferred: | |||
Federal | (966) | (307) | 0 |
State | (198) | (52) | 0 |
Foreign | 0 | 0 | 0 |
Total income tax (benefit) expense | $ (545) | $ 187 | $ 210 |
Income taxes - Reconciliation o
Income taxes - Reconciliation of Income Tax Provision (Benefit) (Detail) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Federal income tax expense at statutory rate | 21.00% | 21.00% | 34.00% |
State income tax, net of federal benefit | 3.70% | 5.10% | 4.30% |
Permanent differences | (1.10%) | 0.90% | 2.70% |
Research and development credit | 5.40% | 6.50% | 12.80% |
Foreign differential | (3.70%) | (4.40%) | (6.90%) |
Federal tax rate change | 0 | 0.001 | (0.316) |
Other | 0.40% | (0.10%) | (0.80%) |
Change in valuation allowance | (25.60%) | (29.10%) | (14.60%) |
Effective income tax rate benefit (expense) | 0.10% | 0.00% | (0.10%) |
Income taxes - Additional Infor
Income taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | ||||||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jan. 01, 2019 | Jan. 01, 2018 | Jan. 01, 2017 | Dec. 31, 2016 | |
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Income tax expense (benefit) | $ (545,000) | $ 187,000 | $ 210,000 | ||||
Effective Income Tax Rate Reconciliation, Percent | 0.10% | 0.00% | (0.10%) | ||||
Approximately valuation allowance increased | $ (201,100,000) | ||||||
Operating loss carryforwards, without expiration dates | 913,800,000 | ||||||
Operating loss carryforwards, with expiration dates | 711,000,000 | ||||||
Cumulative effect of new accounting principle in period of adoption | $ 0 | ||||||
Cumulative-effect adjustment to retained earnings | 439,839,000 | $ 298,701,000 | |||||
Income tax examination, penalties and interest accrued | $ 0 | 0 | $ 0 | ||||
Accumulated Deficit | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Cumulative effect of new accounting principle in period of adoption | $ 6,564,000 | $ (29,375,000) | (491,000) | ||||
ASU 2016-09 | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Cumulative-effect adjustment to retained earnings | 76,700,000 | ||||||
ASU 2016-09 | Accumulated Deficit | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Cumulative effect of new accounting principle in period of adoption | $ 76,700,000 | ||||||
Research Tax Credit Carryforward | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Limitations on use of net operating losses and tax credit carryforwards, period | 3 years | ||||||
Limitations on use of net operating losses and tax credit carryforwards, percentage | 50.00% | ||||||
U.S. | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Operating loss carryforwards | $ 1,620,000,000 | 1,100,000,000 | 716,100,000 | ||||
U.S. | Research Tax Credit Carryforward | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Tax credit carryforward amount | 203,100,000 | 156,200,000 | 124,100,000 | ||||
State and Local Jurisdiction | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Operating loss carryforwards | 1,560,000,000 | 1,080,000,000 | 692,900,000 | ||||
State and Local Jurisdiction | Excess Equity Based Compensation Tax Deductions | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Operating loss carryforwards | $ 195,400,000 | ||||||
State and Local Jurisdiction | Research Tax Credit Carryforward | |||||||
Components Of Income Tax Expense Benefit [Line Items] | |||||||
Tax credit carryforward amount | $ 13,600,000 | $ 14,300,000 | $ 9,100,000 |
Income taxes - Components of De
Income taxes - Components of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
Deferred tax assets: | |||
U.S. net operating loss carryforwards (federal and state) | $ 439,839 | $ 298,701 | |
Tax credit carryforwards (federal and state) | 213,810 | 167,517 | |
Capitalized license fees and research and development expenses | 16,295 | 18,083 | |
60 Binney Street lease | 0 | 42,059 | |
Deferred revenue | 15,119 | 21,442 | |
Stock-based compensation | 46,111 | 31,858 | |
Lease liabilities | 52,631 | 0 | |
Accruals and other | 15,432 | 8,886 | |
Total deferred tax assets | 799,237 | $ 5,300 | 588,546 |
Intangible assets | (2,518) | (3,579) | |
Right-of-use assets | (49,480) | 0 | |
Fixed assets | (2,670) | (41,472) | |
Less valuation allowance | (744,569) | (543,495) | |
Net deferred taxes | $ 0 | $ (1,800) | $ 0 |
Income taxes - Reconciliation_2
Income taxes - Reconciliation of Unrecognized Tax Benefits (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward] | ||
Balance as of beginning of period | $ 12,095 | $ 0 |
Increases for tax positions related to current period | 3,554 | 3,370 |
Increases for tax positions related to prior periods | 296 | 8,725 |
Balance as of end of period | $ 15,945 | $ 12,095 |
Net loss per share - Common Sto
Net loss per share - Common Stock Equivalents Excluded from Calculation of Diluted Net Loss Per Share (Detail) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from the calculation of diluted net loss per share (in shares) | 6,629 | 5,584 | 4,241 |
Outstanding stock options | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from the calculation of diluted net loss per share (in shares) | 5,483 | 4,643 | 3,755 |
Restricted stock units | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from the calculation of diluted net loss per share (in shares) | 1,127 | 931 | 477 |
ESPP shares | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Common stock equivalents excluded from the calculation of diluted net loss per share (in shares) | 19 | 10 | 9 |
Selected quarterly financial _3
Selected quarterly financial data (unaudited) - Quarterly Financial Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | |||||||||||
Total revenues | $ 9,997 | $ 8,910 | $ 13,296 | $ 12,471 | $ 19,243 | $ 11,528 | $ 7,851 | $ 15,957 | $ 44,674 | $ 54,579 | $ 35,427 |
Total operating expenses | 240,531 | 219,326 | 215,998 | 183,645 | 176,204 | 161,347 | 156,465 | 132,586 | 859,500 | 626,602 | 367,592 |
Loss from operations | (230,534) | (210,416) | (202,702) | (171,174) | (156,961) | (149,819) | (148,614) | (116,629) | (814,826) | (572,023) | (332,165) |
Net loss | $ (223,347) | $ (206,033) | $ (195,782) | $ (164,446) | $ (149,023) | $ (145,480) | $ (145,996) | $ (115,126) | $ (789,608) | $ (555,625) | $ (335,643) |
Net loss per share applicable to common stockholders - basic and diluted: | $ (4.04) | $ (3.73) | $ (3.55) | $ (2.99) | $ (2.72) | $ (2.73) | $ (2.91) | $ (2.31) | $ (14.31) | $ (10.68) | $ (7.71) |
Uncategorized Items - blue-2019
Label | Element | Value |
Additional Paid-in Capital [Member] | ||
Cumulative Effect of New Accounting Principle in Period of Adoption | us-gaap_CumulativeEffectOfNewAccountingPrincipleInPeriodOfAdoption | $ 491,000 |
Accounting Standards Update 2014-09 [Member] | ||
Cumulative Effect of New Accounting Principle in Period of Adoption | us-gaap_CumulativeEffectOfNewAccountingPrincipleInPeriodOfAdoption | (29,375,000) |
Cumulative Effect of New Accounting Principle in Period of Adoption | us-gaap_CumulativeEffectOfNewAccountingPrincipleInPeriodOfAdoption | $ 6,564,000 |