Document and Entity Information
Document and Entity Information - USD ($) $ in Billions | 12 Months Ended | ||
Dec. 31, 2021 | Feb. 11, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Title of 12(b) Security | Common Shares, nominal value CHF 0.03 | ||
Trading Symbol | CRSP | ||
Security Exchange Name | NASDAQ | ||
Entity Registrant Name | CRISPR THERAPEUTICS AG | ||
Entity Central Index Key | 0001674416 | ||
Entity Tax Identification Number | 00-0000000 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Shell Company | false | ||
Entity File Number | 001-37923 | ||
Entity Address, Address Line One | Baarerstrasse 14 | ||
Entity Address, City or Town | Zug | ||
Entity Address, Country | CH | ||
Entity Address, Postal Zip Code | 6300 | ||
City Area Code | 41 (0)41 | ||
Local Phone Number | 561 32 77 | ||
Entity Interactive Data Current | Yes | ||
Entity Incorporation, State or Country Code | V8 | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Common Stock, Shares Outstanding | 77,067,587 | ||
Entity Public Float | $ 11.4 | ||
ICFR Auditor Attestation Flag | false | ||
Documents Incorporated by Reference | Portions of the Registrant’s Definitive Proxy Statement relating to the 2022 Annual General Meeting of Shareholders, which the Registrant intends to file with the Securities and Exchange Commission pursuant to Regulation 14A within 120 days after the end of the Registrant’s fiscal year ended December 31, 2021, are incorporated by reference into Part III of this Report. | ||
Auditor Name | Ernst & Young LLP | ||
Auditor Firm ID | 42 | ||
Auditor Location | Boston, Massachusetts |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 923,031 | $ 1,168,620 |
Marketable securities | 1,456,098 | 521,713 |
Accounts receivable | 305 | 144 |
Prepaid expenses and other current assets | 38,079 | 26,143 |
Total current assets | 2,417,513 | 1,716,620 |
Property and equipment, net | 137,575 | 42,160 |
Intangible assets, net | 125 | 180 |
Restricted cash | 16,913 | 16,848 |
Operating lease assets | 174,460 | 50,865 |
Other non-current assets | 5,291 | 1,293 |
Total assets | 2,751,877 | 1,827,966 |
Current liabilities: | ||
Accounts payable | 14,816 | 9,094 |
Accrued expenses | 91,003 | 53,782 |
Deferred revenue, current | 1,011 | 2,341 |
Accrued tax liabilities | 724 | 10,473 |
Operating lease liabilities | 12,158 | 11,362 |
Other current liabilities | 171 | 7,207 |
Total current liabilities | 119,883 | 94,259 |
Deferred revenue, non-current | 12,323 | 11,776 |
Operating lease liabilities, net of current portion | 212,872 | 50,067 |
Other non-current liabilities | 7,339 | 7,630 |
Total liabilities | 352,417 | 163,732 |
Commitments and contingencies (Note 8) | ||
Shareholders’ equity: | ||
Common shares, CHF 0.03 par value, 145,364,335 and 115,172,786 shares authorized at December 31, 2021 and 2020, respectively, 77,170,164 and 74,110,160 shares issued at December 31, 2021 and 2020, respectively 76,989,848 and 73,914,844 shares outstanding at December 31, 2021 and 2020, respectively | 2,391 | 2,277 |
Treasury shares, at cost, 180,316 and 195,316 shares at December 31, 2021 and 2020, respectively | (63) | (63) |
Additional paid-in capital | 2,598,114 | 2,235,679 |
Accumulated deficit | (195,915) | (573,576) |
Accumulated other comprehensive income (loss) | (5,067) | (83) |
Total shareholders’ equity | 2,399,460 | 1,664,234 |
Total liabilities and shareholders’ equity | $ 2,751,877 | $ 1,827,966 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - SFr / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Common stock, par value | SFr 0.03 | SFr 0.03 |
Common stock, shares authorized | 145,364,335 | 115,172,786 |
Common stock, shares issued | 77,170,382 | 74,110,160 |
Common stock, shares outstanding | 76,990,066 | 73,914,844 |
Treasury stock, shares | 180,316 | 195,316 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive (Loss) Income - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | ||
Revenue: | ||||
Total revenue | $ 914,963 | $ 719 | $ 289,590 | |
Operating expenses: | ||||
Research and development | [1] | 438,633 | 266,946 | 179,362 |
General and administrative | 102,802 | 88,208 | 63,488 | |
Total operating expenses | 541,435 | 355,154 | 242,850 | |
Income (loss) from operations | 373,528 | (354,435) | 46,740 | |
Other income (expense): | ||||
Loss from equity method investment | (5,467) | |||
Other income, net | 6,003 | 6,379 | 26,033 | |
Total other income, net | 6,003 | 6,379 | 20,566 | |
Net income (loss) before income taxes | 379,531 | (348,056) | 67,306 | |
Provision for income taxes | (1,870) | (809) | (448) | |
Net income (loss) | 377,661 | (348,865) | 66,858 | |
Foreign currency translation adjustment | (11) | 40 | 15 | |
Unrealized loss on marketable securities | (4,973) | (130) | ||
Comprehensive income (loss) | $ 372,677 | $ (348,955) | $ 66,873 | |
Net income (loss) per common share - basic | $ 4.97 | $ (5.29) | $ 1.23 | |
Basic weighted-average common shares outstanding | 75,948,686 | 65,949,672 | 54,392,304 | |
Net income (loss) per common share - diluted | $ 4.70 | $ (5.29) | $ 1.17 | |
Diluted weighted-average common shares outstanding | 80,393,496 | 65,949,672 | 56,932,798 | |
Collaboration Revenue [Member] | ||||
Revenue: | ||||
Total revenue | [2] | $ 913,081 | $ 543 | $ 289,590 |
Grant Revenue [Member] | ||||
Revenue: | ||||
Total revenue | $ 1,882 | $ 176 | ||
[1] | Including the following amounts of research and development from a related party, see Note 9 | |||
[2] | Including the following amounts of revenue from a related party, see Note 9 |
Consolidated Statements of Op_2
Consolidated Statements of Operations and Comprehensive (Loss) Income (Parenthetical) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Income Statement [Abstract] | |
Revenue from related party | $ 746 |
Research and development expense with a related party | $ 14,459 |
Consolidated Statements of Shar
Consolidated Statements of Shareholders' Equity - USD ($) $ in Thousands | Total | Common Shares [Member] | Treasury Shares [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Income (Loss) [Member] |
Beginning balance at Dec. 31, 2018 | $ 392,195 | $ 1,584 | $ (57) | $ 682,245 | $ (291,569) | $ (8) |
Beginning balance (in shares) at Dec. 31, 2018 | 51,852,862 | 307,936 | ||||
Issuance of common shares, net of issuance costs | 414,789 | $ 230 | 414,559 | |||
Issuance of common shares, net of issuance costs (in shares) | 7,704,068 | (47,297) | ||||
Vesting of restricted shares | 43 | $ 2 | 41 | |||
Vesting of restricted shares (in shares) | 68,009 | |||||
Exercise of vested options, net of issuance costs | 16,001 | $ 31 | $ (6) | 15,976 | ||
Exercise of vested options, net of issuance costs (in shares) | 1,158,860 | (10,413) | ||||
Stock-based compensation expense | 49,524 | 49,524 | ||||
Other comprehensive income (loss) | 15 | 15 | ||||
Net income (loss) | 66,858 | 66,858 | ||||
Ending balance at Dec. 31, 2019 | 939,425 | $ 1,847 | $ (63) | 1,162,345 | (224,711) | 7 |
Ending balance (in shares) at Dec. 31, 2019 | 60,783,799 | 250,226 | ||||
Issuance of common shares, net of issuance costs | 973,381 | $ 366 | 973,015 | |||
Issuance of common shares, net of issuance costs (in shares) | 11,412,519 | |||||
Vesting of restricted shares | 7 | $ 7 | ||||
Vesting of restricted shares (in shares) | 204,650 | |||||
Exercise of vested options, net of issuance costs | 32,775 | $ 57 | 32,718 | |||
Exercise of vested options, net of issuance costs (in shares) | 1,482,636 | (37,080) | ||||
Purchase of common stock under ESPP | 694 | 694 | ||||
Purchase of common stock under ESPP (in shares) | 13,410 | |||||
Stock-based compensation expense | 66,018 | 66,018 | ||||
Issuance of common stock for license agreements | 889 | 889 | ||||
Issuance of common stock for license agreements (in shares) | 17,830 | (17,830) | ||||
Other comprehensive income (loss) | (90) | (90) | ||||
Net income (loss) | (348,865) | (348,865) | ||||
Ending balance at Dec. 31, 2020 | 1,664,234 | $ 2,277 | $ (63) | 2,235,679 | (573,576) | (83) |
Ending balance (in shares) at Dec. 31, 2020 | 73,914,844 | 195,316 | ||||
Issuance of common shares, net of issuance costs | 222,175 | $ 45 | 222,130 | |||
Issuance of common shares, net of issuance costs (in shares) | 1,353,121 | |||||
Vesting of restricted shares | 15 | $ 15 | ||||
Vesting of restricted shares (in shares) | 455,440 | |||||
Exercise of vested options, net of issuance costs | $ 35,874 | $ 54 | 35,820 | |||
Exercise of vested options, net of issuance costs (in shares) | 1,245,071 | 1,245,071 | (15,000) | |||
Purchase of common stock under ESPP | $ 2,095 | 2,095 | ||||
Purchase of common stock under ESPP (in shares) | 21,590 | |||||
Stock-based compensation expense | 102,390 | 102,390 | ||||
Other comprehensive income (loss) | (4,984) | (4,984) | ||||
Net income (loss) | 377,661 | 377,661 | ||||
Ending balance at Dec. 31, 2021 | $ 2,399,460 | $ 2,391 | $ (63) | $ 2,598,114 | $ (195,915) | $ (5,067) |
Ending balance (in shares) at Dec. 31, 2021 | 76,990,066 | 180,316 |
Consolidated Statements of Sh_2
Consolidated Statements of Shareholders' Equity (Parenthetical) $ in Millions | 12 Months Ended | |||||
Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2021SFr / shares | Dec. 31, 2020SFr / shares | Dec. 31, 2019SFr / shares | |
Common shares, par value | SFr / shares | SFr 0.03 | SFr 0.03 | ||||
Stock Options [Member] | ||||||
Issuance costs | $ | $ 2.6 | $ 1.2 | $ 0.3 | |||
Common Shares [Member] | ||||||
Issuance costs | $ | $ 5.4 | $ 46.4 | $ 25.1 | |||
Common shares, par value | SFr / shares | SFr 0.03 | SFr 0.03 | SFr 0.03 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Operating activities | |||
Net income (loss) | $ 377,661 | $ (348,865) | $ 66,858 |
Reconciliation of net income (loss) to net cash used in operating activities: | |||
Depreciation and amortization | 17,953 | 9,184 | 4,725 |
Equity-based compensation | 102,390 | 66,018 | 44,057 |
Loss from equity method investment in Casebia | 5,467 | ||
Gain from consolidation of Casebia | (16,000) | ||
Net amortization of premiums and discounts on marketable securities | 14,109 | 1,857 | |
Changes in: | |||
Accounts receivable | (161) | (45) | (11) |
Prepaid expenses and other assets | (13,912) | 17,338 | (32,618) |
Accounts payable and accrued expenses | 37,514 | 25,747 | 5,025 |
Deferred revenue | (783) | 1,381 | (45,146) |
Operating lease assets and liabilities | 9,506 | (473) | (663) |
Other liabilities, net | (5,305) | (10,508) | 24,983 |
Net cash provided by (used in) operating activities | 538,972 | (238,366) | 56,677 |
Investing activities | |||
Purchase of property, plant and equipment | (81,705) | (18,358) | (6,684) |
Net cash and restricted cash received in connection with the acquisition of Casebia | 8,009 | ||
Purchases of marketable securities | (1,509,327) | (593,998) | |
Maturities of marketable securities | 555,602 | 71,186 | |
Net cash (used in) provided by investing activities | (1,035,430) | (541,170) | 1,325 |
Financing activities | |||
Proceeds from issuance of common shares, net of issuance costs | 213,267 | 982,289 | 415,019 |
Proceeds from exercise of options and ESPP contributions, net of issuance costs | 37,678 | 33,863 | 15,964 |
Net cash provided by financing activities | 250,945 | 1,016,152 | 430,983 |
Effect of exchange rate changes on cash | (11) | 40 | 15 |
(Decrease) increase in cash | (245,524) | 236,656 | 489,000 |
Cash, cash equivalents and restricted cash, beginning of period | 1,185,468 | 948,812 | 459,812 |
Cash, cash equivalents and restricted cash, end of period | 939,944 | 1,185,468 | 948,812 |
Supplemental disclosure of non-cash investing and financing activities | |||
Property and equipment purchases in accounts payable and accrued expenses | 8,348 | 3,412 | 1,811 |
Equity issuance costs in accounts payable and accrued expenses | $ 334 | $ 9,590 | $ 295 |
Consolidated Statements of Ca_2
Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Reconciliation to amounts within the consolidated balance sheets | ||||
Cash and cash equivalents | $ 923,031 | $ 1,168,620 | $ 943,771 | |
Restricted Cash | 16,913 | 16,848 | 5,041 | |
Total | $ 939,944 | $ 1,185,468 | $ 948,812 | $ 459,812 |
Organization and Operations
Organization and Operations | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Operations | 1. Organization and Operations CRISPR Therapeutics AG (“CRISPR” or the “Company”) was incorporated on October 31, 2013 in Basel, Switzerland. The Company was established to translate CRISPR/Cas9, a genome editing technology, into transformative gene-based medicines for the treatment of serious human diseases. The foundational intellectual property underlying the Company’s operations was licensed to the Company in April 2014. The Company devotes substantially all of its efforts to product research and development activities, initial market development and raising capital. The Company’s principal offices are in Zug, Switzerland and operations are in Cambridge, Massachusetts. The Company is subject to risks common to companies in the biotechnology industry, including but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any drug product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and ability to transition from pilot-scale manufacturing to large-scale production of products. The Company had an accumulated deficit of $ 195.9 million as of December 31, 2021 and has financed its operations to date from a series of preferred shares and convertible loan issuances, proceeds obtained from its initial public offering, subsequent public offerings of its common shares, as well as upfront fees and milestones received under its collaboration and joint venture arrangements. The Company will require substantial additional capital to fund its research and development and ongoing operating expenses. As of December 31, 2021, the Company had cash, cash equivalents and marketable securities of $ 2,379.1 million. While the Company was in a net income position in the current and certain previous years due to upfronts associated with the Company's collaborations with Vertex Pharmaceuticals Incorporated and certain of its subsidiaries, or Vertex, the Company has a history of recurring losses and expects to continue to incur losses for the foreseeable future. The Company expects its cash and cash equivalents will be sufficient to fund current planned operations for at least the next twenty-four months. The full extent of the impact of the coronavirus pandemic on the Company’s business, operations and financial results will depend on numerous evolving factors that we may not be able to accurately predict. See Item 1A: "Risk Factors" section set forth in this Annual Report on Form 10-K for additional details. At this stage, the impact on the Company’s results has not been significant. |
Summary of Significant Accounti
Summary of Significant Accounting Policies and Basis of Presentation | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies and Basis of Presentation | 2. Summary of Significant Accounting Policies and Basis of Presentation Basis of Presentation and Use of Estimates The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America, or GAAP, and include the accounts of the Company and its wholly-owned subsidiaries as of December 31, 2021. All intercompany accounts and transactions have been eliminated. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASUs, of the Financial Accounting Standards Board. Prior to December 13, 2019, the Company accounted for its 50 % investment in Casebia Therapeutics, Limited Liability Partnership, or Casebia, under the equity method. As described in Note 9, on December 13, 2019, Casebia became a wholly-owned subsidiary and, as a result, the Company consolidated Casebia’s financial results from that date forward. The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of research and development expenses during the period. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Segment Information The Company and the Company’s chief operating decision maker, namely, the chief executive officer, view the Company’s operations and manage its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Foreign Currency Translation and Transactions The majority of the Company’s operations occur in entities that have the U.S. dollar as their functional currency. Non-U.S. dollar denominated functional currency subsidiaries have assets and liabilities translated into U.S. dollars at rates of exchange in effect at the end of the year. Revenue and expense amounts are translated using the average exchange rates for the period. Net unrealized gains and losses resulting from foreign currency translation are included in “Accumulated other comprehensive income (loss).” Net foreign currency exchange transaction gains or losses are included in “Other income (expense), net” on the Company’s consolidated statement of operations, the impact of which is not significant. Cash and Cash Equivalents The Company considers all highly liquid investments with maturities of three months or less from the purchase date to be cash equivalents. As of December 31, 2021 and 2020 , the Company had $ 923.0 million and $ 1,168.6 million in cash and cash equivalents, respectively. Restricted Cash As of December 31, 2021 , the Company had $ 16.9 million in restricted cash representing letters of credit securing the Company’s obligations under certain leased facilities, as well as certain credit card arrangements, which was unchanged from December 31, 2020. The letters of credit are secured by cash held in a restricted depository account and recorded in restricted cash in the accompanying consolidated balance sheet as of December 31, 2021 . Marketable Securities As of December 31, 2021 and 2020 , the Company had $ 1,456.1 million and $ 521.7 million, respectively in marketable securities. The Company’s investment strategy is focused on capital preservation. The Company invests in instruments that meet the credit quality standards outlined in the Company’s investment policy. The Company classifies marketable securities with a remaining maturity, when purchased, of greater than three months as available-for-sale. Marketable securities are classified as current assets on the consolidated balance sheets if the marketable securities are available to be converted into cash to fund current operations. As a result, the Company classified all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, 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 interest expense over the period of the earliest call date, and any discount arising at purchase is accreted to interest income over the life of the instrument. Realized gains and losses on debt securities are determined using the specific identification method and are included in other income (expense), net. Effective January 1, 2020 , the Company adopted ASU 2016-13, Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Statements , or ASC 326. As the Company did not hold available-for-sale debt securities upon adoption, no related transition provisions were applicable to the Company upon adoption. The Company assesses its available-for-sale debt securities under the available-for-sale debt security impairment model in ASC 326 as of each reporting date in order to determine if a portion of any decline in fair value below carrying value recognized on its available-for-sale debt securities is the result of a credit loss. The Company records credit losses in the consolidated statements of operations and comprehensive loss as credit loss expense within other expense, net, which is limited to the difference between the fair value and the amortized cost of the security. To date, the Company has not recorded any credit losses on its available-for-sale debt securities. Other Receivables Amounts due from collaboration partners where an arrangement is accounted for under ASC 808, Collaborative Arrangements , or ASC 808, are considered other receivables and are included within prepaid and other current assets in the consolidated balance sheet. Other receivables consisted of $ 8.4 million and $ 10.7 million as of December 31, 2021 and 2020 , respectively and are due from Vertex. Other receivables are recorded at invoiced amounts due under the Vertex collaboration agreement, as described further in Note 9. Vertex is a creditworthy entity that maintains an ongoing relationship with the Company and as such, the Company does not have an allowance for estimated credit losses recorded related to these other receivables. Concentrations of Credit Risk and Off-balance Sheet Risk Financial instruments that potentially subject the Company to concentrations of credit risk are primarily cash, cash equivalents and marketable securities. The Company’s cash is held in accounts with financial institutions that management believes are creditworthy. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. The Company has no financial instruments with off-balance sheet risk of loss. 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 — Quoted prices in active markets that are accessible at the market date for identical unrestricted assets or liabilities. Level 2 — Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs for which all significant inputs are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. 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 Measurement ). The carrying amount of accounts receivable, other receivables, accounts payable and accrued expenses as reported on the consolidated balance sheets as of December 31, 2021 and 2020 , approximate fair value, due to the short-term duration of these instruments. Property and Equipment Property and equipment are recorded at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Upon disposal, the related cost and accumulated depreciation is removed from the accounts and any resulting gain or loss is included in the results of operations. Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, which are as follows: Asset Estimated useful life Computer equipment 3 years Furniture, fixtures and other 5 years Laboratory equipment 5 years Leasehold improvements Shorter of useful life or remaining lease term 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. Revenue Recognition The Company records revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers , or ASC 606. ASC 606 applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases and collaboration arrangements. To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: 1) Identify the contract with the customer A contract with a customer exists when (i) the Company enters into an enforceable contract with a customer that defines each party’s rights regarding the goods or services to be transferred and identifies the related payment terms, (ii) the contract has commercial substance and (iii) the Company determines that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration. 2) Identify the performance obligations in the contract Performance obligations promised in a contract are identified based on the goods and services that will be transferred to the customer that are both capable of being distinct, whereby the customer can benefit from the good or service either on its own or together with other available resources, and are distinct in the context of the contract, whereby the transfer of the good or service is separately identifiable from other promises in the contract. To the extent a contract includes multiple promised goods and services, the Company must apply judgment to determine whether promised goods and services are capable of being distinct and distinct in the context of the contract. If these criteria are not met, the promised goods and services are accounted for as a combined performance obligation. 3) Determine the transaction price The transaction price is determined based on the consideration to which the Company will be entitled in exchange for transferring goods and services to the customer. To the extent the transaction price includes variable consideration such as research, development, regulatory and commercial milestones, the Company determines if it is probable that it will receive such amounts and there is no risk of a significant revenue reversal. When the Company cannot conclude that receipt of such amounts is probable, the Company constrains the related variable consideration resulting in its exclusion from transaction consideration. In determining the portion of the transaction consideration to be constrained, the Company considers the probability and uncertainty that the related research, developmental, regulatory and commercial milestones will be achieved given the nature of research and clinical development and the stage of the underlying programs. This assessment is performed at each reporting period. In making this evaluation, the Company considers both internal and external information available, including information from industry publications and other relevant factors. Changes to the constraint of variable consideration can have a material effect on the amount of revenue recognized in the period. 4) Allocate the transaction consideration to performance obligations in the contract If the contract contains a single performance obligation, the entire transaction consideration is allocated to the single performance obligation. Contracts that contain multiple performance obligations require an allocation of the transaction consideration to each performance obligation on a relative standalone selling price basis unless the transaction consideration is variable and meets the criteria to be allocated entirely to a performance obligation or to a distinct service that forms part of a single performance obligation. The consideration to be received is allocated among the separate performance obligations based on relative standalone selling prices. In determining these estimated standalone selling prices, the Company makes a number of significant judgements including, for licenses, management’s assumptions regarding probability weighted projected discounted cash flows for each of the collaboration development programs. The estimated standalone selling prices are sensitive to changes in assumptions, such as probabilities of scientific success, discount rate and certain assumptions that form the basis of forecasted cash flows. In developing these assumptions, management considers both internal and external information available, including information from other guideline companies within the same industry and other relevant factors. Changes to these assumptions can have a material effect on the allocation of the transaction consideration to performance obligations, as well as the amount and timing of revenue recognized. 5) Recognize revenue when or as the Company satisfies a performance obligation The Company satisfies performance obligations over time or at a point in time, depending on the nature of the performance obligation. Revenue is recognized over time if the customer simultaneously receives and consumes the benefits provided by the entity’s performance, the entity’s performance creates or enhances an asset that the customer controls as the asset is created or enhanced, or the entity’s performance does not create an asset with an alternative use to the entity and the entity has an enforceable right to payment for performance completed to date. If the entity does not satisfy a performance obligation over time, the related performance obligation is satisfied at a point in time by transferring the control of a promised good or service to a customer. Contract Balances The Company recognizes a contract asset when the Company transfers goods or services to a customer before the customer pays consideration or before payment is due, excluding any amounts presented as an account or other receivable. A contract asset is an entity’s right to consideration in exchange for goods or services that the entity has transferred to a customer. The contract liabilities, or deferred revenue, primarily relate to contracts where we have received payment, but we have not yet satisfied the related performance obligations. Contract assets are not significant as of December 31, 2021 and 2020. Contract liabilities recorded as deferred revenue as of December 31, 2021 and 2020 are $ 12.3 million and $ 12.2 million, respectively. The change in contract assets and contract liabilities recorded as deferred revenue is related to the collaboration agreement with Vertex described in Note 9. Collaboration Arrangements The Company records the elements of its collaboration agreements that represent joint operating activities in accordance with ASC 808, Collaborative arrangements , or ASC 808. Accordingly, the elements of the collaboration agreements that represent activities in which both parties are active participants and to which both parties are exposed to the significant risks and rewards that are dependent on the commercial success of the activities, are recorded as collaborative arrangements. The Company evaluates the proper presentation of the commercial activities and the profit and loss sharing associated with the collaboration agreements. ASC 808 states that when payments between parties in a collaborative arrangement are not within the scope of other authoritative accounting literature, the income statement classification should be based on the nature of the arrangement, the nature of its business operations and the contractual terms of the arrangement. To the extent that these payments are not within the scope of other authoritative accounting literature, the income statement classification for the payments shall be based on an analogy to authoritative accounting literature or if there is no appropriate analogy, a reasonable, rational and consistently applied accounting policy election. 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 and other related costs. Research and development costs, including up-front fees and milestones paid to collaborators, are also expensed 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 a reduction to research and development expense in the period the services are provided. Leases The Company accounts for its leases in accordance with ASC 842, Leases , or ASC 842. 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 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. 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 of renewal. 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. Equity Based Compensation Expense The Company’s share-based compensation programs grant awards that have included stock options, restricted stock units and restricted stock awards. Grants are awarded to employees and non-employees, including directors. The Company accounts for its stock-based compensation awards in accordance with ASC Topic 718, Compensation—Stock Compensation , or ASC 718. ASC 718 requires all stock-based payments to employees and non-employee directors, including grants of employee stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive income (loss) based on their fair values. The Company uses the Black-Scholes option pricing model to determine the fair value of options granted. The Company accounts for forfeitures as they occur instead of estimating forfeitures at the time of grant and revising those estimates in subsequent periods if actual forfeitures differ from its estimates. Stock-based compensation expense recognized in the financial statements is based on awards for which performance or service conditions are expected to be satisfied. The Company’s stock-based awards are subject to service or performance-based vesting conditions. Compensation expense related to awards to employees, directors and non-employees 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 to employees 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 expenses restricted stock unit awards to employees based on the fair value of the award on a straight-line basis over the associated service period of the award. The Company estimates the fair value of its option awards to employees, directors and non-employees 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. Patent Costs Costs to secure and prosecute patent applications and other legal costs related to the protection of the Company’s intellectual property are expensed as incurred and are classified as general and administrative expenses in the Company’s consolidated statements of operations. Income Taxes Income taxes are recorded in accordance with ASC Topic 740, Income Taxes , or ASC 740, which provides for deferred taxes using an asset and liability approach. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and tax reporting basis of assets and liabilities and are measured using enacted tax rates and laws that are expected to be in effect when 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 has evaluated available evidence and concluded that the Company may not realize all the benefit of its deferred tax assets; therefore, a valuation allowance has been established for the amount of the deferred tax assets that the Company does not believe is more likely than not to 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. As of December 31, 2021 and 2020 , the Company does no t have any significant uncertain tax positions. The Company’s practice is to recognize interest and/or penalties related to income tax matters in income tax expense. See Note 14 for further details. Comprehensive Income (Loss) Comprehensive income (loss) consists of net income or loss and other comprehensive income (loss). Other comprehensive income (loss) consists of foreign currency translation adjustments and unrealized losses on marketable securities. Variable Interest Entities The Company reviews each legal entity formed by parties related to the Company to determine whether or not the Company has a variable interest in the entity and whether or not the entity would meet the definition of a variable interest entity, or VIE, in accordance with ASC Topic 810, Consolidation , or ASC 810. If the entity is a VIE, the Company assesses whether or not the Company is the primary beneficiary of that VIE based on a number of factors, including (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to any contractual agreements and (iii) which party has the obligation to absorb losses or the right to receive benefits from the VIE. If the Company determines it is the primary beneficiary of a VIE, the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements at the time that determination is made. The Company evaluates whether it continues to be the primary beneficiary of any consolidated VIEs on a quarterly basis. If the Company were to determine that it is no longer the primary beneficiary of a consolidated VIE, or no longer has a variable interest in the VIE, it would deconsolidate the VIE in the period that the determination is made. If the Company determines it is the primary beneficiary of a VIE that meets the definition of a business, the Company measures the assets, liabilities and noncontrolling interests of the newly consolidated entity at fair value in accordance with ASC Topic 805, Business Combinations , or ASC 805, at the date the reporting entity first becomes the primary beneficiary. In February 2016, Casebia, was formed in the United Kingdom. In March 2016, upon consummation of the joint venture (“JV”), Bayer Healthcare LLC and certain of its affiliates (“Bayer”) and the Company each received a 50 % equity interest in the entity in exchange for their contributions to the entity. The Company determined that Casebia was considered a VIE and concluded that it is not the primary beneficiary of the VIE. As such, the Company has not historically consolidated Casebia’s results into the consolidated financial statements. As described in Note 9, on December 13, 2019, Casebia became a fully-owned subsidiary and, as a result, the Company consolidated Casebia’s financial results accordingly from that point forward. Net Income (Loss) Per Share Attributable to Common Shareholders Basic net income (loss) per share is calculated by dividing net income (loss) attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net income (loss) per share is calculated by dividing the net income (loss) attributable to common shareholders by the weighted-average number of common equivalent shares outstanding for the period, including any dilutive effect from outstanding stock options and restricted stock units using the treasury stock method. See Note 12 for further details. New Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies that the Company adopts as of the specified effective date. Unless otherwise discussed below, the Company does not believe that the adoption of recently issued standards have or may have a material impact on its consolidated financial statements and disclosures. 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, or 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 Company adopted ASU 2019-12 on January 1, 2021 . The adoption of ASU 2019-12 did not have a material impact on the Company’s financial position or results of operations upon adoption. |
Marketable Securities
Marketable Securities | 12 Months Ended |
Dec. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | 3. Marketable Securities A summary of the Company’s cash equivalents and marketable securities as of December 31, 2021 and 2020 , which are recorded at fair value (and excludes $ 405.6 million and $ 395.1 million of cash at December 31, 2021 and 2020, respectively) is shown below (in thousands): December 31, 2021 Amortized Gross Gross Fair Value Cash equivalents: Money market funds $ 507,386 $ — $ — $ 507,386 Corporate debt securities — — — — Certificates of deposit — — — — Commercial paper 9,997 — ( 1 ) 9,996 Total cash equivalents 517,383 — ( 1 ) 517,382 Marketable securities: U.S. Treasury securities 16,238 6 ( 52 ) 16,192 Corporate debt securities 1,173,659 10 ( 4,903 ) 1,168,766 Certificates of deposit 45,164 — — 45,164 Government-sponsored enterprise securities 13,334 — ( 77 ) 13,257 Commercial paper 212,805 — ( 86 ) 212,719 Total marketable securities 1,461,200 16 ( 5,118 ) 1,456,098 Total cash equivalents and marketable securities $ 1,978,583 $ 16 $ ( 5,119 ) $ 1,973,480 December 31, 2020 Amortized Gross Gross Fair Value Cash equivalents: Money market funds $ 742,958 $ — $ — $ 742,958 Corporate debt securities 2,526 1 ( 24 ) 2,503 Certificates of deposit 12,527 — — 12,527 Commercial paper 15,549 — — 15,549 Total cash equivalents 773,560 1 ( 24 ) 773,537 Marketable securities: U.S. Treasury securities 47,976 3 — 47,979 Corporate debt securities 324,569 43 ( 156 ) 324,456 Certificates of deposit 25,162 — — 25,162 Government-sponsored enterprise securities 33,738 5 ( 2 ) 33,741 Commercial paper 90,375 — — 90,375 Total marketable securities 521,820 51 ( 158 ) 521,713 Total cash equivalents and marketable securities $ 1,295,380 $ 52 $ ( 182 ) $ 1,295,250 As of December 31, 2021 and 2020 , the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $ 1,311.6 million and $ 280.3 million, respectively. As of December 31, 2021 , the aggregate fair value of marketable securities that were in an unrealized loss position for more than twelve months was $ 4.6 million. As of December 31, 2020, no marketable securities were in an unrealized loss position for more than twelve months. The Company has recorded a net unrealized loss of $ 5.0 million and $ 0.1 million, respectively, during the years ended December 31, 2021 and 2020 related to its marketable securities, which is included in comprehensive income (loss) on the consolidated statements of operations and comprehensive income (loss). No net unrealized loss was recorded related to its marketable securities for the year ended December 31, 2019. The Company determined that there was no material credit risk of the above investments as of December 31, 2021. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the years ended December 31, 2021 and 2020. |
Fair Value Measurement
Fair Value Measurement | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurement | 4. Fair Value Measurement The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of December 31, 2021 and 2020 (in thousands): Fair Value Measurements at December 31, 2021 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 405,648 $ 405,648 $ — $ — Money market funds 507,386 507,386 — — Corporate debt securities — — — — Certificates of deposit — — — — Commercial paper 9,997 — 9,997 — Marketable securities: U.S. Treasury securities 16,192 — 16,192 — Corporate debt securities 1,168,766 — 1,168,766 — Certificates of deposit 45,164 — 45,164 — Government-sponsored enterprise securities 13,257 — 13,257 — Commercial paper 212,719 — 212,719 — Other non-current assets 2,212 — — 2,212 Total $ 2,381,341 $ 913,034 $ 1,466,095 $ 2,212 Fair Value Measurements at December 31, 2020 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 395,083 $ 395,083 $ — $ — Money market funds 742,958 742,958 — — Corporate debt securities 2,503 — 2,503 — Certificates of deposit 12,527 — 12,527 — Commercial paper 15,549 — 15,549 — Marketable securities: U.S. Treasury securities 47,979 — 47,979 — Corporate debt securities 324,456 — 324,456 — Certificates of deposit 25,162 — 25,162 — Government-sponsored enterprise securities 33,741 — 33,741 — Commercial paper 90,375 — 90,375 — Other non-current assets 600 — — 600 Total $ 1,690,933 $ 1,138,041 $ 552,292 $ 600 Marketable securities classified as Level 2 within the valuation hierarchy generally consist of 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. The Company holds equity securities classified as Level 3 which are not material to the Company’s financial position. |
Property and Equipment, net
Property and Equipment, net | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 5. Property and Equipment, net Property and equipment, net, consists of the following (in thousands): As of December 31, 2021 2020 Computer equipment $ 1,757 $ 727 Furniture, fixtures, and other 4,371 3,416 Laboratory equipment 30,123 25,353 Leasehold improvements 86,735 25,473 Construction work in process 52,396 8,366 Total property and equipment, gross 175,382 63,335 Accumulated Depreciation ( 37,807 ) ( 21,175 ) Total property and equipment, net $ 137,575 $ 42,160 Depreciation expense for the year ended December 31, 2021, 2020 and 2019 was $ 17.9 million, $ 9.1 million, and $ 4.7 million, respectively. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses consist of the following (in thousands): As of December 31, 2021 2020 Payroll and employee-related costs $ 23,661 $ 22,402 Research costs 47,986 21,684 Licensing fees 138 1,401 Professional fees 4,720 1,670 Intellectual property costs 6,120 3,625 Accrued property and equipment 7,113 2,835 Other 1,265 165 Total $ 91,003 $ 53,782 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
Leases | 7. Leases In June 2015, the Company entered into a lease agreement for the lease of research facility space in Cambridge, Massachusetts, with a commencement date of November 15, 2015 , or the 2015 Lease. The lease was subsequently amended in both 2017 and 2020 and now expires in 2022 with no further option to extend . The 2015 Lease contains escalating rent clauses, which require higher rent payments in future years. In May 2016, the Company entered into a sublease agreement for its primary office and research facility in Cambridge, Massachusetts, with a commencement date of December 23, 2016 , or the 2016 Sublease. The sublease was subsequently amended in 2021 and now expires in 2022 . The right-of-use assets and right-of-use liabilities were adjusted in the second quarter of 2021, accordingly. The 2016 Sublease contains escalating rent clauses, which require higher rent payments in future years. In May 2019, the Company entered into a lease agreement for office facility space in Cambridge, Massachusetts, with a commencement date of June 1, 2019 , or the 2019 Lease. The lease expires in November 2026 , and the Company has an option to extend the term of the lease for an additional five-year period based on certain conditions within the Company’s control. The 2019 Lease contains escalating rent clauses which require higher rent payments in future years. The right-of-use asset and corresponding lease liability does not include the additional five-year period under the renewal option as the Company is not reasonably certain to exercise that option. In December 2019, Casebia became a wholly-owned subsidiary of the Company. In connection therewith, Casebia assigned its sublease for an office and research facility in Cambridge, Massachusetts, or the 2019 Sublease, to the Company. The sublease was subsequently amended in 2021 and now expires in 2022 with no further option to extend . The right-of-use assets and right-of-use liabilities were adjusted in the second quarter of 2021, accordingly. The 2019 Sublease contains escalating rent clauses which require higher rent payments in future years. In May 2020, the Company entered into a lease agreement for a cell therapy manufacturing facility in Framingham, Massachusetts, or the Framingham Lease, for clinical and commercial production of the Company’s investigational cell therapy product candidates. The Framingham Lease expires in March 2036 and the Company has an option to extend the term of the lease for two additional seven-year periods. The right-of-use asset and corresponding lease liability does not include the additional seven-year periods under the renewal option as the Company is not reasonably certain to exercise that option. In July 2020, the Company entered into a lease agreement for an office and laboratory facility in Boston, Massachusetts, or the 2020 Lease. The 2020 Lease commenced in the second quarter of 2021, and at lease commencement, the Company recorded a right-of-use asset of $ 149.8 million and a corresponding operating lease liability of $ 147.9 million. Tenant incentives of $ 49.2 million were recorded as a reduction to the operating lease asset and liability at lease commencement. The right-of-use asset and corresponding lease liability does not include the additional five-year periods under the renewal option as the Company is not reasonably certain to exercise that option. The lease expires in March 2034 and the Company has an option to extend the term of the lease for two additional five-year periods . In addition, the Company rents certain office space in Zug, Switzerland, on a short-term basis for which a right-of-use asset and liability are not recorded, in accordance with the practical expedient elected. The Company has embedded leases in certain research and license agreements for which the Company has recorded a right of use asset and liability. These arrangements are not significant in comparison to the Company’s total operating lease assets and liabilities. In addition, the Company has identified certain short-term leases embedded within its manufacturing contracts which are not recorded on the Company’s balance sheet in accordance with the practical expedient elected. The Company identified and assessed the following estimates in recognizing the right-of-use asset and corresponding liability: • Expected lease term : The expected lease term for those leases commencing prior to January 1, 2019 did not change with the adoption of ASC 842. The expected lease term for leases commencing after the adoption of ASC 842 includes noncancelable lease periods and, when applicable, periods covered by an option to extend the lease if the Company is reasonably certain to exercise that option, as well as periods covered by an option to terminate the lease if the Company is reasonably certain not to exercise that option. • Incremental borrowing rate : As the discount rates in the Company’s leases are not implicit, the Company estimated the incremental borrowing rate based on the rate of interest the Company would have to pay to borrow a similar amount on a collateralized basis over a similar term. The following table summarizes the lease assets and liabilities as of December 31, 2021 and 2020 (in thousands): As of December 31, 2021 2020 Assets Operating lease assets $ 174,460 $ 50,865 Total lease assets 174,460 50,865 Liabilities Current Operating lease liabilities 12,158 11,362 Non-current Operating lease liabilities, net of current portion 212,872 50,067 Total lease liabilities $ 225,030 $ 61,429 The following table summarizes operating lease costs included in research and development and general and administrative expense, as well as sublease income for the twelve months ended December 31, 2021, 2020 and 2019 (in thousands): Years Ended December 31, 2021 2020 2019 Operating lease costs $ 22,520 $ 14,342 $ 8,067 Short-term lease costs 11,087 7,339 4,554 Variable lease costs 8,402 6,368 4,282 Sublease income ( 5,253 ) ( 587 ) ( 525 ) Net lease cost $ 36,756 $ 27,462 $ 16,378 The following table summarizes the maturity of undiscounted payments due under lease liabilities and the present value of those liabilities as of December 31, 2021 (in thousands): Total 2022 18,256 2023 26,633 2024 26,137 2025 26,347 2026 26,844 Thereafter 227,528 Total $ 351,745 Present value adjustment ( 126,715 ) Present value of lease liabilities $ 225,030 The following table summarizes the lease term (in years) and discount rate for operating leases as of December 31, 2021 and 2020: As of December 31, 2021 2020 Weighted-average remaining lease term 12.4 9.1 Weighted-average discount rate 5.9 % 9.3 % The following table summarizes the cash paid for amounts included in the measurement of lease liabilities for the year ended December 31, 2021 and 2020 (in thousands): Years Ended December 31, 2021 2020 2019 Cash paid for amounts included in measurement of lease liabilities: Operating cash flows used in operating leases $ ( 19,753 ) $ ( 13,161 ) $ ( 8,420 ) Operating lease non-cash items: Right-of-use assets (decreased) increased through lease modifications and reassessments ( 14,230 ) 3,169 826 Right-of-use assets obtained in exchange for operating lease liabilities 152,486 13,956 18,088 Leasehold improvements paid directly by landlord 30,500 — — |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 8. Commitments and Contingencies Intellectual Property Agreements Charpentier License Agreements In April 2014, the Company entered into certain technology license agreements with Dr. Emmanuelle Charpentier pursuant to which the Company licensed certain intellectual property rights under joint ownership from Dr. Charpentier to develop and commercialize products for the treatment or prevention of human diseases. In connection therewith, Dr. Charpentier is entitled to receive nominal clinical milestone payments, low single digit percentage of sublicensing payments received under any sublicense agreement with a third party, and low single-digit percentage royalties based on annual net sales of licensed products and services by the Company and its affiliates and sublicensees. Patent Assignment Agreement In November 2014, the Company entered into a patent assignment agreement with Dr. Charpentier, Dr. Ines Fonfara, and Vienna (collectively, the “Assignors”), pursuant to which the Company was assigned all rights, title and interest in and to certain patent rights claimed in the U.S. Patent Application No.61/905,835. As a result, the Assignors are entitled to receive certain low single digit clinical milestone payments and low single digit royalties based on annual net sales of licensed products and licensed services by the Company, its affiliates and sublicensees. During the years ended December 31, 2021, 2020 and 2019, the Company paid an immaterial amount of fees to Dr. Charpentier under the Charpentier License Agreements and the Assignors under the Patent Assignment Agreement, which were recorded as research and development expense. Research, Manufacturing and License Agreements The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene-editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services. In association with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales. Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $ 395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit percentage royalties on future net sales related to a specified target under an amendment to the 2015 Collaboration Agreement defined in Note 9 below. In addition, Vertex has the option to conduct research at their own cost in certain defined areas that, if beneficial to the CTX001 program and ultimately achieves regulatory approval, then the Company could owe Vertex certain milestone payments aggregating to high eight digits, subject to certain limitations on the profitability of the CTX001 program. Refer to Note 9 for further discussion on the Company’s arrangements with Vertex. Other Matters On December 15, 2016, the Company entered into a Consent to Assignments, Licensing and Common Ownership and Invention Management Agreement (the “Invention Management Agreement”) with UC, Vienna, Dr. Charpentier, Intellia Therapeutics, Inc., Caribou Biosciences, Inc., ERS Genomics Ltd. and one of the Company’s subsidiaries. Under the Invention Management Agreement, the Company is obligated to share costs related to patent maintenance, defense and prosecution. For the years ended December 31, 2021, 2020 and 2019 , the Company incurred $ 5.8 million, $ 4.5 million and $ 2.9 million, respectively, in shared costs. The Company recorded accrued legal costs from the cost sharing of $ 4.0 million and $ 2.5 million as of December 31, 2021 and 2020, respectively. The Company is unable to predict the outcome of these matters and is unable to make a meaningful estimate of the amount or range of loss, if any, that could result from an unfavorable outcome. Litigation In the ordinary course of business, the Company is from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving the Company’s intellectual property estate including certain in-licensed intellectual property. T he outcome of any of the foregoing, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected. |
Significant Contracts
Significant Contracts | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Significant Contracts | 9. Significant Contracts Agreements with Vertex Pharmaceuticals Incorporated and certain of its subsidiaries Summary On October 26, 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene-editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. On December 12, 2017, the Company and Vertex entered into Amendment No. 1 to the 2015 Collaboration Agreement, or Amendment No. 1, and the Joint Development Agreement, or the JDA. Amendment No. 1, among other things, modified certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA and clarified how many options are exercised (or deemed exercised) in connection with certain targets specified under the 2015 Collaboration Agreement. Amendment No. 1 also amended other provisions of the 2015 Collaboration Agreement, including the expiration terms. In connection with the 2015 Collaboration Agreement, Vertex made a nonrefundable upfront payment of $ 75.0 million. Under the 2015 Collaboration Agreement, Vertex agreed to fund the discovery activities conducted pursuant to the agreement while retaining options to co-exclusive and exclusive licenses. In December 2017, upon execution of the JDA and Amendment No. 1, Vertex exercised its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets. As such, for potential hemoglobinopathy treatments, including treatments for sickle cell disease, the Company and Vertex agreed to share equally all research and development costs and worldwide revenues. In connection with the JDA, the Company received a $ 7.0 million up-front payment from Vertex and subsequently received a one-time low seven-digit milestone payment upon the dosing of the second patient in a clinical trial with the initial product candidate. In addition, upon execution of the JDA and Amendment No. 1, it was clarified that Vertex may elect to license up to four remaining targets, for which it will lead global development and commercialization activities, and the Company received the right to receive up to $ 420.0 million in development, regulatory and commercial milestones and royalties on net product sales for each of the targets (inclusive of $ 10 million due upon exercise of each exclusive option). In June 2019, the Company and Vertex entered into a series of agreements, which closed on July 23, 2019, including a strategic collaboration and license agreement, or the 2019 Collaboration Agreement, for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and Myotonic Dystrophy Type 1, or DM1. Under the terms of the 2019 Collaboration Agreement, the Company received an upfront, nonrefundable payment of $ 175.0 million. In addition, the Company was initially eligible to receive potential aggregate payments of up to $ 825 .0 million based upon the successful achievement of specified research, development, regulatory and commercial milestones for the DMD and DM1 programs. The Company is also eligible to receive tiered royalties on future net sales on any products that may result from this collaboration. For the DMD program, Vertex is responsible for all research, development, manufacturing and commercialization activities and all related costs. For the DM1 program, the Company performed specified guide RNA research and Vertex is responsible for all other research, development, manufacturing and commercialization costs. Upon Investigational New Drug, or IND, application filing, the Company has the option to forego the DM1 milestones and royalties, and instead, co-develop and co-commercialize all DM1 products globally in exchange for payment of 50 % of research and development costs incurred by Vertex from the effective date of the agreement through IND filing. In connection with the execution of the 2019 Collaboration Agreement, the Company and Vertex entered into a second amendment to the 2015 Collaboration Agreement, or Amendment No. 2. Among other things, Amendment No. 2 modified certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the 2019 Collaboration Agreement and set forth the number and identity of the collaboration targets under the 2015 Collaboration Agreement. The Company and Vertex agreed that one of the four remaining options under the 2015 Collaboration Agreement, as amended, would not be exercised; instead, the Company reacquired the exclusive rights and agreed to conduct research and development activities for the specified target. Vertex will have the option to co-develop and co-commercialize the specified target upon IND filing in exchange for payment of 50 % of research and development costs incurred by the Company from the effective date of the agreement through IND filing. If Vertex does not exercise its option to co-develop and co-commercialize the specified target, Vertex is eligible to receive up to $ 395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit royalties on future net sales. In October 2019, Vertex exercised the remaining three options granted to it under the 2015 Collaboration Agreement to exclusively license the collaboration targets developed under the 2015 Collaboration Agreement, resulting in a payment of $ 30.0 million to the Company in the fourth quarter of 2019. In addition, the Company achieved the first milestone under the 2019 Collaboration Agreement in the first quarter of 2020 and, in connection therewith, received a payment of $ 25.0 million in April 2020. The Company achieved the second milestone under the 2019 Collaboration Agreement in the fourth quarter of 2021 and, in connection therewith, received a payment of $ 12.5 million in December 2021. As of December 31, 2021, the Company is eligible to receive remaining potential future milestones of $ 775.0 under the 2019 Collaboration Agreement. In April 2021, the Company and Vertex agreed to amend and restate the JDA and entered into an Amended and Restated Joint Development and Commercialization Agreement, or the “A&R Vertex JDCA,” pursuant to which the parties agreed to, among other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex shall lead and have all decision making (i.e., control) in relation to the CTX001 program prospectively; (b) adjust the allocation of net profits and net losses between the parties with respect to CTX001 only, which will be allocated 40 % to the Company and 60 % to Vertex, prospectively; and (c) exclusively license (subject to the Company’s reserved rights to conduct certain activities) certain intellectual property rights to Vertex relating to the specified product candidates and products (including CTX001) that may be researched, developed, manufactured and commercialized on a worldwide basis under such agreement. The transaction contemplated by the A&R Vertex JDCA closed in the second quarter of 2021. The Company will provide certain specified transition services to Vertex in connection with the agreement. In connection with the closing of the transaction contemplated by the A&R Vertex JDCA, the Company received a $ 900.0 million up-front payment from Vertex. Additionally, the Company is eligible to receive a one-time $ 200.0 million milestone payment upon receipt by Vertex of the first marketing approval of the initial product candidate from the U.S. Food and Drug Administration or the European Commission. With respect to CTX001 only, the net profits and net losses, as applicable, incurred under the A&R Vertex JDCA through July 1, 2021 in connection with the initial shared product (i.e., CTX001) were shared equally between the Company and Vertex, and beginning July 1, 2021, the net profits and net losses, as applicable, incurred under the A&R Vertex JDCA are allocated 40 % to the Company and 60 % to Vertex. Additionally, the A&R Vertex JDCA allows the Company to defer a portion of its share of costs under the arrangement if spending on the CTX001 program exceeds specified amounts. Any deferred amounts are only payable to Vertex as an offset against future profitability of the CTX001 program and the amounts payable are capped at a specified maximum amount per year. Accounting for the Vertex Agreements The 2015 Collaboration Agreement, Amendment No. 1, and JDA are collectively the “2015 Agreements” and the 2019 Collaboration Agreement and Amendment No. 2. are collectively the “2019 Agreements.” The 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, JDA, A&R Vertex JDCA and 2019 Collaboration Agreement are collectively the “Vertex Agreements.” The Vertex Agreements include components of a customer-vendor relationship as defined under ASC 606, collaborative arrangements as defined under ASC 808 and research and development costs as defined under ASC 730, Research and Development , or ASC 730. Additionally, the A&R Vertex JDCA allows the Company to defer a portion of its share of costs under the arrangement if spending on the CTX001 program exceeds specified amounts, which are only payable to Vertex as an offset against future profitability of the CTX001 program up to a maximum amount per year. Accounting Analysis Under ASC 606 Accounting for the A&R Vertex JDCA Identification of the Contract The A&R Vertex JDCA represented a contractual modification to the JDA. For accounting purposes, the A&R Vertex JDCA was treated as a separate contract. Identification of Performance Obligations The Company concluded the A&R Vertex JDCA contained a single material promise, an exclusive worldwide license granting Vertex an additional 10 % economic interest in the CTX001 program and the right to control development and commercialization of CTX001, or the “CTX001 Exclusive License.” The Company concluded the CTX001 Exclusive License was both capable of being distinct and distinct within the context of the A&R Vertex JDCA, and the CTX001 Exclusive License was sold at its estimated standalone selling price, or “ESSP.” As such, the CTX001 Exclusive License represented a separate performance obligation. Determination of Transaction Price The transaction price was comprised of the upfront payment of $ 900.0 million. The Company determined that all other possible variable consideration resulting from milestones and royalties discussed above was fully constrained at the time of the transaction. The Company will reevaluate the transaction price in each reporting period. Allocation of Transaction Price to Performance Obligations The selling price of the performance obligation was determined based on the Company’s ESSP. The Company developed the ESSP for the CTX001 Exclusive License with the objective of determining the price at which it would sell such an item if it were to be sold regularly on a standalone basis. The ESSP for the CTX001 Exclusive License was determined to be approximately $ 900.0 million. The ESSP was determined based on 10% of the probability and present value adjusted cash flows from projected worldwide net profit for CTX001 based on probability assessments, projections based on internal forecasts, industry data, and information from other guideline companies within the same industry and other relevant factors. As the Company determined the CTX001 Exclusive License was the only performance obligation, the entire transaction price was allocated to the CTX001 Exclusive License. The aforementioned ESSP reflects the level of risk and expected probability of success inherent in the nature of the associated research area. Recognition of Revenue The Company determined that the CTX001 Exclusive License represented functional intellectual property, as the intellectual property provides Vertex with the ability to perform a function or task in the form of research and development, manufacturing and commercialization. As such, the revenue related to the CTX001 Exclusive License was recognized at the point in time, which was upon transfer in the second quarter of 2021. Accounting for the 2019 Agreements Identification of the Contract The 2019 Agreements represented a contract modification to the 2015 Agreements. As a result, the 2019 Agreements and the 2015 Agreements are combined for accounting purposes and treated as a single arrangement. Identification of Performance Obligations The Company concluded the following material promises were both capable of being distinct and distinct within the context of the 2019 Agreements and represented separate performance obligations: (i) an exclusive license for worldwide rights for DMD gene editing products, or DMD License; (ii) an exclusive license for worldwide rights for DM1 gene editing products, or DM1 License; (iii) the performance of specified guide RNA research for DM1, or DM1 R&D Services; (iv) a material right representing the option to obtain a co-exclusive development and commercialization license for a specified target, or Specified Target Option; (v) three material rights representing the option for up to three exclusive licenses to develop and commercialize the collaboration targets, or Collaboration Target Options; and (vi) the waiving of Vertex’s material right associated with its option to a fourth exclusive license in connection with the Company’s reacquisition of exclusive rights to the specified target. Determination of Transaction Price The initial overall transaction price was determined based on the remaining transaction price from the 2015 Agreements, as well as the transaction price from the 2019 Agreements. The initial transaction price included variable consideration estimated using the most likely amount methodology. The Company determined the initial transaction price totaling $ 268.6 million was comprised of: (i) $ 57.8 million of pre-existing deferred revenue from the 2015 Agreements; (ii) non-cash consideration of $ 10.0 million related to the waiving of Vertex’s material right associated with its option to a fourth exclusive license in connection with the Company’s reacquisition of exclusive rights to the specified target; (iii) an upfront payment of $ 175.0 million; (iv) variable consideration of $ 25.0 million which represented the Company’s estimate related to a near-term research and development milestone for which the Company determined that it is not probable that a significant reversal of cumulative consideration will occur at the onset of the transaction; and (v) variable consideration of $ 0.8 million which represents the Company’s estimate of payments from Vertex for DM1 R&D Services. In December 2021, the Company achieved a milestone under the 2019 Collaboration Agreement. In connection therewith, the Company adjusted the transaction price to include $ 12.5 million in previously constrained variable consideration. The milestone was allocated to the performance obligations using the relative standalone selling price method, and revenue of $ 12.0 million was recognized for amounts allocated to fully satisfied performance obligations. Amounts allocated to unsatisfied performance obligations of $ 0.5 million were included in deferred revenue. The Company determined that all other possible variable consideration resulting from milestones and royalties discussed above was fully constrained as of December 31, 2021. The Company will re-evaluate the transaction price in each reporting period. Allocation of Transaction Price to Performance Obligations The selling price of each performance obligation was determined based on the Company’s ESSP. The Company developed the ESSP for all the performance obligations included in the Vertex Agreements with the objective of determining the price at which it would sell such an item if it were to be sold regularly on a standalone basis. The Company then allocated the transaction price to each performance obligation on a relative standalone selling price basis. The ESSP for the DMD License and DM1 License was determined to be $ 224.6 million and $ 76.2 million, respectively. The ESSP was determined based on probability and present value adjusted cash flows from projected worldwide net profit for each of the respective programs based on probability assessments, projections based on internal forecasts, industry data, and information from other guideline companies within the same industry and other relevant factors. On a relative basis, $ 151.1 million and $ 51.3 million of the initial transaction price was allocated to the DMD License and DM1 License, respectively. The ESSP for the Specified Target Option material right was determined to be $ 17.5 million, which was based on the incremental discount between (i) the value of the probability and present value adjusted cash flows from the equal sharing of projected worldwide net profit increased by the value of the option provided to Vertex less (ii) the expected exercise price at the time of option exercise. The present value adjusted cash flows also considered projections based on internal forecasts, industry data, and information from other guideline companies within the same industry and other relevant factors. On a relative basis $ 11.8 million of the initial transaction price was allocated to the Specified Target Option material right. The ESSP for each of the three Collaboration Target Option material rights was determined to be $ 25.0 million, $ 22.2 million and $ 22.2 million, respectively, which was determined based on the probability and present value adjusted cash flows from milestone payments owed for exclusive licenses, less the price paid to exercise each option. On a relative basis, $ 46.7 million of the initial transaction price was allocated to the Collaboration Target Option material rights. The aforementioned ESSPs reflect the level of risk and expected probability of success inherent in the nature of the associated research area. The ESSP for the waiving of Vertex’s material right associated with its option to a fourth exclusive license under the 2015 Agreements was determined to be $ 10.0 million, or the contractual value of the option. On a relative basis, $ 6.7 million of the initial transaction price was allocated to the waiving of Vertex’s material right associated with its option to a fourth exclusive license under the 2015 Agreements. The ESSP for the DM1 R&D Services was determined to be $ 1.7 million, which was based on estimates of the associated effort and cost of the services, adjusted for a reasonable profit margin that would be expected to be realized under similar contracts. On a relative basis, $ 1.1 million of the initial transaction price was allocated to the DM1 R&D Services. As discussed above, in December 2021, the Company adjusted the transaction price of the 2019 Collaboration Agreement to include $ 12.5 million of previously constrained variable consideration associated with a research milestone. The milestone was allocated to the performance obligations using the relative standalone selling price method based on the ESSP's described herein. As a result, revenue of $ 12.0 million was recognized for amounts allocated to fully satisfied performance obligations. Amounts allocated to unsatisfied performance obligations of $ 0.5 million were included in deferred revenue. Recognition of Revenue The Company determined that the DMD License and DM1 License represent functional intellectual property, as the intellectual property provides Vertex with the ability to perform a function or task in the form of research and development. As such, the revenue related to the licenses was recognized at the point in time in which they were delivered during the third quarter of 2019. The revenue allocated to the waiving of Vertex’s material right associated with its option to a fourth exclusive license in connection with Company’s reacquisition of exclusive rights to the specified target was recognized at the point in time in which the option was waived, on the effective date of the 2019 Agreements. The Company concluded that the Specified Target Option and Collaboration Target Options were considered material rights under the Vertex Agreements. Revenue related to the three Collaboration Target Options material right was recognized at the point in time in which Vertex exercised the Collaboration Target Options, which occurred in the fourth quarter of 2019. The Company recognized revenue related to the DM1 R&D Services over time as the services were rendered, which concluded in the fourth quarter of 2021. Revenue recognized in connection with the Vertex Agreements Revenue recognized under the Vertex Agreements for the year ended December 31, 2021 was $ 913.1 million and was comprised of (i) revenue related to the exclusive worldwide license for CTX001 of $ 900.0 million, (ii) revenue related to the second DM1 milestone under the 2019 Agreements of $ 12.0 million, and (iii) revenue recognized in connection with research and development services. Revenue recognized under the Vertex Agreements for the year ended December 31, 2020 was not material. Revenue recognized under the Vertex Agreements for the year ended December 31, 2019 was $ 289.1 million. The $ 289.1 million of revenue recognized for the year-ended December 31, 2019 was comprised of (i) revenue related to the DMD License and DM1 License of $ 202.4 million, which was recognized at the point in time in which the licenses were delivered, (ii) revenue related to the Collaboration Target Options material right of $ 76.7 million, which was recognized upon the exercise of the Collaboration Target Options by Vertex and is inclusive of the $ 30.0 million payment made by Vertex to exercise those options, (iii) revenue allocated to the waiving of Vertex’s material right associated with its option to a fourth exclusive license in connection with the Company’s reacquisition of exclusive rights to the specified target of $ 6.7 million, which was recognized at the point in time in which the option was waived, (iv) revenue recognized in connection with DM1 R&D Services of $ 0.1 million and (v) revenue recognized of $ 0.1 million related to both research and development services as well as the amortization of the non-exclusive research license under the 2015 Agreements. Additionally, the Company recognized revenue related to a one-time low seven-digit milestone payment upon the dosing of the second patient in a clinical trial with the initial product candidate in the third quarter of 2019. As of December 31, 2021 and 2020 there was $ 0.0 million and $ 0.4 million of current deferred revenue related to the collaboration with Vertex, respectively. As of December 31, 2021 and 2020 , there was $ 12.3 million and $ 11.8 million of non-current deferred revenue, respectively, related to the collaboration with Vertex. The transaction price allocated to the remaining performance obligations was $ 12.3 million. Future Milestones under the Vertex Agreements The Company has evaluated the milestones that may be received in connection with the Vertex Agreements. As discussed above, the Company is eligible to receive up to $ 410.0 million in additional development, regulatory and commercial milestones and royalties on net product sales for each of the three collaboration targets that Vertex licensed in the fourth quarter of 2019. Each milestone is payable only once per collaboration target, regardless of the number of products directed to such collaboration target that achieve the relevant milestone event. The Company is eligible to receive additional potential future payments of up to $ 775.0 million based upon the successful achievement of specified development, regulatory and commercial milestones for the DMD and DM1 programs. The Company is also eligible to receive tiered royalties on future net sales on any products that may result from this collaboration; however, the Company has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally. The Company is eligible to receive additional potential future payments of up to $ 200.0 million upon receipt by Vertex of the first marketing approval of the initial product candidate from the U.S. Food and Drug Administration or the European Commission. In addition, the Company has the option to conduct research at their own cost in certain defined areas that, if beneficial to the CTX001 program and CTX001 ultimately achieves regulatory approval in such areas, then the Company could be entitled to certain milestone payments aggregating to high eight digits from Vertex. Each of the remaining milestones are fully constrained as of December 31, 2021. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs. Accounting Analysis under ASC 808 In connection with the Vertex Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to CTX001 under the A&R JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing associated with research and development is included in research and development expense. Expenses related to services performed by the Company are classified as research and development expense. Payments received from Vertex for partial reimbursement of expenses are recorded as a reduction of research and development expense. During the years ended December 31, 2021, 2020 and 2019 , the Company recognized $ 111.6 million, $ 48.6 million, and $ 29.2 million of research and development expense related to the Vertex Agreements, respectively. Research and development expense for the years ended December 31, 2021, 2020 and 2019 is net of $ 47.4 million, $ 28.2 million, and $ 15.9 million of reimbursements from Vertex, respectively. Accounting Analysis under ASC 730 In connection with the 2019 Agreements, the Company and Vertex agreed that one of the four remaining options under the 2015 Agreements, as amended, would not be exercised; instead, the Company will conduct research and development activities for a specified target. Vertex will have the option to co-develop and co-commercialize the specified target upon IND filing in exchange for payment of 50 % of research and development costs incurred by the Company from the effective date of the agreement through IND filing. If Vertex does not exercise its option to do so within a specified time period, Vertex is eligible to receive up to $ 395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit royalties on future net sales. In connection therewith, the Company determined that in order for the Company to obtain the right to conduct research and development activities on the specified target, the Company had waived its right to receive an option exercise payment of $ 10.0 million from Vertex, which was included as non-cash consideration in the transaction price for the 2019 Agreements described above. The Company then subsequently reacquired its rights to the specified target by waiving payment owed by Vertex of $ 10.0 million for a license that represents in-process research and development and therefore, $ 10.0 million of non-cash consideration was fully expensed upon the execution of the 2019 Agreements. The Company also determined that research and development services through IND for the specified target and any payment of future development and commercialization milestones, as well as sales-based milestones and royalties for the specified target, would be accounted for as research and development costs under ASC 730 and expensed as incurred. In addition, the Company also determined that should the Company elect its option to co-develop and co-commercialize all DM1 products globally, it will record the option fee as research and development expense upon exercise. In connection with the A&R Vertex JDCA, Vertex has the option to conduct research at their own cost in certain defined areas that, if beneficial to the CTX001 program and CTX001 ultimately achieves regulatory approval in such areas, then the Company could owe Vertex certain milestone payments aggregating to high eight digits, subject to certain limitations on the profitability of the CTX001 program. Agreements with Bayer Healthcare LLC Summary On December 19, 2015 , the Company entered into an agreement with Bayer, to establish a joint venture to focus on the research and the development of new therapeutics to cure blood disorders, blindness and congenital heart disease. On February 12, 2016 , the Company and Bayer completed the formation of the joint venture entity, Casebia. Bayer and the Company each received a 50 % equity interest in the entity in exchange for their respective contributions to the entity. At that time, the Company also entered into a separate service agreement with Casebia, under which the Company agreed to provide compensated research and development services. Collectively, these agreements are referred to as the “2015 Casebia Agreements.” On December 13, 2019, the Company, Bayer and Casebia entered into a series of transactions by which, among other things, the Company acquired 100 % of the partnership interests in Casebia, or the Retirement Agreement, the Company and Bayer terminated their joint venture, or the Joint Venture Termination Agreement, and the Company and Bayer entered into a new option agreement, or the 2019 Option Agreement. Collectively, these agreements are referred to as the “2019 Casebia Agreements.” In connection with the Retirement Agreement, Casebia retired Bayer’s outstanding partnership interests in exchange for $ 22.0 million less certain estimated interim operating expenses of $ 6.0 million, and the Company acquired 100 % of the partnership interests in Casebia. In connection with entering into the Retirement Agreement, the Company, Bayer and Casebia entered into the Joint Venture Termination Agreement. In connection therewith, the Company and Bayer agreed to terminate the Joint Venture Agreement from December 2015. Under the Joint Venture Termination Agreement, Casebia-owned patents are now co-owned by the Company and Bayer, subject to certain exclusive licenses granted therein. Under the Joint Venture Termination Agreement, the Company and Bayer each retained rights to their respective contributed intellectual property. In connection with entering into the Retirement Agreement and the Joint Venture Termination Agreement, the Company and Bayer also entered into the 2019 Option Agreement, under which, among other things, the Company committed to invest a specified amount in certain research and development activities as described under “Accounting Analysis – Accounting for 2019 Casebia Agreements”. In addition, Bayer has an option (exercisable during a specified exercise period defined by future events, but in no event longer than 5 years after the effective date of the 2019 Option Agreement) to co-develop and co-commercialize two products for the diagnosis, treatment or prevention of certain autoimmune disorders, eye disorders, or hemophilia A disorders. In the event Bayer elects to co-develop and co-commercialize a product, the parties will negotiate and enter into a co-development and co-commercialization agreement, or the Co-Commercialization Agreement, for such product, and Bayer would be responsible for 50 % of the research and development costs incurred by the Company for such product going forward. Bayer would receive 50 % of all profits from sales |
Share Capital
Share Capital | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Share Capital | 10. Share Capital The Company had 145,364,335 and 115,172,786 authorized common shares as of December 31, 2021 and 2020 , respectively, with a par value of CHF 0.03 per share. Share Capital consisted of the following: As of December 31, Type of Share Capital Conditional Capital 2021 2020 Common shares Registered share capital 80,321,227 75,133,951 Common shares Authorized share capital 39,316,975 17,625,426 Common shares Conditional share capital - Bonds or similar debt instruments 4,919,700 4,919,700 Common shares Conditional share capital - Employee benefit plans 20,806,433 17,493,709 Total 145,364,335 115,172,786 Included in registered share capital are 5,038,262 shares registered, which are held by the Company and its subsidiaries and are reserved for future issuance for financings. Common Share Issuances Recent Public Offerings In November 2019, the Company sold 4.9 million common shares through an underwritten public offering (inclusive of shares sold pursuant to the exercise of the underwriters’ option to purchase additional shares) at a public offering price of $ 64.50 per share for aggregate net proceeds of $ 297.4 million, which were net of equity issuance costs of $ 17.8 million. Additional equity issuance costs of $ 3.0 million for stamp taxes were also paid in 2019. In July 2020, the Company sold 7.4 million common shares through an underwritten public offering (inclusive of shares sold pursuant to the exercise of the underwriters’ option to purchase additional shares) at a public offering price of $ 70.00 per share for aggregate net proceeds of $ 484.8 million, which were net of equity issuance costs and stamp tax of $ 32.5 million. At-the-Market Offerings In the first quarter of 2019, the Company began to issue and sell securities under an Open Market Sale Agreement SM entered into with Jefferies LLC, or Jefferies, in August 2018, under which the Company was able to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $ 125.0 million, or the 2018 ATM. During the year ended December 31, 2019, the Company issued and sold an aggregate of 2.8 million common shares at an average price of $ 44.38 per share for aggregate net proceeds of $ 120.6 million, which were net of equity issuance costs of $ 4.4 million. In addition, the Company paid approximately $ 0.9 million in stamp taxes during the year ended December 31, 2019 and accrued an additional $ 0.3 million for stamp taxes as of December 31, 2019. The Company paid the $ 0.3 million for stamp taxes in 2020. In August 2019, the Company entered into an Open Market Sale Agreement SM with Jefferies under which the Company was able to offer and sell, from time to time at its sole discretion through Jefferies, as its sales agent, its common shares, or the August 2019 Sales Agreement. In August 2019, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $ 200.0 million, or the 2019 ATM. During the year ended December 31, 2020, the Company issued and sold an aggregate of 2.2 million common shares under the 2019 ATM at an average price of $ 89.47 per share for aggregate proceeds of $ 195.5 million, which were net of equity issuance costs of $ 4.5 million. In December 2020, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $ 350.0 million, or the 2020 ATM. During the year ended December 31, 2020, the Company issued and sold an aggregate of 1.8 million common shares under the 2020 ATM at an average price of $ 169.57 per share for aggregate proceeds of $ 298.0 million, which were net of equity issuance costs of $ 4.5 million. Additional equity issuance costs for stamp taxes related to shares sold in 2020 related to the 2019 ATM and 2020 ATM were $ 4.9 million, of which $ 4.0 million was accrued as of December 31, 2020 and paid in 2021. In January 2021, the Company issued and sold under the 2020 ATM an aggregate of 0.3 million common shares at an average price of $ 162.46 per share with aggregate proceeds of $ 46.7 million, which were net of equity issuance costs of $ 0.7 million. An additional $ 0.5 million of stamp taxes related to this amount was paid in 2021. In January 2021, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $ 600.0 million, or the 2021 ATM. As of December 31, 2021 , the Company has issued and sold an aggregate of 1.1 million common shares under the 2021 ATM at an average price of $ 169.82 per share for aggregate proceeds of $ 177.8 million, which were net of equity issuance costs of $ 2.4 million. An additional $ 1.8 million of stamp taxes related to this amount was paid in 2021. The Common Shares have the following characteristics: Voting Rights The holders of common shares are entitled to one vote for each common share held at all meetings of shareholders. Dividends The holders of common shares are entitled to receive dividends, if and when resolved upon by the general meeting of shareholders based on a respective proposal by the Board of Directors and provided that the Company disposes of sufficient freely distributable reserves. As of December 31, 2021 , no dividends have been declared or paid since the Company’s inception. Liquidation The holders of the common shares are entitled to share ratably in the Company’s assets available for distribution to shareholders in the event of any voluntary or involuntary liquidation, dissolution or winding up of the Company or upon the occurrence of a deemed liquidation event. |
Equity-based Compensation
Equity-based Compensation | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Equity-based Compensation | 11. Equity-based Compensation Option and Grant Plans In April 2015, the Company’s shareholders approved the 2015 Stock Option and Grant Plan, or the 2015 Plan, and in July 2016, the Company’s shareholders approved the 2016 Stock Option and Incentive Plan, or the 2016 Plan. In May 2018, the Company’s shareholders approved the 2018 Stock Option and Incentive Plan, or the 2018 Plan (collectively, the “Plans”). Subsequent to the IPO, no further options were granted under the 2015 Plan. The Plans provide for the issuance of equity awards in the form of restricted shares, options to purchase common shares which may constitute incentive stock options, or ISOs, or non-statutory stock options, or NSOs, unrestricted stock unit grants, and qualified performance and market-based awards to eligible employees, officers, directors, non-employee consultants and other key personnel. Terms of the equity awards, including vesting requirements, are determined by the Company’s board of directors, subject to the provisions of the Plans. Options granted by the Company typically vest over four years and have a contractual life of ten years . Restricted stock unit grants typically vest over two to three years . At December 31, 2021, the Company had 25,005,365 common shares authorized for issuance under the 2018 Plan and 10,298,664 common shares available for future grant under the 2018 Plan. Equity-Based Compensation Expense The Company recognized stock-based compensation expense totaling $ 102.4 million, $ 66.0 million, and $ 49.5 million during the years ended December 31, 2021, 2020 and 2019 , respectively. Stock‑based compensation expense by classification within the consolidated statements of operations and comprehensive income (loss) is as follows (in thousands): Years Ended December 31, 2021 2020 2019 Research and development $ 59,683 $ 35,120 $ 23,273 General and administrative 42,707 30,898 20,784 Loss from equity method investment — — 5,467 Total $ 102,390 $ 66,018 $ 49,524 As of December 31, 2021 , there was $ 150.6 million and $ 71.4 million of unrecognized compensation expense related to unvested stock options and restricted stock units, respectively, that is expected to be recognized over a weighted-average period of 2.7 and 2.5 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: Years Ended December 31, 2021 2020 2019 Options granted 1,616,255 2,182,773 2,832,784 Weighted-average exercise price $ 124.32 $ 68.91 $ 39.16 Weighted-average grant date fair value $ 77.38 $ 42.28 $ 24.57 Assumptions: Expected volatility 70.3 % 69.2 % 68.9 % Expected term (in years) 6.0 6.0 6.0 Risk-free interest rate 1.0 % 0.6 % 2.2 % Expected dividend yield 0.0 % 0.0 % 0.0 % The following table summarizes stock option activity under the Company’s equity award plans (intrinsic value in thousands): Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2020 8,101,980 $ 42.44 7.8 $ 896,666 Granted 1,616,255 $ 124.32 Exercised ( 1,245,071 ) $ 30.96 Cancelled or forfeited ( 660,182 ) $ 79.54 Outstanding at December 31, 2021 7,812,982 $ 58.07 7.4 219,103 Exercisable at December 31, 2021 4,598,353 $ 40.86 6.6 $ 174,056 Vested and expected to vest at December 31, 2021 7,812,982 $ 58.07 7.4 $ 219,103 During 2021 and 2020, the Company did no t grant stock option awards subject to performance-based or market-based vesting conditions. As of December 31, 2021 , options to purchase 998,504 common shares subject to performance-based vesting conditions were vested, as performance conditions were achieved, and there were 123,057 options to purchase common shares subject to performance-based vesting conditions outstanding. Activity related to stock options subject to performance-based vesting conditions is included in the table above. During 2017, the Company granted 150,000 options with market-based vesting conditions, of which 75 % vest at the end of a three-year service period and 25 % vest at the end of a four-year service period. Upon achieving a specified average stock price in prior years, the market conditions were satisfied. Expense for the options is being recognized over the requisite service period. As of December 31, 2021 , 150,000 of the stock options had vested. The total intrinsic value (the amount by which the fair market value exceeded the exercise price) of stock options exercised during the year ended December 31, 2021, 2020 and 2019 was $ 119.5 million, $ 104.2 million, and $ 42.2 million, respectively. Restricted Stock The following table summarizes the restricted stock activity under the Company’s equity award plans: Shares Weighted- Unvested balance at December 31, 2020 894,092 $ 70.55 Granted 628,175 111.73 Vested ( 455,440 ) 58.00 Cancelled or forfeited ( 132,652 ) 100.25 Unvested balance at December 31, 2021 934,175 $ 100.14 During the years ended December 31, 2021, 2020 and 2019 , the total fair value of restricted stock vested was $ 45.3 million, $ 21.6 million, and $ 3.6 million, respectively. Award modifications Equity award modifications for certain equity awards held by departing employees and non-employees for the years ended December 31, 2021, 2020 and 2019 were not material to the Company's stock-based compensation expense. Employee Stock Purchase Plan On July 19, 2016, the Company’s board of directors adopted its 2016 Employee Stock Purchase Plan, or the ESPP Plan, which was subsequently approved by its shareholders and became effective on October 19, 2016. The ESPP Plan authorizes the initial issuance of up to a total of 0.4 million shares of the Company’s common stock to participating employees. The Company activated its ESPP Plan on January 1, 2020. The Company issued 21,590 and 13,410 shares under the ESPP during the years ended December 31, 2021 and 2020, respectively . |
Net Income (Loss) Per Share Att
Net Income (Loss) Per Share Attributable to Common Shareholders | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Share Attributable to Common Shareholders | 12. Net Income (Loss) Per Share Attributable to Common Shareholders Basic net income (loss) per share is calculated by dividing net income (loss) attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net income (loss) per share is calculated by dividing the net income (loss) attributable to common shareholders by the weighted-average number of common share equivalents outstanding for the period, including any dilutive effect from outstanding stock options and warrants using the treasury stock method. The Company’s net income (loss) is net income (loss) attributable to common shareholders for all periods presented. The following table sets forth the computation of basic and diluted net income (loss) per share for the periods ended (in thousands, except share and per share amounts): Year ended December 31, 2021 2020 2019 Net income (loss) $ 377,661 $ ( 348,865 ) $ 66,858 Basic weighted-average common shares outstanding 75,948,686 65,949,672 54,392,304 Effect of potentially dilutive securities: Outstanding options 3,990,579 — 2,406,962 Unvested restricted common shares 454,231 — 133,532 Diluted weighted-average common shares outstanding 80,393,496 65,949,672 56,932,798 Net income (loss) per common share — basic $ 4.97 $ ( 5.29 ) $ 1.23 Net income (loss) per common share — diluted $ 4.70 $ ( 5.29 ) $ 1.17 The Company did not include the securities in the following table in the computation of the net income (loss) per share calculations because the effect would have been anti-dilutive during each period: Year ended December 31, 2021 2020 2019 Outstanding options 1,765,881 8,101,980 3,789,129 Unvested restricted common shares 225,904 894,092 108,625 ESPP 6,671 11,257 — Total 1,998,456 9,007,329 3,897,754 |
401 (k) Savings Plan
401 (k) Savings Plan | 12 Months Ended |
Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |
401(k) Savings Plan | 13. 401(k) Savings Plan The Company established a defined‑contribution savings plan under Section 401(k) of the Internal Revenue Code (the “401(k) Plan”) in November 2016. 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. The Company contributed $ 3.4 million, $ 1.9 million, and $ 1.1 million to the 401(k) Plan for the year ended December 31, 2021, 2020 and 2019 , respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income Taxes The Company is subject to U.S. federal and various state corporate income taxes as well as taxes in foreign jurisdictions for the foreign parent and where foreign subsidiaries have been established. Net income (loss) before taxes For the years ended December 31, 2021, 2020 and 2019, the income (loss) before provision for income taxes consist of the following (in thousands): Years Ended December 31, 2021 2020 2019 Domestic $ 4,569 $ 7,630 $ 9,155 Foreign 374,962 ( 355,686 ) 58,151 Total $ 379,531 $ ( 348,056 ) $ 67,306 The (provision for) benefit from income taxes consist of the following (in thousands): Years Ended December 31, 2021 2020 2019 Current income taxes: Federal $ ( 80 ) $ ( 248 ) $ ( 423 ) State ( 42 ) ( 151 ) ( 59 ) Foreign — ( 1 ) 0 Total current income taxes ( 122 ) ( 400 ) ( 482 ) Deferred income taxes: Federal ( 1,748 ) ( 409 ) 34 State — — — Foreign — — — Total deferred income taxes ( 1,748 ) ( 409 ) 34 Total income tax (provision) benefit $ ( 1,870 ) $ ( 809 ) $ ( 448 ) A reconciliation of income tax expense computed at the statutory corporate income tax rate to the effective income tax rate for the years ended December 31, 2021, 2020 and 2019 is as follows: Years Ended December 31, 2021 2020 2019 Income tax expense at statutory rate 11.9 % 11.9 % 9.3 % State income tax, net of federal benefit ( 1.0 )% 1.0 % ( 2.1 )% Nondeductible expenses 0.7 % 0.1 % ( 0.1 )% Foreign rate differential 0.6 % ( 0.1 )% 2.0 % Statutory to US GAAP permanent differences 0.0 % 0.0 % 0.1 % Stock-based compensation ( 2.5 )% 2.3 % ( 2.0 )% Impact of deferred rate change 0.0 % 0.0 % ( 12.2 )% Research credits ( 4.2 )% 3.3 % ( 5.2 )% Change in valuation allowance ( 5.0 )% ( 18.7 )% 10.9 % Effective income tax rate 0.5 % ( 0.2 %) 0.7 % The federal statutory rate reflects the Switzerland mixed company service rate. 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 are comprised of the following (in thousands): Years Ended December 31, 2021 2020 Deferred tax assets: Net operating loss carryforwards $ 47,190 $ 84,531 Accruals and reserves 5,878 4,785 Operating lease liabilities 61,476 16,782 Other deferred tax assets 6,362 1,517 Stock-based compensation 14,042 9,605 Deferred revenue — 86 Research credit 37,878 19,526 Total deferred tax assets 172,826 136,832 Less valuation allowance ( 98,649 ) ( 116,640 ) Net deferred tax assets 74,177 20,192 Deferred tax liabilities: Depreciation ( 28,579 ) ( 6,778 ) Operating lease assets ( 47,521 ) ( 13,776 ) Intangible assets ( 31 ) ( 31 ) Other deferred tax liabilities ( 192 ) ( 4 ) Total deferred tax liabilities ( 76,323 ) ( 20,589 ) Long term deferred taxes $ ( 2,146 ) $ ( 397 ) The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on the Company’s history of worldwide operating losses, the Company has concluded that it is more-likely-than-not that the benefit of its U.S. and non-U.S. deferred tax assets will not be realized. Accordingly, as of December 31, 2021 and 2020 , the Company has provided a full valuation allowance against its net deferred tax assets in Switzerland and the United Kingdom. The Company has also provided a valuation allowance against the U.S. deferred tax assets that cannot be realized by existing deferred tax liabilities based upon when they are scheduled to reverse. The valuation allowance decreased by $ 18.0 million during 2021, which is primarily attributable to decreases in net operating loss carryforwards as a result of current year net income. As of December 31, 2021, the Company had available U.S. federal net operating loss carryforwards of $ 3.3 million. The U.S. federal net operating losses can be carried forward indefinitely. As of December 31, 2021 , the Company had available non-U.S. net operating loss carryforwards of $ 772.9 million of which $ 385.2 million relate to Switzerland, $ 385.2 million relate to the Canton of Zug, and $ 2.5 million relate to the Company’s wholly-owned subsidiary in the United Kingdom. The net operating losses generated in Switzerland and the Canton of Zug begin to expire in 2027 and the net operating losses generated in the United Kingdom can be carried forward indefinitely. As of December 31, 2021 , the Company had U.S. domestic federal research and development credit carryforwards of $ 19.4 million begin to expire in 2039 for federal purposes, which are net of uncertain tax positions of $ 9.9 million. As of December 31, 2021, the Company had U.S. domestic federal orphan drug credit carryforwards of $ 10.6 million which begin to expire in 2040 for federal purposes, which are net of uncertain tax positions of $ 4.5 million. As of December 31, 2021 , the Company had U.S. domestic state research and development credit carryforwards of $ 10.0 million which begin to expire in 2035 , which are net of uncertain tax positions of $ 7.0 million. ASC 740 clarifies the accounting for uncertainty in income taxes recognized in an enterprise’s financial statement by prescribing the minimum recognition threshold and measurement of a tax position taken or expected to be taken in a tax return. As of December 31, 2021 , the Company had gross unrecognized tax benefits of $ 21.4 million of which $ 19.9 million would favorably impact the effective tax rate if recognized. The Company will recognize interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2021, 2020 and 2019 , the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’s consolidated statements of operations and comprehensive loss. The aggregate changes in gross unrecognized tax benefits were as follows (in thousands): Years Ended December 31, 2021 2020 2019 Balance at beginning of year $ 11,967 $ 5,231 $ 1,595 Increases for tax positions taken during current period 9,911 7,004 2,754 Increases for tax positions taken in prior periods — — 882 Decreases for tax positions taken during current period — — — Decreases for tax positions taken in prior periods ( 483 ) ( 268 ) — Balance at end of year $ 21,395 $ 11,967 $ 5,231 The Company files income tax returns in the U.S. federal jurisdiction, Massachusetts, California and certain non-U.S. jurisdictions. The Company is subject to U.S. federal, Massachusetts, California and non-U.S. income tax examinations by authorities for tax years ending after December 31, 2017. Research credits generated in prior tax years that are closed for examination may still be adjusted upon future examination if they have or will be used in a future period. The Company is subject to income tax examinations by authorities in its non-U.S. jurisdictions for all years. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 15. Related Party Transactions Casebia Prior to the termination of the joint venture, Casebia was a related party under ASC 850, Related Party Disclosures (“ASC 850”) . Refer to Note 9, “Agreements with Bayer Healthcare LLC”. Vertex In the fourth quarter of 2018, upon becoming owners of record of more than 10 % of the voting interest of the Company, Vertex became a related party under ASC 850 . As of July 2, 2019, upon becoming owners of record of less than 10% of the voting interest of the Company, Vertex was no longer a related party under ASC 850. Refer to Note 9, “Agreements with Vertex Pharmaceuticals Incorporated and certain of its subsidiaries”. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies and Basis of Presentation (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Use of Estimates | Basis of Presentation and Use of Estimates The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America, or GAAP, and include the accounts of the Company and its wholly-owned subsidiaries as of December 31, 2021. All intercompany accounts and transactions have been eliminated. Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the Accounting Standards Codification, or ASC, and Accounting Standards Updates, or ASUs, of the Financial Accounting Standards Board. Prior to December 13, 2019, the Company accounted for its 50 % investment in Casebia Therapeutics, Limited Liability Partnership, or Casebia, under the equity method. As described in Note 9, on December 13, 2019, Casebia became a wholly-owned subsidiary and, as a result, the Company consolidated Casebia’s financial results from that date forward. The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of research and development expenses during the period. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. |
Segment Information | Segment Information The Company and the Company’s chief operating decision maker, namely, the chief executive officer, view the Company’s operations and manage its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. |
Foreign Currency Translation and Transactions | Foreign Currency Translation and Transactions The majority of the Company’s operations occur in entities that have the U.S. dollar as their functional currency. Non-U.S. dollar denominated functional currency subsidiaries have assets and liabilities translated into U.S. dollars at rates of exchange in effect at the end of the year. Revenue and expense amounts are translated using the average exchange rates for the period. Net unrealized gains and losses resulting from foreign currency translation are included in “Accumulated other comprehensive income (loss).” Net foreign currency exchange transaction gains or losses are included in “Other income (expense), net” on the Company’s consolidated statement of operations, the impact of which is not significant. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments with maturities of three months or less from the purchase date to be cash equivalents. As of December 31, 2021 and 2020 , the Company had $ 923.0 million and $ 1,168.6 million in cash and cash equivalents, respectively. |
Restricted Cash | Restricted Cash As of December 31, 2021 , the Company had $ 16.9 million in restricted cash representing letters of credit securing the Company’s obligations under certain leased facilities, as well as certain credit card arrangements, which was unchanged from December 31, 2020. The letters of credit are secured by cash held in a restricted depository account and recorded in restricted cash in the accompanying consolidated balance sheet as of December 31, 2021 . |
Marketable Securities | Marketable Securities As of December 31, 2021 and 2020 , the Company had $ 1,456.1 million and $ 521.7 million, respectively in marketable securities. The Company’s investment strategy is focused on capital preservation. The Company invests in instruments that meet the credit quality standards outlined in the Company’s investment policy. The Company classifies marketable securities with a remaining maturity, when purchased, of greater than three months as available-for-sale. Marketable securities are classified as current assets on the consolidated balance sheets if the marketable securities are available to be converted into cash to fund current operations. As a result, the Company classified all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, 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 interest expense over the period of the earliest call date, and any discount arising at purchase is accreted to interest income over the life of the instrument. Realized gains and losses on debt securities are determined using the specific identification method and are included in other income (expense), net. Effective January 1, 2020 , the Company adopted ASU 2016-13, Financial Instruments – Credit Losses (Topic 326): Measurement of Credit Losses on Financial Statements , or ASC 326. As the Company did not hold available-for-sale debt securities upon adoption, no related transition provisions were applicable to the Company upon adoption. The Company assesses its available-for-sale debt securities under the available-for-sale debt security impairment model in ASC 326 as of each reporting date in order to determine if a portion of any decline in fair value below carrying value recognized on its available-for-sale debt securities is the result of a credit loss. The Company records credit losses in the consolidated statements of operations and comprehensive loss as credit loss expense within other expense, net, which is limited to the difference between the fair value and the amortized cost of the security. To date, the Company has not recorded any credit losses on its available-for-sale debt securities. |
Other Receivables | Other Receivables Amounts due from collaboration partners where an arrangement is accounted for under ASC 808, Collaborative Arrangements , or ASC 808, are considered other receivables and are included within prepaid and other current assets in the consolidated balance sheet. Other receivables consisted of $ 8.4 million and $ 10.7 million as of December 31, 2021 and 2020 , respectively and are due from Vertex. Other receivables are recorded at invoiced amounts due under the Vertex collaboration agreement, as described further in Note 9. Vertex is a creditworthy entity that maintains an ongoing relationship with the Company and as such, the Company does not have an allowance for estimated credit losses recorded related to these other receivables. |
Concentrations of Credit Risk and Off-balance Sheet Risk | Concentrations of Credit Risk and Off-balance Sheet Risk Financial instruments that potentially subject the Company to concentrations of credit risk are primarily cash, cash equivalents and marketable securities. The Company’s cash is held in accounts with financial institutions that management believes are creditworthy. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds. The Company has no financial instruments with off-balance sheet risk of loss. |
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 — Quoted prices in active markets that are accessible at the market date for identical unrestricted assets or liabilities. Level 2 — Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs for which all significant inputs are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. 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 Measurement ). The carrying amount of accounts receivable, other receivables, accounts payable and accrued expenses as reported on the consolidated balance sheets as of December 31, 2021 and 2020 , approximate fair value, due to the short-term duration of these instruments. |
Property and Equipment | Property and Equipment Property and equipment are recorded at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred. Upon disposal, the related cost and accumulated depreciation is removed from the accounts and any resulting gain or loss is included in the results of operations. Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, which are as follows: Asset Estimated useful life Computer equipment 3 years Furniture, fixtures and other 5 years Laboratory equipment 5 years Leasehold improvements Shorter of 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. |
Revenue Recognition | Revenue Recognition The Company records revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers , or ASC 606. ASC 606 applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases and collaboration arrangements. To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: 1) Identify the contract with the customer A contract with a customer exists when (i) the Company enters into an enforceable contract with a customer that defines each party’s rights regarding the goods or services to be transferred and identifies the related payment terms, (ii) the contract has commercial substance and (iii) the Company determines that collection of substantially all consideration for goods and services that are transferred is probable based on the customer’s intent and ability to pay the promised consideration. 2) Identify the performance obligations in the contract Performance obligations promised in a contract are identified based on the goods and services that will be transferred to the customer that are both capable of being distinct, whereby the customer can benefit from the good or service either on its own or together with other available resources, and are distinct in the context of the contract, whereby the transfer of the good or service is separately identifiable from other promises in the contract. To the extent a contract includes multiple promised goods and services, the Company must apply judgment to determine whether promised goods and services are capable of being distinct and distinct in the context of the contract. If these criteria are not met, the promised goods and services are accounted for as a combined performance obligation. 3) Determine the transaction price The transaction price is determined based on the consideration to which the Company will be entitled in exchange for transferring goods and services to the customer. To the extent the transaction price includes variable consideration such as research, development, regulatory and commercial milestones, the Company determines if it is probable that it will receive such amounts and there is no risk of a significant revenue reversal. When the Company cannot conclude that receipt of such amounts is probable, the Company constrains the related variable consideration resulting in its exclusion from transaction consideration. In determining the portion of the transaction consideration to be constrained, the Company considers the probability and uncertainty that the related research, developmental, regulatory and commercial milestones will be achieved given the nature of research and clinical development and the stage of the underlying programs. This assessment is performed at each reporting period. In making this evaluation, the Company considers both internal and external information available, including information from industry publications and other relevant factors. Changes to the constraint of variable consideration can have a material effect on the amount of revenue recognized in the period. 4) Allocate the transaction consideration to performance obligations in the contract If the contract contains a single performance obligation, the entire transaction consideration is allocated to the single performance obligation. Contracts that contain multiple performance obligations require an allocation of the transaction consideration to each performance obligation on a relative standalone selling price basis unless the transaction consideration is variable and meets the criteria to be allocated entirely to a performance obligation or to a distinct service that forms part of a single performance obligation. The consideration to be received is allocated among the separate performance obligations based on relative standalone selling prices. In determining these estimated standalone selling prices, the Company makes a number of significant judgements including, for licenses, management’s assumptions regarding probability weighted projected discounted cash flows for each of the collaboration development programs. The estimated standalone selling prices are sensitive to changes in assumptions, such as probabilities of scientific success, discount rate and certain assumptions that form the basis of forecasted cash flows. In developing these assumptions, management considers both internal and external information available, including information from other guideline companies within the same industry and other relevant factors. Changes to these assumptions can have a material effect on the allocation of the transaction consideration to performance obligations, as well as the amount and timing of revenue recognized. 5) Recognize revenue when or as the Company satisfies a performance obligation The Company satisfies performance obligations over time or at a point in time, depending on the nature of the performance obligation. Revenue is recognized over time if the customer simultaneously receives and consumes the benefits provided by the entity’s performance, the entity’s performance creates or enhances an asset that the customer controls as the asset is created or enhanced, or the entity’s performance does not create an asset with an alternative use to the entity and the entity has an enforceable right to payment for performance completed to date. If the entity does not satisfy a performance obligation over time, the related performance obligation is satisfied at a point in time by transferring the control of a promised good or service to a customer. Contract Balances The Company recognizes a contract asset when the Company transfers goods or services to a customer before the customer pays consideration or before payment is due, excluding any amounts presented as an account or other receivable. A contract asset is an entity’s right to consideration in exchange for goods or services that the entity has transferred to a customer. The contract liabilities, or deferred revenue, primarily relate to contracts where we have received payment, but we have not yet satisfied the related performance obligations. Contract assets are not significant as of December 31, 2021 and 2020. Contract liabilities recorded as deferred revenue as of December 31, 2021 and 2020 are $ 12.3 million and $ 12.2 million, respectively. The change in contract assets and contract liabilities recorded as deferred revenue is related to the collaboration agreement with Vertex described in Note 9. |
Collaboration Arrangements | Collaboration Arrangements The Company records the elements of its collaboration agreements that represent joint operating activities in accordance with ASC 808, Collaborative arrangements , or ASC 808. Accordingly, the elements of the collaboration agreements that represent activities in which both parties are active participants and to which both parties are exposed to the significant risks and rewards that are dependent on the commercial success of the activities, are recorded as collaborative arrangements. The Company evaluates the proper presentation of the commercial activities and the profit and loss sharing associated with the collaboration agreements. ASC 808 states that when payments between parties in a collaborative arrangement are not within the scope of other authoritative accounting literature, the income statement classification should be based on the nature of the arrangement, the nature of its business operations and the contractual terms of the arrangement. To the extent that these payments are not within the scope of other authoritative accounting literature, the income statement classification for the payments shall be based on an analogy to authoritative accounting literature or if there is no appropriate analogy, a reasonable, rational and consistently applied accounting policy election. |
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 and other related costs. Research and development costs, including up-front fees and milestones paid to collaborators, are also expensed 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 a reduction to research and development expense in the period the services are provided. |
Leases | Leases The Company accounts for its leases in accordance with ASC 842, Leases , or ASC 842. 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 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. 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 of renewal. 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. |
Equity-Based Compensation Expense | Equity Based Compensation Expense The Company’s share-based compensation programs grant awards that have included stock options, restricted stock units and restricted stock awards. Grants are awarded to employees and non-employees, including directors. The Company accounts for its stock-based compensation awards in accordance with ASC Topic 718, Compensation—Stock Compensation , or ASC 718. ASC 718 requires all stock-based payments to employees and non-employee directors, including grants of employee stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive income (loss) based on their fair values. The Company uses the Black-Scholes option pricing model to determine the fair value of options granted. The Company accounts for forfeitures as they occur instead of estimating forfeitures at the time of grant and revising those estimates in subsequent periods if actual forfeitures differ from its estimates. Stock-based compensation expense recognized in the financial statements is based on awards for which performance or service conditions are expected to be satisfied. The Company’s stock-based awards are subject to service or performance-based vesting conditions. Compensation expense related to awards to employees, directors and non-employees 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 to employees 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 expenses restricted stock unit awards to employees based on the fair value of the award on a straight-line basis over the associated service period of the award. The Company estimates the fair value of its option awards to employees, directors and non-employees 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. |
Patent Costs | Patent Costs Costs to secure and prosecute patent applications and other legal costs related to the protection of the Company’s intellectual property are expensed as incurred and are classified as general and administrative expenses in the Company’s consolidated statements of operations. |
Income Taxes | Income Taxes Income taxes are recorded in accordance with ASC Topic 740, Income Taxes , or ASC 740, which provides for deferred taxes using an asset and liability approach. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and tax reporting basis of assets and liabilities and are measured using enacted tax rates and laws that are expected to be in effect when 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 has evaluated available evidence and concluded that the Company may not realize all the benefit of its deferred tax assets; therefore, a valuation allowance has been established for the amount of the deferred tax assets that the Company does not believe is more likely than not to 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. As of December 31, 2021 and 2020 , the Company does no t have any significant uncertain tax positions. The Company’s practice is to recognize interest and/or penalties related to income tax matters in income tax expense. See Note 14 for further details. |
Comprehensive (Loss) Income | Comprehensive Income (Loss) Comprehensive income (loss) consists of net income or loss and other comprehensive income (loss). Other comprehensive income (loss) consists of foreign currency translation adjustments and unrealized losses on marketable securities. |
Variable Interest Entities | Variable Interest Entities The Company reviews each legal entity formed by parties related to the Company to determine whether or not the Company has a variable interest in the entity and whether or not the entity would meet the definition of a variable interest entity, or VIE, in accordance with ASC Topic 810, Consolidation , or ASC 810. If the entity is a VIE, the Company assesses whether or not the Company is the primary beneficiary of that VIE based on a number of factors, including (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to any contractual agreements and (iii) which party has the obligation to absorb losses or the right to receive benefits from the VIE. If the Company determines it is the primary beneficiary of a VIE, the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements at the time that determination is made. The Company evaluates whether it continues to be the primary beneficiary of any consolidated VIEs on a quarterly basis. If the Company were to determine that it is no longer the primary beneficiary of a consolidated VIE, or no longer has a variable interest in the VIE, it would deconsolidate the VIE in the period that the determination is made. If the Company determines it is the primary beneficiary of a VIE that meets the definition of a business, the Company measures the assets, liabilities and noncontrolling interests of the newly consolidated entity at fair value in accordance with ASC Topic 805, Business Combinations , or ASC 805, at the date the reporting entity first becomes the primary beneficiary. In February 2016, Casebia, was formed in the United Kingdom. In March 2016, upon consummation of the joint venture (“JV”), Bayer Healthcare LLC and certain of its affiliates (“Bayer”) and the Company each received a 50 % equity interest in the entity in exchange for their contributions to the entity. The Company determined that Casebia was considered a VIE and concluded that it is not the primary beneficiary of the VIE. As such, the Company has not historically consolidated Casebia’s results into the consolidated financial statements. As described in Note 9, on December 13, 2019, Casebia became a fully-owned subsidiary and, as a result, the Company consolidated Casebia’s financial results accordingly from that point forward. |
Net (Loss) Income Per Share Attributable to Common Shareholders | Net Income (Loss) Per Share Attributable to Common Shareholders Basic net income (loss) per share is calculated by dividing net income (loss) attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net income (loss) per share is calculated by dividing the net income (loss) attributable to common shareholders by the weighted-average number of common equivalent shares outstanding for the period, including any dilutive effect from outstanding stock options and restricted stock units using the treasury stock method. See Note 12 for further details. |
New Accounting Pronouncements | New Accounting Pronouncements From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies that the Company adopts as of the specified effective date. Unless otherwise discussed below, the Company does not believe that the adoption of recently issued standards have or may have a material impact on its consolidated financial statements and disclosures. 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, or 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 Company adopted ASU 2019-12 on January 1, 2021 . The adoption of ASU 2019-12 did not have a material impact on the Company’s financial position or 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, 2021 | |
Accounting Policies [Abstract] | |
Schedule of Estimated Useful Lives of Assets | Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, which are as follows: Asset Estimated useful life Computer equipment 3 years Furniture, fixtures and other 5 years Laboratory equipment 5 years Leasehold improvements Shorter of useful life or remaining lease term |
Marketable Securities (Tables)
Marketable Securities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value | A summary of the Company’s cash equivalents and marketable securities as of December 31, 2021 and 2020 , which are recorded at fair value (and excludes $ 405.6 million and $ 395.1 million of cash at December 31, 2021 and 2020, respectively) is shown below (in thousands): December 31, 2021 Amortized Gross Gross Fair Value Cash equivalents: Money market funds $ 507,386 $ — $ — $ 507,386 Corporate debt securities — — — — Certificates of deposit — — — — Commercial paper 9,997 — ( 1 ) 9,996 Total cash equivalents 517,383 — ( 1 ) 517,382 Marketable securities: U.S. Treasury securities 16,238 6 ( 52 ) 16,192 Corporate debt securities 1,173,659 10 ( 4,903 ) 1,168,766 Certificates of deposit 45,164 — — 45,164 Government-sponsored enterprise securities 13,334 — ( 77 ) 13,257 Commercial paper 212,805 — ( 86 ) 212,719 Total marketable securities 1,461,200 16 ( 5,118 ) 1,456,098 Total cash equivalents and marketable securities $ 1,978,583 $ 16 $ ( 5,119 ) $ 1,973,480 December 31, 2020 Amortized Gross Gross Fair Value Cash equivalents: Money market funds $ 742,958 $ — $ — $ 742,958 Corporate debt securities 2,526 1 ( 24 ) 2,503 Certificates of deposit 12,527 — — 12,527 Commercial paper 15,549 — — 15,549 Total cash equivalents 773,560 1 ( 24 ) 773,537 Marketable securities: U.S. Treasury securities 47,976 3 — 47,979 Corporate debt securities 324,569 43 ( 156 ) 324,456 Certificates of deposit 25,162 — — 25,162 Government-sponsored enterprise securities 33,738 5 ( 2 ) 33,741 Commercial paper 90,375 — — 90,375 Total marketable securities 521,820 51 ( 158 ) 521,713 Total cash equivalents and marketable securities $ 1,295,380 $ 52 $ ( 182 ) $ 1,295,250 |
Fair Value Measurement (Tables)
Fair Value Measurement (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets Measured at Fair Value on Recurring Basis | The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of December 31, 2021 and 2020 (in thousands): Fair Value Measurements at December 31, 2021 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 405,648 $ 405,648 $ — $ — Money market funds 507,386 507,386 — — Corporate debt securities — — — — Certificates of deposit — — — — Commercial paper 9,997 — 9,997 — Marketable securities: U.S. Treasury securities 16,192 — 16,192 — Corporate debt securities 1,168,766 — 1,168,766 — Certificates of deposit 45,164 — 45,164 — Government-sponsored enterprise securities 13,257 — 13,257 — Commercial paper 212,719 — 212,719 — Other non-current assets 2,212 — — 2,212 Total $ 2,381,341 $ 913,034 $ 1,466,095 $ 2,212 Fair Value Measurements at December 31, 2020 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 395,083 $ 395,083 $ — $ — Money market funds 742,958 742,958 — — Corporate debt securities 2,503 — 2,503 — Certificates of deposit 12,527 — 12,527 — Commercial paper 15,549 — 15,549 — Marketable securities: U.S. Treasury securities 47,979 — 47,979 — Corporate debt securities 324,456 — 324,456 — Certificates of deposit 25,162 — 25,162 — Government-sponsored enterprise securities 33,741 — 33,741 — Commercial paper 90,375 — 90,375 — Other non-current assets 600 — — 600 Total $ 1,690,933 $ 1,138,041 $ 552,292 $ 600 |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net, consists of the following (in thousands): As of December 31, 2021 2020 Computer equipment $ 1,757 $ 727 Furniture, fixtures, and other 4,371 3,416 Laboratory equipment 30,123 25,353 Leasehold improvements 86,735 25,473 Construction work in process 52,396 8,366 Total property and equipment, gross 175,382 63,335 Accumulated Depreciation ( 37,807 ) ( 21,175 ) Total property and equipment, net $ 137,575 $ 42,160 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consist of the following (in thousands): As of December 31, 2021 2020 Payroll and employee-related costs $ 23,661 $ 22,402 Research costs 47,986 21,684 Licensing fees 138 1,401 Professional fees 4,720 1,670 Intellectual property costs 6,120 3,625 Accrued property and equipment 7,113 2,835 Other 1,265 165 Total $ 91,003 $ 53,782 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
Summary of Lease Assets and Liabilities | The following table summarizes the lease assets and liabilities as of December 31, 2021 and 2020 (in thousands): As of December 31, 2021 2020 Assets Operating lease assets $ 174,460 $ 50,865 Total lease assets 174,460 50,865 Liabilities Current Operating lease liabilities 12,158 11,362 Non-current Operating lease liabilities, net of current portion 212,872 50,067 Total lease liabilities $ 225,030 $ 61,429 |
Summary of Operating Lease Costs Included in Research and Development and General and Administrative Expense, as well as Sublease Income | The following table summarizes operating lease costs included in research and development and general and administrative expense, as well as sublease income for the twelve months ended December 31, 2021, 2020 and 2019 (in thousands): Years Ended December 31, 2021 2020 2019 Operating lease costs $ 22,520 $ 14,342 $ 8,067 Short-term lease costs 11,087 7,339 4,554 Variable lease costs 8,402 6,368 4,282 Sublease income ( 5,253 ) ( 587 ) ( 525 ) Net lease cost $ 36,756 $ 27,462 $ 16,378 |
Summary of Maturity of Undiscounted Payments Due under Lease Liabilities and Present Value of Those Liabilities | The following table summarizes the maturity of undiscounted payments due under lease liabilities and the present value of those liabilities as of December 31, 2021 (in thousands): Total 2022 18,256 2023 26,633 2024 26,137 2025 26,347 2026 26,844 Thereafter 227,528 Total $ 351,745 Present value adjustment ( 126,715 ) Present value of lease liabilities $ 225,030 |
Summary of Lease Term and Discount Rate | The following table summarizes the lease term (in years) and discount rate for operating leases as of December 31, 2021 and 2020: As of December 31, 2021 2020 Weighted-average remaining lease term 12.4 9.1 Weighted-average discount rate 5.9 % 9.3 % |
Lessee, Operating Lease Cash Flow Payments | The following table summarizes the cash paid for amounts included in the measurement of lease liabilities for the year ended December 31, 2021 and 2020 (in thousands): Years Ended December 31, 2021 2020 2019 Cash paid for amounts included in measurement of lease liabilities: Operating cash flows used in operating leases $ ( 19,753 ) $ ( 13,161 ) $ ( 8,420 ) Operating lease non-cash items: Right-of-use assets (decreased) increased through lease modifications and reassessments ( 14,230 ) 3,169 826 Right-of-use assets obtained in exchange for operating lease liabilities 152,486 13,956 18,088 Leasehold improvements paid directly by landlord 30,500 — — |
Significant Contracts (Tables)
Significant Contracts (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Schedule of Fair Value of Assets and Liabilities Received | As a result of the Retirement Agreement, the Company determined that it had obtained a controlling interest in a VIE, for which it became the primary beneficiary. As such, under ASC 810, Consolidation, the Company accounted for the net assets obtained under ASC 805, Business Combinations . In accordance therewith, the Company determined the set of acquired assets and assumed liabilities did not meet the definition of a business, as the Company did not acquire an assembled workforce and thus the Company did not acquire substantive processes capable of producing outputs. As such, no goodwill was recorded. The Company measured the fair value of the assets and liabilities received, determining the relative fair value was $ 16.0 million (after paying the $ 16.0 million for Bayer’s 50 % interest) and recorded the difference between that amount and the Company’s carrying amount, which was zero , as a gain within other income (expense). The relative fair value of the assets and liabilities received (exclusive of the $ 16.0 million paid from Casebia to Bayer to retire Bayer’s interest in the JV) was determined as follows (in thousands): Fair value Amount Cash and cash equivalents $ 6,784 Prepaid expenses and other current assets 2,565 Property, plant and equipment, net 9,340 Operating lease assets 11,003 Restricted cash 1,226 Accrued expenses and other current liabilities ( 3,915 ) Operating lease liabilities ( 11,003 ) Net assets $ 16,000 |
Share Capital (Tables)
Share Capital (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Schedule of Share Capital | Share Capital consisted of the following: As of December 31, Type of Share Capital Conditional Capital 2021 2020 Common shares Registered share capital 80,321,227 75,133,951 Common shares Authorized share capital 39,316,975 17,625,426 Common shares Conditional share capital - Bonds or similar debt instruments 4,919,700 4,919,700 Common shares Conditional share capital - Employee benefit plans 20,806,433 17,493,709 Total 145,364,335 115,172,786 |
Equity-based Compensation (Tabl
Equity-based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Schedule of Stock-Based Compensation Expense | Stock‑based compensation expense by classification within the consolidated statements of operations and comprehensive income (loss) is as follows (in thousands): Years Ended December 31, 2021 2020 2019 Research and development $ 59,683 $ 35,120 $ 23,273 General and administrative 42,707 30,898 20,784 Loss from equity method investment — — 5,467 Total $ 102,390 $ 66,018 $ 49,524 |
Fair Value of Each Option Issued to Employees Estimated Using Black-Scholes Option-Pricing Model | 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: Years Ended December 31, 2021 2020 2019 Options granted 1,616,255 2,182,773 2,832,784 Weighted-average exercise price $ 124.32 $ 68.91 $ 39.16 Weighted-average grant date fair value $ 77.38 $ 42.28 $ 24.57 Assumptions: Expected volatility 70.3 % 69.2 % 68.9 % Expected term (in years) 6.0 6.0 6.0 Risk-free interest rate 1.0 % 0.6 % 2.2 % Expected dividend yield 0.0 % 0.0 % 0.0 % |
Summary of Stock Option Activity | The following table summarizes stock option activity under the Company’s equity award plans (intrinsic value in thousands): Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2020 8,101,980 $ 42.44 7.8 $ 896,666 Granted 1,616,255 $ 124.32 Exercised ( 1,245,071 ) $ 30.96 Cancelled or forfeited ( 660,182 ) $ 79.54 Outstanding at December 31, 2021 7,812,982 $ 58.07 7.4 219,103 Exercisable at December 31, 2021 4,598,353 $ 40.86 6.6 $ 174,056 Vested and expected to vest at December 31, 2021 7,812,982 $ 58.07 7.4 $ 219,103 |
Summary of Restricted Stock Activity | The following table summarizes the restricted stock activity under the Company’s equity award plans: Shares Weighted- Unvested balance at December 31, 2020 894,092 $ 70.55 Granted 628,175 111.73 Vested ( 455,440 ) 58.00 Cancelled or forfeited ( 132,652 ) 100.25 Unvested balance at December 31, 2021 934,175 $ 100.14 |
Net Income (Loss) Per Share A_2
Net Income (Loss) Per Share Attributable to Common Shareholders (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Schedule of Computation of Basic and Diluted Net Income (Loss) per Share | The following table sets forth the computation of basic and diluted net income (loss) per share for the periods ended (in thousands, except share and per share amounts): Year ended December 31, 2021 2020 2019 Net income (loss) $ 377,661 $ ( 348,865 ) $ 66,858 Basic weighted-average common shares outstanding 75,948,686 65,949,672 54,392,304 Effect of potentially dilutive securities: Outstanding options 3,990,579 — 2,406,962 Unvested restricted common shares 454,231 — 133,532 Diluted weighted-average common shares outstanding 80,393,496 65,949,672 56,932,798 Net income (loss) per common share — basic $ 4.97 $ ( 5.29 ) $ 1.23 Net income (loss) per common share — diluted $ 4.70 $ ( 5.29 ) $ 1.17 |
Schedule of Antidilutive Securities not Include in Computation of Net Income (Loss) per Share | The Company did not include the securities in the following table in the computation of the net income (loss) per share calculations because the effect would have been anti-dilutive during each period: Year ended December 31, 2021 2020 2019 Outstanding options 1,765,881 8,101,980 3,789,129 Unvested restricted common shares 225,904 894,092 108,625 ESPP 6,671 11,257 — Total 1,998,456 9,007,329 3,897,754 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of Income (Loss) Before Provision for Income Taxes | For the years ended December 31, 2021, 2020 and 2019, the income (loss) before provision for income taxes consist of the following (in thousands): Years Ended December 31, 2021 2020 2019 Domestic $ 4,569 $ 7,630 $ 9,155 Foreign 374,962 ( 355,686 ) 58,151 Total $ 379,531 $ ( 348,056 ) $ 67,306 |
Schedule of (Provision for) Benefit from Income Taxes | The (provision for) benefit from income taxes consist of the following (in thousands): Years Ended December 31, 2021 2020 2019 Current income taxes: Federal $ ( 80 ) $ ( 248 ) $ ( 423 ) State ( 42 ) ( 151 ) ( 59 ) Foreign — ( 1 ) 0 Total current income taxes ( 122 ) ( 400 ) ( 482 ) Deferred income taxes: Federal ( 1,748 ) ( 409 ) 34 State — — — Foreign — — — Total deferred income taxes ( 1,748 ) ( 409 ) 34 Total income tax (provision) benefit $ ( 1,870 ) $ ( 809 ) $ ( 448 ) |
Schedule of Reconciliation of Income Tax Expense Computed at Statutory Corporate Income Tax Rate to Effective Income Tax Rate | A reconciliation of income tax expense computed at the statutory corporate income tax rate to the effective income tax rate for the years ended December 31, 2021, 2020 and 2019 is as follows: Years Ended December 31, 2021 2020 2019 Income tax expense at statutory rate 11.9 % 11.9 % 9.3 % State income tax, net of federal benefit ( 1.0 )% 1.0 % ( 2.1 )% Nondeductible expenses 0.7 % 0.1 % ( 0.1 )% Foreign rate differential 0.6 % ( 0.1 )% 2.0 % Statutory to US GAAP permanent differences 0.0 % 0.0 % 0.1 % Stock-based compensation ( 2.5 )% 2.3 % ( 2.0 )% Impact of deferred rate change 0.0 % 0.0 % ( 12.2 )% Research credits ( 4.2 )% 3.3 % ( 5.2 )% Change in valuation allowance ( 5.0 )% ( 18.7 )% 10.9 % Effective income tax rate 0.5 % ( 0.2 %) 0.7 % |
Schedule of Significant Components of Company's Deferred Tax Assets | 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 are comprised of the following (in thousands): Years Ended December 31, 2021 2020 Deferred tax assets: Net operating loss carryforwards $ 47,190 $ 84,531 Accruals and reserves 5,878 4,785 Operating lease liabilities 61,476 16,782 Other deferred tax assets 6,362 1,517 Stock-based compensation 14,042 9,605 Deferred revenue — 86 Research credit 37,878 19,526 Total deferred tax assets 172,826 136,832 Less valuation allowance ( 98,649 ) ( 116,640 ) Net deferred tax assets 74,177 20,192 Deferred tax liabilities: Depreciation ( 28,579 ) ( 6,778 ) Operating lease assets ( 47,521 ) ( 13,776 ) Intangible assets ( 31 ) ( 31 ) Other deferred tax liabilities ( 192 ) ( 4 ) Total deferred tax liabilities ( 76,323 ) ( 20,589 ) Long term deferred taxes $ ( 2,146 ) $ ( 397 ) |
Schedule of Aggregate Changes in Gross Unrecognized Tax Benefits | The aggregate changes in gross unrecognized tax benefits were as follows (in thousands): Years Ended December 31, 2021 2020 2019 Balance at beginning of year $ 11,967 $ 5,231 $ 1,595 Increases for tax positions taken during current period 9,911 7,004 2,754 Increases for tax positions taken in prior periods — — 882 Decreases for tax positions taken during current period — — — Decreases for tax positions taken in prior periods ( 483 ) ( 268 ) — Balance at end of year $ 21,395 $ 11,967 $ 5,231 |
Organization and Operations - A
Organization and Operations - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Company incorporation date | Oct. 31, 2013 | |
Accumulated deficit | $ (195,915) | $ (573,576) |
Cash, cash equivalents and marketable securities | $ 2,379,100 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies and Basis of Presentation - Additional Information (Detail) | 12 Months Ended | |||||
Dec. 31, 2021USD ($)Segment | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 12, 2019 | Mar. 31, 2016 | Feb. 12, 2016 | |
Summary Of Significant Accounting Policies [Line Items] | ||||||
Number of operating segments | Segment | 1 | |||||
Cash and cash equivalents | $ 923,031,000 | $ 1,168,620,000 | $ 943,771,000 | |||
Restricted Cash | 16,913,000 | 16,848,000 | 5,041,000 | |||
Marketable securities | 1,456,098,000 | 521,713,000 | ||||
Contract liabilities | 12,300,000 | 12,200,000 | ||||
Net lease assets | 174,460,000 | 50,865,000 | ||||
Net lease liabilities | 225,030,000 | 61,429,000 | ||||
Significant uncertain tax positions | $ 0 | 0 | $ 0 | |||
Minimum [Member] | ||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||
Marketable securities maturity period | 1 year | |||||
Vertex and Casebia [Member] | Collaborative Arrangement [Member] | ||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||
Other receivables | $ 8,400 | $ 10,700,000 | ||||
ASC 326 [Member] | ||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||
Change in accounting principle, accounting standards update, adopted | true | |||||
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2020 | |||||
Change in accounting principle, accounting standards update, immaterial effect | true | |||||
ASU 2019-12 [Member] | ||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||
Change in accounting principle, accounting standards update, adopted | true | |||||
Change in accounting principle, accounting standards update, adoption date | Jan. 1, 2021 | |||||
Change in accounting principle, accounting standards update, immaterial effect | true | |||||
Casebia Therapeutics Llp [Member] | ||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||
Equity method investment, ownership percentage | 50.00% | 50.00% | 50.00% | 50.00% |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies and Basis of Presentation - Schedule of Estimated Useful Lives of Assets (Detail) | 12 Months Ended |
Dec. 31, 2021 | |
Computer Equipment [Member] | |
Property Plant And Equipment [Line Items] | |
Estimated useful lives of assets | 3 years |
Furniture, Fixtures, and Other [Member] | |
Property Plant And Equipment [Line Items] | |
Estimated useful lives of assets | 5 years |
Laboratory Equipment [Member] | |
Property Plant And Equipment [Line Items] | |
Estimated useful lives of assets | 5 years |
Leasehold Improvements [Member] | |
Property Plant And Equipment [Line Items] | |
Leasehold improvements | Shorter of useful life or remaining lease term |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Marketable Securities [Line Items] | |||
Marketable securities | $ 405,600 | $ 395,100 | |
Marketable securities unrealized loss, Less than twelve months | 1,311,600 | 280,300,000 | |
Marketable securities unrealized loss, more than twelve months | 4,600 | 0 | |
Unrealized loss, net | $ 0 | ||
Other Comprehensive Income (Loss) [Member] | |||
Marketable Securities [Line Items] | |||
Unrealized loss, net | $ 5,000 | $ 100,000 |
Marketable Securities - Summary
Marketable Securities - Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Marketable Securities [Line Items] | ||
Cash equivalents, amortized cost | $ 517,383 | $ 773,560 |
Cash equivalents, gross unrealized gains | 1 | |
Cash equivalents, gross unrealized losses | (1) | (24) |
Cash equivalents, fair value | 517,382 | 773,537 |
Marketable securities, amortized cost | 1,461,200 | 521,820 |
Marketable securities, gross unrealized gains | 16 | 51 |
Marketable securities, gross unrealized losses | (5,118) | (158) |
Marketable securities, fair value | 1,456,098 | 521,713 |
Cash equivalents and marketable securities, amortized cost | 1,978,583 | 1,295,380 |
Cash equivalents and marketable securities, gross unrealized gains | 16 | 52 |
Cash equivalents and marketable securities, gross unrealized losses | (5,119) | (182) |
Cash equivalents and marketable securities, fair value | 1,973,480 | 1,295,250 |
Money Market Funds [Member] | ||
Marketable Securities [Line Items] | ||
Cash equivalents, amortized cost | 507,386 | 742,958 |
Cash equivalents, fair value | 507,386 | 742,958 |
Certificates of Deposit [Member] | ||
Marketable Securities [Line Items] | ||
Cash equivalents, amortized cost | 12,527 | |
Cash equivalents, fair value | 12,527 | |
Marketable securities, amortized cost | 45,164 | 25,162 |
Marketable securities, fair value | 45,164 | 25,162 |
Commercial Paper [Member] | ||
Marketable Securities [Line Items] | ||
Cash equivalents, amortized cost | 9,997 | 15,549 |
Cash equivalents, gross unrealized losses | (1) | |
Cash equivalents, fair value | 9,996 | 15,549 |
Marketable securities, amortized cost | 212,805 | 90,375 |
Marketable securities, gross unrealized losses | (86) | |
Marketable securities, fair value | 212,719 | 90,375 |
US Treasury Securities [Member] | ||
Marketable Securities [Line Items] | ||
Marketable securities, amortized cost | 16,238 | 47,976 |
Marketable securities, gross unrealized gains | 6 | 3 |
Marketable securities, gross unrealized losses | (52) | |
Marketable securities, fair value | 16,192 | 47,979 |
Corporate Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Cash equivalents, amortized cost | 2,526 | |
Cash equivalents, gross unrealized gains | 1 | |
Cash equivalents, gross unrealized losses | (24) | |
Cash equivalents, fair value | 2,503 | |
Marketable securities, amortized cost | 1,173,659 | 324,569 |
Marketable securities, gross unrealized gains | 10 | 43 |
Marketable securities, gross unrealized losses | (4,903) | (156) |
Marketable securities, fair value | 1,168,766 | 324,456 |
Government-sponsored Enterprise Securities [Member] | ||
Marketable Securities [Line Items] | ||
Marketable securities, amortized cost | 13,334 | 33,738 |
Marketable securities, gross unrealized gains | 5 | |
Marketable securities, gross unrealized losses | (77) | (2) |
Marketable securities, fair value | $ 13,257 | $ 33,741 |
Fair Value Measurement - Schedu
Fair Value Measurement - Schedule of Financial Assets Measured at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 6,784 | |
Marketable securities, fair value | 1,456,098 | $ 521,713 |
Recurring Basis [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Other non-current assets | 2,212 | 600 |
Total | 2,381,341 | 1,690,933 |
Recurring Basis [Member] | Cash [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 405,648 | 395,083 |
Recurring Basis [Member] | Money Market Funds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 507,386 | 742,958 |
Recurring Basis [Member] | Corporate Debt Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 2,503 | |
Marketable securities, fair value | 1,168,766 | 324,456 |
Recurring Basis [Member] | Certificates of Deposit [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 12,527 | |
Marketable securities, fair value | 45,164 | 25,162 |
Recurring Basis [Member] | Commercial Paper [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 9,997 | 15,549 |
Marketable securities, fair value | 212,719 | 90,375 |
Recurring Basis [Member] | US Treasury Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable securities, fair value | 16,192 | 47,979 |
Recurring Basis [Member] | Government-sponsored Enterprise Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable securities, fair value | 13,257 | 33,741 |
Recurring Basis [Member] | Level 1 [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total | 913,034 | 1,138,041 |
Recurring Basis [Member] | Level 1 [Member] | Cash [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 405,648 | 395,083 |
Recurring Basis [Member] | Level 1 [Member] | Money Market Funds [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 507,386 | 742,958 |
Recurring Basis [Member] | Level 2 [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Total | 1,466,095 | 552,292 |
Recurring Basis [Member] | Level 2 [Member] | Corporate Debt Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 2,503 | |
Marketable securities, fair value | 1,168,766 | 324,456 |
Recurring Basis [Member] | Level 2 [Member] | Certificates of Deposit [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 12,527 | |
Marketable securities, fair value | 45,164 | 25,162 |
Recurring Basis [Member] | Level 2 [Member] | Commercial Paper [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | 9,997 | 15,549 |
Marketable securities, fair value | 212,719 | 90,375 |
Recurring Basis [Member] | Level 2 [Member] | US Treasury Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable securities, fair value | 16,192 | 47,979 |
Recurring Basis [Member] | Level 2 [Member] | Government-sponsored Enterprise Securities [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Marketable securities, fair value | 13,257 | 33,741 |
Recurring Basis [Member] | Level 3 [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Other non-current assets | 2,212 | 600 |
Total | $ 2,212 | $ 600 |
Property and Equipment, net - S
Property and Equipment, net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | $ 175,382 | $ 63,335 |
Accumulated Depreciation | (37,807) | (21,175) |
Total property and equipment, net | 137,575 | 42,160 |
Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | 1,757 | 727 |
Furniture, Fixtures, and Other [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | 4,371 | 3,416 |
Laboratory Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | 30,123 | 25,353 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | 86,735 | 25,473 |
Construction Work in Process [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Total property and equipment, gross | $ 52,396 | $ 8,366 |
Property and Equipment, net - A
Property and Equipment, net - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation expense | $ 17.9 | $ 9.1 | $ 4.7 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Accrued Liabilities, Current [Abstract] | ||
Payroll and employee-related costs | $ 23,661 | $ 22,402 |
Research costs | 47,986 | 21,684 |
Licensing fees | 138 | 1,401 |
Professional fees | 4,720 | 1,670 |
Intellectual property costs | 6,120 | 3,625 |
Accrued property and equipment | 7,113 | 2,835 |
Other | 1,265 | 165 |
Total | $ 91,003 | $ 53,782 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 1 Months Ended | 12 Months Ended | ||||||
Jul. 31, 2020USD ($)Term | May 31, 2020Term | Dec. 31, 2019 | May 31, 2019 | May 31, 2016 | Jun. 30, 2015 | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | |
Lessee Lease Description [Line Items] | ||||||||
Operating lease, right-of-use asset | $ 174,460 | $ 50,865 | ||||||
Operating lease liability | $ 225,030 | $ 61,429 | ||||||
2015 Lease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lease commencement date | Nov. 15, 2015 | |||||||
Lessee, operating lease, existence of option to extend | false | |||||||
Lessee, operating lease, option to extend | expires in 2022 with no further option to extend | |||||||
Lease expiration year | 2022 | |||||||
2016 Sublease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lease commencement date | Dec. 23, 2016 | |||||||
Lease expiration year | 2022 | |||||||
2019 Lease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lease commencement date | Jun. 1, 2019 | |||||||
Lessee, operating lease, existence of option to extend | true | |||||||
Lessee, operating lease, option to extend | an option to extend the term of the lease for an additional five-year period based on certain conditions within the Company’s control. | |||||||
Lease expiration, month and year | 2026-11 | |||||||
Extended term of lease expiration | 5 years | |||||||
2019 Sublease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lessee, operating lease, option to extend | expires in 2022 with no further option to extend | |||||||
Lease expiration year | 2022 | |||||||
Framingham Lease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lease expiration, month and year | 2036-03 | |||||||
Lessee, operating sublease, existence of option to extend | true | |||||||
Number of additional renewal terms | Term | 2 | |||||||
Lease renewal term under each extension option | 7 years | |||||||
2020 Boston Lease [Member] | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Lessee, operating lease, existence of option to extend | true | |||||||
Lessee, operating lease, option to extend | an option to extend the term of the lease for two additional five-year periods | |||||||
Lease expiration, month and year | 2034-03 | |||||||
Extended term of lease expiration | 5 years | |||||||
Number of additional renewal terms | Term | 2 | |||||||
Operating lease, right-of-use asset | $ 149,800 | |||||||
Operating lease liability | 147,900 | |||||||
Tenant incentives | $ 49,200 |
Leases - Summary of Lease Asset
Leases - Summary of Lease Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Assets | ||
Operating lease assets | $ 174,460 | $ 50,865 |
Total lease assets | 174,460 | 50,865 |
Liabilities | ||
Operating lease liabilities, current | 12,158 | 11,362 |
Operating lease liabilities, net of current portion | 212,872 | 50,067 |
Total lease liabilities | $ 225,030 | $ 61,429 |
Leases - Summary of Operating L
Leases - Summary of Operating Lease Costs Included in Research and Development and General and Administrative Expense, as well as Sublease Income (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Leases [Abstract] | |||
Operating lease costs | $ 22,520 | $ 14,342 | $ 8,067 |
Short-term lease costs | 11,087 | 7,339 | 4,554 |
Variable lease costs | 8,402 | 6,368 | 4,282 |
Sublease income | (5,253) | (587) | (525) |
Net lease cost | $ 36,756 | $ 27,462 | $ 16,378 |
Leases - Summary of Maturity of
Leases - Summary of Maturity of Undiscounted Payments Due under Lease Liabilities and Present Value of Those Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Leases [Abstract] | ||
2022 | $ 18,256 | |
2023 | 26,633 | |
2024 | 26,137 | |
2025 | 26,347 | |
2026 | 26,844 | |
Thereafter | 227,528 | |
Total | 351,745 | |
Present value adjustment | (126,715) | |
Present value of lease liabilities | $ 225,030 | $ 61,429 |
Leases - Summary of Lease Term
Leases - Summary of Lease Term and Discount Rate (Detail) | Dec. 31, 2021 | Dec. 31, 2020 |
Leases [Abstract] | ||
Operating leases, weighted-average remaining lease term (years) | 12 years 4 months 24 days | 9 years 1 month 6 days |
Operating leases, weighted-average discount rate | 5.90% | 9.30% |
Leases - Summary of Cash Paid f
Leases - Summary of Cash Paid for Amounts Included in Measurement of Lease Liabilities (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Cash paid for amounts included in measurement of lease liabilities: | |||
Operating cash flows used in operating leases | $ (19,753) | $ (13,161) | $ (8,420) |
Operating lease non-cash items: | |||
Right-of-use assets increased through lease modifications and reassessments | (14,230) | 3,169 | 826 |
Right-of-use assets obtained in exchange for operating lease liabilities | 152,486 | $ 13,956 | $ 18,088 |
Leasehold improvements paid directly by landlord | $ 30,500 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Contingencies And Commitments [Line Items] | |||
Cost sharing expensed relating to patent maintenance, defense and prosecution, incurred | $ 5.8 | $ 4.5 | $ 2.9 |
Cost sharing expensed relating to patent maintenance, defense and prosecution, accrued | 4 | $ 2.5 | |
2015 Collaboration Agreement [Member] | Vertex [Member] | |||
Contingencies And Commitments [Line Items] | |||
Milestone payment receivable | $ 395 |
Significant Contracts - Additio
Significant Contracts - Additional Information (Detail) | Apr. 16, 2021USD ($) | Dec. 13, 2019USD ($)Product | Oct. 26, 2015USD ($) | Dec. 31, 2021USD ($) | Oct. 31, 2019License | Dec. 31, 2019USD ($) | Dec. 31, 2021USD ($)Option | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Jul. 01, 2021 | Apr. 30, 2021 | Apr. 30, 2020USD ($) | Dec. 12, 2019 | Mar. 31, 2016 | Feb. 12, 2016 | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Non-current deferred revenue | $ 12,323,000 | $ 12,323,000 | $ 11,776,000 | |||||||||||||
Current deferred revenue | $ 1,011,000 | 1,011,000 | 2,341,000 | |||||||||||||
Research and development expense | [1] | $ 438,633,000 | 266,946,000 | $ 179,362,000 | ||||||||||||
Date of joint venture agreement | Dec. 19, 2015 | |||||||||||||||
Operating expenses | $ 541,435,000 | 355,154,000 | 242,850,000 | |||||||||||||
Stock-based compensation expense | 102,390,000 | 66,018,000 | 49,524,000 | |||||||||||||
Net loss of joint venture | 377,661,000 | (348,865,000) | 66,858,000 | |||||||||||||
Issuance of common shares, value | 222,175,000 | 973,381,000 | 414,789,000 | |||||||||||||
Proceeds from Issuance of Common Stock | 213,267,000 | 982,289,000 | 415,019,000 | |||||||||||||
Other (expense) income | $ 6,003,000 | 6,379,000 | 26,033,000 | |||||||||||||
Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Date of formation of joint venture entity | Feb. 12, 2016 | |||||||||||||||
Equity method investment, ownership percentage | 50.00% | 50.00% | 50.00% | 50.00% | 50.00% | |||||||||||
Percentage of partnership interests | 100.00% | |||||||||||||||
Partnership outstanding maximum exchange amount | $ 22,000,000 | |||||||||||||||
Operating expenses | $ 6,000,000 | |||||||||||||||
Number of products | Product | 2 | |||||||||||||||
Percentage of research and development costs | 50.00% | |||||||||||||||
Percentage sharing of profit (loss) from sale of product | 50.00% | |||||||||||||||
Non-refundable one-time option payment | $ 20,000,000 | |||||||||||||||
Equity method investment | $ 0 | $ 0 | ||||||||||||||
Stock-based compensation expense | 5,500,000 | |||||||||||||||
Unrecognized equity method losses in excess of Company's interest | 72,000,000 | |||||||||||||||
Business combination, consideration transferred | 41,000,000 | |||||||||||||||
Net assets acquired | 16,000,000 | 16,000,000 | ||||||||||||||
Fair value of assets and liabilities | 16,000,000 | 16,000,000 | ||||||||||||||
Payment to acquire business | $ 16,000,000 | |||||||||||||||
Number of options exercised under agreement | Option | 2 | |||||||||||||||
Casebia Therapeutics Limited Liability Partnership [Member] | Equity Method Investment, Nonconsolidated Investee or Group of Investees [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Net loss of joint venture | $ 58,800,000 | |||||||||||||||
Research and Development [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Stock-based compensation expense | $ 59,683,000 | 35,120,000 | 23,273,000 | |||||||||||||
Research and Development Services [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Fair value of obligations | 20,200,000 | 20,200,000 | ||||||||||||||
Future Delivery of up to Two Options [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Fair value of obligations | 4,800,000 | 4,800,000 | ||||||||||||||
A&R Vertex JDCA [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Percentage of net profits and net losses | 40.00% | 40.00% | ||||||||||||||
2019 Option Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Research and development expense | 7,000,000 | 13,200,000 | ||||||||||||||
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Business combination, consideration transferred | 25,000,000 | |||||||||||||||
Fair value of controlling interest obtained | 32,000,000 | |||||||||||||||
Payment to acquire business | 16,000,000 | |||||||||||||||
2019 Option Agreement [Member] | Research and Development [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Business combination, consideration transferred | 20,200,000 | |||||||||||||||
2019 Option Agreement [Member] | Certain Options [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Business combination, consideration transferred | 4,800,000 | |||||||||||||||
2019 Casebia Agreement [Member] | Other Current Liabilities [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Research obligation | 7,000,000 | |||||||||||||||
2019 Casebia Agreement [Member] | Other Long-term Liabilities [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Research obligation | 4,800,000 | 4,800,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Nonrefundable upfront payment received | $ 75,000,000 | $ 175,000,000 | ||||||||||||||
Percentage of exchange payment of research and development costs | 50.00% | |||||||||||||||
Number of options would not be exercised under agreement | Option | 1 | |||||||||||||||
Number of remaining options under agreement | Option | 4 | |||||||||||||||
Transaction price | $ 268,600,000 | |||||||||||||||
Non-current deferred revenue | 12,300,000 | 12,300,000 | 11,800,000 | |||||||||||||
Non-cash consideration received | 10,000,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 12,300,000 | 12,300,000 | ||||||||||||||
Revenue | 913,100,000 | 289,100,000 | ||||||||||||||
Current deferred revenue | 0 | 0 | 400,000 | |||||||||||||
Collaboration revenue | 30,000,000 | |||||||||||||||
Maximum potential future payments | 775,000,000 | |||||||||||||||
Research and development expense | 111,600,000 | 48,600,000 | 29,200,000 | |||||||||||||
Reimbursements from research and license agreements | 47,400,000 | $ 28,200,000 | 15,900,000 | |||||||||||||
Collaborative arrangement, license rights reacquired by waiving payment owed | 10,000,000 | |||||||||||||||
Non-cash consideration expensed upon execution of collaborative agreement | 10,000,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Research and Development [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Variable consideration received | 25,000,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Milestone payment receivable | 420,000,000 | 420,000,000 | ||||||||||||||
Milestone payment receivable | 410,000,000 | 410,000,000 | ||||||||||||||
Transaction price allocated to remaining performance obligations | 46,700,000 | 46,700,000 | ||||||||||||||
Revenue | 76,700,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 R&D Services [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 1,700,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 1,100,000 | 1,100,000 | ||||||||||||||
Revenue | 100,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 R&D Services [Member] | Research and Development [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Variable consideration received | 800,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DMD License [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 224,600,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 151,100,000 | 151,100,000 | ||||||||||||||
Revenue | 289,100,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 License [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 76,200,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 51,300,000 | 51,300,000 | ||||||||||||||
Revenue | 12,000,000 | 202,400,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Specified Target Option Material Right [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 17,500,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 11,800,000 | 11,800,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights One [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 25,000,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights Two [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 22,200,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights Three [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 22,200,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaborative Arrangement Material Rights Fourth Exclusive License [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 10,000,000 | |||||||||||||||
Transaction price allocated to remaining performance obligations | 6,700,000 | 6,700,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Exclusive License [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Revenue | 900,000,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Waiving of Vertex Material Right [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Revenue | 6,700,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Research and Development Services [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Revenue | $ 100,000 | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | 2015 Collaboration Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Milestone payment receivable | 395,000,000 | $ 395,000,000 | ||||||||||||||
Percentage of exchange payment of research and development costs | 50.00% | |||||||||||||||
Number of options would not be exercised under agreement | Option | 1 | |||||||||||||||
Number of remaining options under agreement | Option | 4 | |||||||||||||||
Non-current deferred revenue | 57,800,000 | $ 57,800,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | 2019 Collaboration Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Nonrefundable upfront payment received | 175,000,000 | |||||||||||||||
Variable consideration adjusted to transaction price | 12,500,000 | |||||||||||||||
Revenue recognized | 12,000,000 | |||||||||||||||
Maximum potential payments | 825,000,000 | $ 825,000,000 | ||||||||||||||
Percentage of exchange payment of research and development costs | 50.00% | |||||||||||||||
Transaction price allocated to remaining performance obligations | 500,000 | $ 500,000 | ||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Joint Development Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Up-front payment received | $ 7,000,000 | |||||||||||||||
Agreement description | In connection with the JDA, the Company received a $7.0 million up-front payment from Vertex and subsequently received a one-time low seven-digit milestone payment upon the dosing of the second patient in a clinical trial with the initial product candidate. | |||||||||||||||
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Exercise of Exclusive Option [Member] | Collaboration Target Options [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Milestone payment receivable | 10,000,000 | $ 10,000,000 | ||||||||||||||
Vertex Pharmaceuticals Inc [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Maximum potential future payments | 200,000,000 | |||||||||||||||
Vertex Pharmaceuticals Inc [Member] | Exclusive License [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Estimated standalone selling price for single collaboration | 900,000,000 | |||||||||||||||
Vertex Pharmaceuticals Inc [Member] | A&R Vertex JDCA [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Up-front payment received | $ 900,000,000 | $ 900,000,000 | ||||||||||||||
Milestone payment receivable | $ 200,000,000 | |||||||||||||||
Percentage of net profits and net losses | 60.00% | 60.00% | ||||||||||||||
Additional interest of percentage | 10.00% | |||||||||||||||
Vertex Pharmaceuticals Inc [Member] | 2015 Collaboration Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Aggregate amount in option exercise payments received | $ 30,000,000 | |||||||||||||||
Number of exclusive license targets | License | 3 | |||||||||||||||
Vertex Pharmaceuticals Inc [Member] | 2019 Collaboration Agreement [Member] | ||||||||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||||||||
Milestone payment receivable | 12,500,000 | $ 12,500,000 | $ 25,000,000 | |||||||||||||
Remaining milestone payment receivable | $ 775,000,000 | $ 775,000,000 | ||||||||||||||
[1] | Including the following amounts of research and development from a related party, see Note 9 |
Significant Contracts - Schedul
Significant Contracts - Schedule of Fair Value of Assets and Liabilities Received (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Research and Development [Abstract] | |
Cash and cash equivalents | $ 6,784 |
Prepaid expenses and other current assets | 2,565 |
Property, plant and equipment, net | 9,340 |
Operating lease assets | 11,003 |
Restricted cash | 1,226 |
Accrued expenses and other current liabilities | (3,915) |
Operating lease liabilities | (11,003) |
Net assets | $ 16,000 |
Share Capital - Additional Info
Share Capital - Additional Information (Detail) | 1 Months Ended | 12 Months Ended | |||||||||||
Jan. 31, 2021USD ($)$ / sharesshares | Dec. 31, 2020USD ($) | Jul. 31, 2020USD ($)$ / sharesshares | Nov. 30, 2019USD ($)$ / sharesshares | Aug. 31, 2019USD ($) | Aug. 31, 2018USD ($) | Dec. 31, 2021USD ($)$ / sharesshares | Dec. 31, 2020USD ($)$ / sharesshares | Dec. 31, 2019USD ($)$ / sharesshares | Dec. 31, 2021SFr / shares | Dec. 31, 2021USD ($)shares | Dec. 31, 2020SFr / shares | Dec. 31, 2020USD ($)shares | |
Class of Stock [Line Items] | |||||||||||||
Common stock, shares authorized | shares | 145,364,335 | 115,172,786 | |||||||||||
Common shares, par value | SFr / shares | SFr 0.03 | SFr 0.03 | |||||||||||
Capital reserved for future issuance | shares | 5,038,262 | ||||||||||||
Proceeds from issuance of common shares | $ 213,267,000 | $ 982,289,000 | $ 415,019,000 | ||||||||||
Common Stock, Voting Rights | one vote for each common share | ||||||||||||
Dividends declared and paid | $ 0 | ||||||||||||
Underwritten Public Offering [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Common shares sold | shares | 7,400,000 | 4,900,000 | |||||||||||
Common shares price per share | $ / shares | $ 70 | $ 64.50 | |||||||||||
Proceeds from issuance of common shares | $ 484,800,000 | $ 297,400,000 | |||||||||||
Equity issuance costs | $ 32,500,000 | $ 17,800,000 | |||||||||||
Payments of stamp taxes related to securities | $ 3,000,000 | ||||||||||||
2018 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Common shares sold | shares | 2,800,000 | ||||||||||||
Proceeds from issuance of common shares | $ 120,600,000 | ||||||||||||
Equity issuance costs | 4,400,000 | ||||||||||||
Payments of stamp taxes related to securities | $ 300,000 | $ 900,000 | |||||||||||
Average issue price of common shares | $ / shares | $ 44.38 | ||||||||||||
Accrued stamp taxes | $ 300,000 | ||||||||||||
2018 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | Maximum [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Aggregate estimated gross proceeds | $ 125,000,000 | ||||||||||||
2019 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Common shares sold | shares | 2,200,000 | ||||||||||||
Proceeds from issuance of common shares | $ 195,500,000 | ||||||||||||
Equity issuance costs | $ 4,500,000 | ||||||||||||
Average issue price of common shares | $ / shares | $ 89.47 | ||||||||||||
2019 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | Maximum [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Aggregate estimated gross proceeds | $ 200,000,000 | ||||||||||||
2020 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Common shares sold | shares | 300,000 | 1,800,000 | |||||||||||
Proceeds from issuance of common shares | $ 46,700,000 | $ 298,000,000 | |||||||||||
Equity issuance costs | $ 700,000 | 4,500,000 | |||||||||||
Payments of stamp taxes related to securities | $ 500,000 | $ 4,900,000 | |||||||||||
Payable of stamp taxes related to securities | $ 4,000,000 | $ 4,000,000 | |||||||||||
Average issue price of common shares | $ / shares | $ 162.46 | $ 169.57 | |||||||||||
2020 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | Maximum [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Aggregate estimated gross proceeds | $ 350,000 | ||||||||||||
2021 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Common shares sold | shares | 1,100,000 | ||||||||||||
Proceeds from issuance of common shares | $ 177,800,000 | ||||||||||||
Equity issuance costs | 2,400,000 | ||||||||||||
Payments of stamp taxes related to securities | $ 1,800,000 | ||||||||||||
Average issue price of common shares | $ / shares | $ 169.82 | ||||||||||||
2021 ATM [Member] | Sales Agreement With Jefferies LLC [Member] | Maximum [Member] | |||||||||||||
Class of Stock [Line Items] | |||||||||||||
Aggregate estimated gross proceeds | $ 600,000 |
Share Capital - Schedule of Sha
Share Capital - Schedule of Share Capital (Detail) - shares | Dec. 31, 2021 | Dec. 31, 2020 |
Class of Stock [Line Items] | ||
Registered share capital | 80,321,227 | 75,133,951 |
Authorized share capital | 145,364,335 | 115,172,786 |
Employee Benefit Plans [Member] | ||
Class of Stock [Line Items] | ||
Conditional share capital - Bonds or similar debt instruments | 20,806,433 | 17,493,709 |
Bonds or Similar Debt Instruments [Member] | ||
Class of Stock [Line Items] | ||
Conditional share capital - Bonds or similar debt instruments | 4,919,700 | 4,919,700 |
Swiss Law [Member] | ||
Class of Stock [Line Items] | ||
Authorized share capital | 39,316,975 | 17,625,426 |
Equity-based Compensation - Opt
Equity-based Compensation - Option and Grant Plans - Additional Information (Detail) - shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Share-based payment award, number of shares granted | 1,616,255 | 2,182,773 | 2,832,784 |
Minimum [Member] | Restricted Stock Unit [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Share-based compensation, vesting period | 2 years | ||
Maximum [Member] | Restricted Stock Unit [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Share-based compensation, vesting period | 3 years | ||
Option and Grant Plans [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Share-based compensation, vesting period | 4 years | ||
Share-based compensation, options, contractual life | 10 years | ||
Option and Grant Plans [Member] | IPO [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Share-based payment award, number of shares granted | 0 | ||
2018 Plan [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of authorized for issuance | 25,005,365 | ||
Number of shares available for future grant | 10,298,664 |
Equity-based Compensation - Equ
Equity-based Compensation - Equity-Based Compensation Expense - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 102,390 | $ 66,018 | $ 49,524 |
Total unrecognized compensation expense related to stock options | 150,600 | ||
Restricted Stock Units [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total unrecognized compensation expense | $ 71,400 | ||
Unrecognized compensation expense, remaining weighted-average period for recognition | 2 years 6 months | ||
Stock Options [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Unrecognized compensation expense, remaining weighted-average period for recognition | 2 years 8 months 12 days |
Equity-based Compensation - Sch
Equity-based Compensation - Schedule of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Stock-based compensation expense | $ 102,390 | $ 66,018 | $ 49,524 |
Research and Development [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Stock-based compensation expense | 59,683 | 35,120 | 23,273 |
General and Administrative [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Stock-based compensation expense | $ 42,707 | $ 30,898 | 20,784 |
Loss from Equity Method Investment [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Stock-based compensation expense | $ 5,467 |
Equity-based Compensation - Fai
Equity-based Compensation - Fair Value of Each Option Issued to Employees and Non-employees Estimated Using Black-Scholes Option-Pricing Model (Detail) - $ / shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Payment Arrangement [Abstract] | |||
Options granted | 1,616,255 | 2,182,773 | 2,832,784 |
Weighted-average exercise price | $ 124.32 | $ 68.91 | $ 39.16 |
Weighted-average grant date fair value | $ 77.38 | $ 42.28 | $ 24.57 |
Expected volatility | 70.30% | 69.20% | 68.90% |
Expected term (in years) | 6 years | 6 years | 6 years |
Risk-free interest rate | 1.00% | 0.60% | 2.20% |
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Equity-based Compensation - Sum
Equity-based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | |||
Shares, Outstanding, Beginning Balance | 8,101,980 | ||
Options granted | 1,616,255 | 2,182,773 | 2,832,784 |
Shares, Exercised | (1,245,071) | ||
Shares, Cancelled or forfeited | (660,182) | ||
Shares, Outstanding, Ending Balance | 7,812,982 | 8,101,980 | |
Shares, Exercisable | 4,598,353 | ||
Shares, Vested or expected to vest | 7,812,982 | ||
Weighted-Average Exercise Price, Outstanding, Beginning Balance | $ 42.44 | ||
Weighted-Average Exercise Price, Granted | 124.32 | $ 68.91 | $ 39.16 |
Weighted-Average Exercise Price, Exercised | 30.96 | ||
Weighted-Average Exercise Price, Cancelled or forfeited | 79.54 | ||
Weighted-Average Exercise Price, Outstanding, Ending Balance | 58.07 | $ 42.44 | |
Weighted-Average Exercise Price, Exercisable | 40.86 | ||
Weighted-Average Exercise Price, Vested or expected to vest | $ 58.07 | ||
Weighted-Average Remaining Contractual Term | 7 years 4 months 24 days | 7 years 9 months 18 days | |
Weighted-Average Remaining Contractual Term, Exercisable | 6 years 7 months 6 days | ||
Weighted-Average Remaining Contractual Term, Vested or expected to vest | 7 years 4 months 24 days | ||
Aggregate Intrinsic Value, Outstanding Balance | $ 219,103 | $ 896,666 | |
Aggregate Intrinsic Value, Exercisable | 174,056 | ||
Aggregate Intrinsic Value, Vested or expected to vest | $ 219,103 |
Equity-based Compensation - Sto
Equity-based Compensation - Stock Options - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation, options, outstanding | 7,812,982 | 8,101,980 | ||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Grants in Period, Gross | 1,616,255 | 2,182,773 | 2,832,784 | |
Total intrinsic value of stock options exercised | $ 119.5 | $ 104.2 | $ 42.2 | |
Performance-Based [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Grants in Period, Gross | 0 | 0 | ||
Performance-Based [Member] | Vesting Percentage on First Anniversary [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation, options, vested | 998,504 | |||
Share-based compensation, options, outstanding | 123,057 | |||
Market-Based [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation, options, vested | 150,000 | |||
Share-based Compensation Arrangement by Share-based Payment Award, Options, Grants in Period, Gross | 0 | 0 | 150,000 | |
Market-Based [Member] | Vesting Percentage on First Anniversary [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation, requisite service period | 3 years | |||
Share-based compensation, vesting percentage | 75.00% | |||
Market-Based [Member] | Vesting Percentage on Second Anniversary [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share-based compensation, requisite service period | 4 years | |||
Share-based compensation, vesting percentage | 25.00% |
Equity-based Compensation - S_2
Equity-based Compensation - Summary of Restricted Stock Activity (Detail) - Restricted Stock [Member] | 12 Months Ended |
Dec. 31, 2021$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Unvested, Shares, Beginning Balance | shares | 894,092 |
Unvested, Shares, Granted | shares | 628,175 |
Unvested, Shares, Vested | shares | (455,440) |
Unvested, Shares, Cancelled or forfeited | shares | (132,652) |
Unvested, Shares, Ending Balance | shares | 934,175 |
Unvested, Weighted-Average Grant Date Fair Value, Beginning Balance | $ / shares | $ 70.55 |
Unvested, Weighted-Average Grant Date Fair Value, Granted | $ / shares | 111.73 |
Unvested, Weighted-Average Grant Date Fair Value, Vested | $ / shares | 58 |
Unvested, Weighted-Average Grant Date Fair Value, Cancelled or forfeited | $ / shares | 100.25 |
Unvested, Weighted-Average Grant Date Fair Value, Ending Balance | $ / shares | $ 100.14 |
Equity-based Compensation - Res
Equity-based Compensation - Restricted Stock - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 102,390 | $ 66,018 | $ 49,524 |
Restricted Stock [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Total fair value of restricted stock vested | $ 45,300 | $ 21,600 | $ 3,600 |
Equity-based Compensation - Emp
Equity-based Compensation - Employee Stock Purchase Plan - Additional Information (Detail) - 2016 Employee Stock Purchase Plan [Member] - shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Oct. 19, 2016 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of shares issued | 21,590 | 13,410 | |
Maximum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of shares authorized | 400,000 |
Net Income (Loss) Per Share A_3
Net Income (Loss) Per Share Attributable to Common Shareholders - Schedule of Computation of Basic and Diluted Net Income (Loss) per Share (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Net income (loss) | $ 377,661 | $ (348,865) | $ 66,858 |
Basic weighted-average common shares outstanding | 75,948,686 | 65,949,672 | 54,392,304 |
Effect of potentially dilutive securities: | |||
Diluted weighted-average common shares outstanding | 80,393,496 | 65,949,672 | 56,932,798 |
Net income (loss) per common share - basic | $ 4.97 | $ (5.29) | $ 1.23 |
Net income (loss) per common share - diluted | $ 4.70 | $ (5.29) | $ 1.17 |
Outstanding Options [Member] | |||
Effect of potentially dilutive securities: | |||
Effect of potentially dilutive securities | 3,990,579 | 2,406,962 | |
Unvested Restricted Common Shares [Member] | |||
Effect of potentially dilutive securities: | |||
Effect of potentially dilutive securities | 454,231 | 133,532 |
Net Income (Loss) Per Share A_4
Net Income (Loss) Per Share Attributable to Common Shareholders - Schedule of Antidilutive Securities not Include in Computation of Net Income (Loss) per Share (Detail) - shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Anti-dilutive securities not include in computation of net income (loss) per share | 1,998,456 | 9,007,329 | 3,897,754 |
Outstanding Options [Member] | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Anti-dilutive securities not include in computation of net income (loss) per share | 1,765,881 | 8,101,980 | 3,789,129 |
Unvested Restricted Common Shares [Member] | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Anti-dilutive securities not include in computation of net income (loss) per share | 225,904 | 894,092 | 108,625 |
ESPP [Member] | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Anti-dilutive securities not include in computation of net income (loss) per share | 6,671 | 11,257 |
401(k) Savings Plan - Additiona
401(k) Savings Plan - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Retirement Benefits [Abstract] | |||
Company contributions toward the plan | $ 3.4 | $ 1.9 | $ 1.1 |
Income Taxes - Schedule of Inco
Income Taxes - Schedule of Income (Loss) Before Provision for Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ 4,569 | $ 7,630 | $ 9,155 |
Foreign | 374,962 | (355,686) | 58,151 |
Net income (loss) before income taxes | $ 379,531 | $ (348,056) | $ 67,306 |
Income Taxes - Schedule of (Pro
Income Taxes - Schedule of (Provision for) Benefit from Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Current income taxes: | |||
Federal | $ (80) | $ (248) | $ (423) |
State | (42) | (151) | (59) |
Foreign | (1) | 0 | |
Total current income taxes | (122) | (400) | (482) |
Deferred income taxes: | |||
Federal | (1,748) | (409) | 34 |
Total deferred income taxes | (1,748) | (409) | 34 |
Total income tax (provision) benefit | $ (1,870) | $ (809) | $ (448) |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense Computed at Statutory Corporate Income Tax Rate to Effective Income Tax Rate (Detail) | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Reconciliation of Effective Income Tax Rate | |||
Income tax expense at statutory rate | 11.90% | 11.90% | 9.30% |
State income tax, net of federal benefit | (1.00%) | 1.00% | (2.10%) |
Nondeductible expenses | 0.70% | 0.10% | (0.10%) |
Foreign rate differential | 0.60% | (0.10%) | 2.00% |
Statutory to US GAAP permanent differences | 0.00% | 0.00% | 0.10% |
Stock-based compensation | (2.50%) | 2.30% | (2.00%) |
Impact of deferred rate change | 0.00% | 0.00% | (12.20%) |
Research credits | (4.20%) | 3.30% | (5.20%) |
Change in valuation allowance | (5.00%) | (18.70%) | 10.90% |
Effective income tax rate | 0.50% | (0.20%) | 0.70% |
Income Taxes - Schedule of Sign
Income Taxes - Schedule of Significant Components of Company's Deferred Tax Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 47,190 | $ 84,531 |
Accruals and reserves | 5,878 | 4,785 |
Operating lease liabilities | 61,476 | 16,782 |
Other deferred tax assets | 6,362 | 1,517 |
Stock-based compensation | 14,042 | 9,605 |
Deferred revenue | 86 | |
Research credit | 37,878 | 19,526 |
Total deferred tax assets | 172,826 | 136,832 |
Less valuation allowance | (98,649) | (116,640) |
Net deferred tax assets | 74,177 | 20,192 |
Deferred tax liabilities: | ||
Depreciation | (28,579) | (6,778) |
Operating lease assets | (47,521) | (13,776) |
Intangible assets | (31) | (31) |
Other deferred tax liabilities | (192) | (4) |
Total deferred tax liabilities | (76,323) | (20,589) |
Long term deferred taxes, liabilities | $ (2,146) | $ (397) |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Valuation Allowance [Line Items] | ||||
Decrease in valuation allowance | $ 18,000,000 | |||
U.S. federal net operating loss carryforwards | 3,300,000 | |||
Non-US Net operating loss carryforwards | $ 772,900,000 | |||
Operating loss carryforwards expiration period | 2027 | |||
Unrecognized tax benefits, gross | $ 21,395,000 | $ 11,967,000 | $ 5,231,000 | $ 1,595,000 |
Unrecognized tax benefits would favorably impact the effective tax rate if recognized | 19,900,000 | |||
Accrued interest or penalties related to uncertain tax positions | 0 | 0 | 0 | |
Uncertain tax positions recognized | 0 | $ 0 | $ 0 | |
U.S. Domestic Federal [Member] | Research and Development [Member] | ||||
Valuation Allowance [Line Items] | ||||
Tax credit carryforward amount | $ 19,400,000 | |||
Tax credit carryforward, expiration year | 2039 | |||
Uncertain tax positions | $ 9,900,000 | |||
U.S. Domestic Federal [Member] | Orphan Drug Credit Carryforwards [Member] | ||||
Valuation Allowance [Line Items] | ||||
Tax credit carryforward amount | $ 10,600,000 | |||
Tax credit carryforward, expiration year | 2040 | |||
Uncertain tax positions | $ 4,500,000 | |||
U.S. Domestic State [Member] | Research and Development [Member] | ||||
Valuation Allowance [Line Items] | ||||
Tax credit carryforward amount | $ 10,000,000 | |||
Tax credit carryforward, expiration year | 2035 | |||
Uncertain tax positions | $ 7,000,000 | |||
Switzerland [Member] | ||||
Valuation Allowance [Line Items] | ||||
Non-US Net operating loss carryforwards | 385,200,000 | |||
Canton of Zug [Member] | ||||
Valuation Allowance [Line Items] | ||||
Non-US Net operating loss carryforwards | 385,200,000 | |||
United Kingdom [Member] | ||||
Valuation Allowance [Line Items] | ||||
Non-US Net operating loss carryforwards | $ 2,500,000 |
Income Taxes - Schedule of Aggr
Income Taxes - Schedule of Aggregate Changes in Gross Unrecognized Tax Benefits (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |||
Balance at beginning of year | $ 11,967 | $ 5,231 | $ 1,595 |
Increases for tax positions taken during current period | 9,911 | 7,004 | 2,754 |
Increases for tax positions taken in prior periods | 882 | ||
Decreases for tax positions taken in prior periods | (483) | (268) | |
Balance at end of year | $ 21,395 | $ 11,967 | $ 5,231 |
Selected Quarterly Financial Da
Selected Quarterly Financial Data (Unaudited) - Summary of Quarterly Financial Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |||
Total revenue | $ 914,963 | $ 719 | $ 289,590 |
Operating Expenses | 541,435 | 355,154 | 242,850 |
Loss from operations | 373,528 | (354,435) | 46,740 |
Net (loss) income | $ 377,661 | $ (348,865) | $ 66,858 |
Net (loss) income per common share: | |||
Basic net (loss) income per common share | $ 4.97 | $ (5.29) | $ 1.23 |
Diluted net (loss) income per common share | $ 4.70 | $ (5.29) | $ 1.17 |
Weighted-average common shares outstanding: | |||
Basic | 75,948,686 | 65,949,672 | 54,392,304 |
Diluted | 80,393,496 | 65,949,672 | 56,932,798 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) | 3 Months Ended |
Dec. 31, 2018 | |
Collaboration Agreement [Member] | Vertex Pharmaceuticals Inc [Member] | Minimum [Member] | |
Related Party Transaction [Line Items] | |
Voting interest Percentage | 10.00% |