Cover
Cover - shares | 9 Months Ended | |
Sep. 30, 2022 | Oct. 31, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2022 | |
Document Transition Report | false | |
Entity File Number | 000-30171 | |
Entity Registrant Name | SANGAMO THERAPEUTICS, INC. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 68-0359556 | |
Entity Address, Address Line One | 7000 Marina Blvd. | |
Entity Address, City or Town | Brisbane | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 94005 | |
City Area Code | 510 | |
Local Phone Number | 970-6000 | |
Title of 12(b) Security | Common Stock, par value $0.01 per share | |
Trading Symbol | SGMO | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 163,880,007 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q3 | |
Entity Central Index Key | 0001001233 | |
Current Fiscal Year End Date | --12-31 |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 94,570 | $ 178,872 |
Marketable securities | 218,998 | 197,676 |
Interest receivable | 524 | 349 |
Accounts receivable | 4,522 | 6,013 |
Prepaid expenses and other current assets | 18,007 | 15,859 |
Total current assets | 336,621 | 398,769 |
Marketable securities, non-current | 36,700 | 88,169 |
Property and equipment, net | 55,986 | 51,523 |
Intangible assets | 46,439 | 53,760 |
Goodwill | 34,511 | 39,702 |
Operating lease right-of-use assets | 64,956 | 73,181 |
Other non-current assets | 17,200 | 15,319 |
Restricted cash | 1,500 | 1,500 |
Total assets | 593,913 | 721,923 |
Current liabilities: | ||
Accounts payable | 17,380 | 9,759 |
Accrued compensation and employee benefits | 19,045 | 20,840 |
Other accrued liabilities | 14,046 | 11,577 |
Deferred revenues | 71,890 | 85,711 |
Total current liabilities | 122,361 | 127,887 |
Deferred revenues, non-current | 115,641 | 166,776 |
Long-term portion of lease liabilities | 39,705 | 44,055 |
Deferred income tax | 5,740 | 6,645 |
Other non-current liabilities | 1,312 | 1,217 |
Total liabilities | 284,759 | 346,580 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Preferred stock | 0 | 0 |
Common stock | 1,619 | 1,459 |
Additional paid-in capital | 1,423,105 | 1,334,138 |
Accumulated deficit | (1,096,572) | (956,267) |
Accumulated other comprehensive loss | (18,998) | (3,987) |
Total stockholders’ equity | 309,154 | 375,343 |
Total liabilities and stockholders’ equity | $ 593,913 | $ 721,923 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) shares in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Income Statement [Abstract] | ||||
Revenues | $ 26,460,000 | $ 28,563,000 | $ 84,069,000 | $ 82,715,000 |
Operating expenses: | ||||
Research and development | 65,116,000 | 62,498,000 | 183,719,000 | 179,018,000 |
General and administrative | 16,238,000 | 14,501,000 | 46,239,000 | 47,135,000 |
Total operating expenses | 81,354,000 | 76,999,000 | 229,958,000 | 226,153,000 |
Loss from operations | (54,894,000) | (48,436,000) | (145,889,000) | (143,438,000) |
Interest and other income, net | 1,769,000 | 834,000 | 5,754,000 | 3,010,000 |
Loss before income taxes | (53,125,000) | (47,602,000) | (140,135,000) | (140,428,000) |
Income tax expense | 30,000 | 86,000 | 170,000 | 373,000 |
Net loss | (53,155,000) | (47,688,000) | (140,305,000) | (140,801,000) |
Net loss attributable to non-controlling interest | 0 | 0 | 0 | (11,000) |
Net loss attributable to Sangamo Therapeutics, Inc. stockholders, basic | (53,155,000) | (47,688,000) | (140,305,000) | (140,790,000) |
Net loss attributable to Sangamo Therapeutics, Inc. stockholders, diluted | $ (53,155,000) | $ (47,688,000) | $ (140,305,000) | $ (140,790,000) |
Earnings per share, basic (in dollars per share) | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) |
Earnings per share, diluted (in dollars per share) | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) |
Shares used in computing basic net loss per share attributable to Sangamo Therapeutics, Inc. stockholders (in shares) | 158,042 | 145,399 | 150,850 | 144,173 |
Shares used in computing diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders (in shares) | 158,042 | 145,399 | 150,850 | 144,173 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (53,155) | $ (47,688) | $ (140,305) | $ (140,801) |
Foreign currency translation adjustment | (6,028) | (2,565) | (14,040) | (6,006) |
Net pension gains | 55 | 0 | 130 | 0 |
Unrealized gain (loss) on marketable securities, net of tax | 135 | (45) | (1,101) | (36) |
Comprehensive loss | (58,993) | (50,298) | (155,316) | (146,843) |
Comprehensive loss attributable to non-controlling interest | 0 | 0 | 0 | (11) |
Comprehensive loss attributable to Sangamo Therapeutics, Inc. | $ (58,993) | $ (50,298) | $ (155,316) | $ (146,832) |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive (Loss) Income | Non- Controlling Interest |
Beginning balance (in shares) at Dec. 31, 2020 | 142,063,203 | |||||
Beginning balance at Dec. 31, 2020 | $ 497,366 | $ 1,421 | $ 1,269,375 | $ (777,981) | $ 5,419 | $ (868) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares) | 2,007,932 | |||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses | 27,099 | $ 20 | 27,079 | |||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares) | 1,200,871 | |||||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax | 1,594 | $ 12 | 1,582 | |||
Issuance of common stock under employee stock purchase plan (in shares) | 244,570 | |||||
Issuance of common stock under employee stock purchase plan | 2,054 | $ 2 | 2,052 | |||
Stock-based compensation | 24,880 | 24,880 | ||||
Acquisition of additional shares of Sangamo France | (134) | (70) | (64) | |||
Foreign currency translation adjustment | (6,006) | (6,006) | ||||
Net pension gains | 0 | |||||
Unrealized gain (loss) on marketable securities, net of tax | (36) | (36) | ||||
Buy-out of non-controlling interest | (943) | (943) | 943 | |||
Net loss | (140,801) | (140,790) | (11) | |||
Ending balance (in shares) at Sep. 30, 2021 | 145,516,576 | |||||
Ending balance at Sep. 30, 2021 | 406,016 | $ 1,455 | 1,323,955 | (918,771) | (623) | 0 |
Beginning balance (in shares) at Jun. 30, 2021 | 145,106,901 | |||||
Beginning balance at Jun. 30, 2021 | 445,457 | $ 1,451 | 1,313,102 | (871,083) | 1,987 | 0 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares) | 202,705 | |||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses | 2,367 | $ 2 | 2,365 | |||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares) | 206,970 | |||||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax | 617 | $ 2 | 615 | |||
Stock-based compensation | 7,873 | 7,873 | ||||
Foreign currency translation adjustment | (2,565) | (2,565) | ||||
Net pension gains | 0 | |||||
Unrealized gain (loss) on marketable securities, net of tax | (45) | (45) | ||||
Net loss | (47,688) | (47,688) | 0 | |||
Ending balance (in shares) at Sep. 30, 2021 | 145,516,576 | |||||
Ending balance at Sep. 30, 2021 | 406,016 | $ 1,455 | 1,323,955 | (918,771) | (623) | $ 0 |
Beginning balance (in shares) at Dec. 31, 2021 | 145,921,530 | |||||
Beginning balance at Dec. 31, 2021 | 375,343 | $ 1,459 | 1,334,138 | (956,267) | (3,987) | |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares) | 14,711,770 | |||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses | 66,448 | $ 147 | 66,301 | |||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares) | 925,455 | |||||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax | (1,838) | $ 9 | (1,847) | |||
Issuance of common stock under employee stock purchase plan (in shares) | 359,468 | |||||
Issuance of common stock under employee stock purchase plan | 1,115 | $ 4 | 1,111 | |||
Stock-based compensation | 23,402 | 23,402 | ||||
Foreign currency translation adjustment | (14,040) | (14,040) | ||||
Net pension gains | 130 | 130 | ||||
Unrealized gain (loss) on marketable securities, net of tax | (1,101) | (1,101) | ||||
Buy-out of non-controlling interest | 0 | |||||
Net loss | (140,305) | (140,305) | ||||
Ending balance (in shares) at Sep. 30, 2022 | 161,918,223 | |||||
Ending balance at Sep. 30, 2022 | 309,154 | $ 1,619 | 1,423,105 | (1,096,572) | (18,998) | |
Beginning balance (in shares) at Jun. 30, 2022 | 153,352,502 | |||||
Beginning balance at Jun. 30, 2022 | 318,281 | $ 1,534 | 1,373,324 | (1,043,417) | (13,160) | |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares) | 8,483,104 | |||||
Issuance of common stock in connection with at-the-market offering, net of offering expenses | 42,174 | $ 85 | 42,089 | |||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares) | 82,617 | |||||
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax | (101) | (101) | ||||
Stock-based compensation | 7,793 | 7,793 | ||||
Foreign currency translation adjustment | (6,028) | (6,028) | ||||
Net pension gains | 55 | 55 | ||||
Unrealized gain (loss) on marketable securities, net of tax | 135 | 135 | ||||
Net loss | (53,155) | (53,155) | ||||
Ending balance (in shares) at Sep. 30, 2022 | 161,918,223 | |||||
Ending balance at Sep. 30, 2022 | $ 309,154 | $ 1,619 | $ 1,423,105 | $ (1,096,572) | $ (18,998) |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Operating Activities: | ||
Net loss | $ (140,305) | $ (140,801) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 8,765 | 6,675 |
Amortization of (discount) premium on marketable securities | (324) | 2,394 |
Amortization and other changes in operating lease right-of-use assets | 6,357 | 6,104 |
Gain on free shares | 0 | (18) |
Stock-based compensation | 23,402 | 24,880 |
Gain on disposal of property and equipment | 0 | (30) |
Net changes in operating assets and liabilities: | ||
Interest receivable | (175) | 415 |
Accounts receivable | 1,491 | (2,743) |
Prepaid expenses and other assets | (5,848) | (6,467) |
Accounts payable and other accrued liabilities | 9,443 | (4,699) |
Accrued compensation and employee benefits | (1,225) | (1,932) |
Deferred revenues | (64,956) | (62,292) |
Lease liabilities | (3,298) | (3,227) |
Other non-current liabilities | 95 | 1,204 |
Net cash used in operating activities | (166,578) | (180,537) |
Investing Activities: | ||
Purchases of marketable securities | (225,621) | (300,387) |
Maturities of marketable securities | 254,990 | 509,620 |
Sales of marketable securities | 0 | 6,870 |
Purchases of property and equipment | (12,697) | (20,420) |
Purchase of additional Sangamo France shares | 0 | (119) |
Net cash provided by investing activities | 16,672 | 195,564 |
Financing Activities: | ||
Taxes paid related to net share settlement of equity awards | (1,962) | (2,988) |
Proceeds from exercise of stock options | 124 | 4,582 |
Net cash provided by financing activities | 65,125 | 30,746 |
Effect of exchange rate changes on cash, cash equivalents, and restricted cash | 479 | (148) |
Net (decrease) increase in cash, cash equivalents, and restricted cash | (84,302) | 45,625 |
Cash, cash equivalents, and restricted cash, beginning of period | 180,372 | 132,829 |
Cash, cash equivalents, and restricted cash, end of period | 96,070 | 178,454 |
Supplemental cash flow disclosures: | ||
Property and equipment included in unpaid liabilities | 3,927 | 650 |
Right-of-use assets obtained in exchange for lease obligations | 0 | 1,349 |
Tenant improvement allowance included in contra-lease liability | 1,531 | 0 |
Buy-out of non-controlling interest | 0 | 943 |
At the Market Offering | ||
Financing Activities: | ||
Proceeds from at-the market offering/ issuance of common stock | 65,848 | 27,099 |
Employee Stock | ||
Financing Activities: | ||
Proceeds from at-the market offering/ issuance of common stock | $ 1,115 | $ 2,053 |
ORGANIZATION, BASIS OF PRESENTA
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | ORGANIZATION, BASIS OF PRESENTATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Organization and Description of Business Sangamo Therapeutics, Inc. (“Sangamo” or “the Company”) was incorporated in the State of Delaware in June 1995 and changed its name from Sangamo Biosciences, Inc. in January 2017. Sangamo is a clinical-stage genomic medicine company committed to translating ground-breaking science into medicines that transform the lives of patients with serious diseases. Basis of Presentation The accompanying unaudited Condensed Consolidated Financial Statements have been prepared in accordance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) for interim financial information and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Accordingly, they do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. In the opinion of management, all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation of these financial statements for the periods presented have been included. Operating results for the three and nine months ended September 30, 2022 are not necessarily indicative of the results that may be expected for the year ending December 31, 2022. The Condensed Consolidated Balance Sheet data at December 31, 2021 was derived from the audited Consolidated Financial Statements included in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2021 (the “2021 Annual Report”) as filed with the SEC on February 24, 2022. The accompanying Condensed Consolidated Financial Statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in the Condensed Consolidated Financial Statements. For consolidated entities where the Company owns or is exposed to less than 100% of the economics, the Company records net loss attributable to non-controlling interests on its Condensed Consolidated Statements of Operations equal to the percentage of the economic or ownership interest retained in such entities by the respective non-controlling parties. The accompanying Condensed Consolidated Financial Statements and related financial information should be read together with the audited Consolidated Financial Statements and footnotes for the year ended December 31, 2021, included in the 2021 Annual Report. Liquidity and Management’s Plan Sangamo is currently working on a number of long-term development projects that involve experimental technologies. The projects may require several years and substantial expenditures to complete and ultimately may be unsuccessful. The Company plans to finance operations with available cash resources, collaborations and strategic partnerships funds, research grants and from the issuance of equity or debt securities. Sangamo believes that its available cash, cash equivalents, and marketable securities as of September 30, 2022, and expected future milestones and research services revenue from collaborations, strategic partnerships and research grants, will be adequate to fund its currently planned operations through at least the next 12 months from the date these Condensed Consolidated Financial Statements are issued. Sangamo will require substantial additional financial resources to complete the development and commercialization of its product candidates. Additional capital may not be available on terms acceptable to the Company, if at all. If adequate funds are not available, or if the terms of potential funding sources are unfavorable, the Company’s business and ability to develop its technology and therapeutic products would be harmed. Furthermore, any sales of additional equity securities may result in dilution to the Company’s stockholders, and any debt financing may include covenants that restrict the Company’s business. Summary of Significant Accounting Policies Use of Estimates The preparation of Condensed Consolidated Financial Statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the Condensed Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates including critical accounting policies or estimates related to revenue recognition, clinical trial accruals, income taxes, fair value of assets and liabilities, including from acquisitions, and stock-based compensation. Estimates are based on historical experience and on various other market specific and other relevant assumptions that the Company believes to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates. In September 2021, the Company recorded an adjustment to revenue related to a change in estimate in connection with the collaboration agreement with Sanofi S.A. (“Sanofi”) as a result of an increase in the project costs for its beta thalassemia program and the increase in project scope and the corresponding costs for its sickle cell disease program, both of which resulted in a decrease in the measure of proportional cumulative performance. This adjustment decreased revenue by $2.5 million, increased net loss by $2.5 million and increased the Company's basic and diluted net loss per share by $0.02 for the three and nine months ended September 30, 2021. Revenue Recognition The Company accounts for its revenues pursuant to the provisions of Accounting Standards Codification (“ASC”) Topic 606, Revenue from Contracts with Customers (“ASC Topic 606”). The Company’s contract revenues are derived from collaboration agreements including licensing arrangements and research activity grants. Research and licensing agreements typically include upfront signing or license fees, cost reimbursements for research services, minimum sublicense fees, milestone payments and royalties on future licensees’ product sales. The Company has agreements with both fixed and variable consideration. Non-refundable upfront fees and funding of research and development activities are considered fixed, while milestone payments are generally identified as variable consideration. Sangamo’s research grants are typically multi-year agreements and provide for the reimbursement of qualified expenses for research and development as defined under the terms of the grant agreement. Revenues under research grant agreements are generally recognized when the related qualified research expenses are incurred. Deferred revenue primarily represents the portion of research or license payments received but not earned. In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under its agreements, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC Topic 606. The Company’s performance obligations include license rights, development services and services associated with regulatory submission and approval processes. Revenues from research services earned under collaboration agreements are generally recognized as revenue as the related services are provided. Revenues from non-refundable upfront fees are recognized over time either by measuring progress towards satisfaction of the relevant performance obligation, using the input method (i.e., cumulative actual costs incurred relative to total estimated costs) or on a straight-line basis when a performance obligation is expected to be satisfied evenly over a period of time (or when the entity has a stand-ready obligation). Significant management judgment is required to determine the level of effort required under an arrangement, and the period over which the Company expects to complete its performance obligations under the arrangement, which may include total internal personnel costs and external costs to be incurred as well as, in certain cases, the estimated stand-ready obligation period. Changes in these estimates can have a material effect on revenue recognized. If the Company cannot reasonably estimate when its performance obligations either are completed or become inconsequential, then revenue recognition is deferred until the Company can reasonably make such estimates. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint and, if necessary, adjusts its estimate of the overall transaction price. Revenue is then recognized over the remaining estimated period of performance using the cumulative catch-up method. The estimated period of performance and project costs, such as personnel and manufacturing cost, are reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverables. As part of the accounting for these arrangements, the Company must develop assumptions that require judgment to determine the stand-alone selling price of each performance obligation identified in the contract. The Company uses key assumptions to determine the stand-alone selling price, which may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Related costs and expenses under these arrangements have historically approximated the revenues recognized. Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows: Three Months Ended Nine Months Ended 2022 2021 2022 2021 Novartis Institutes for BioMedical Research, Inc. 36 % 40 % 35 % 35 % Biogen MA, Inc. 36 % 38 % 36 % 39 % Kite Pharma, Inc. 26 % 22 % 23 % 23 % Sanofi S.A. — % (3) % 4 % 2 % Funds received from the Company’s collaboration partners are generally not refundable and are recorded as revenue as the Company fulfills its performance obligations, which are satisfied over time (i.e., stand ready obligations) or by using the input method (i.e., cumulative actual costs incurred relative to total estimated costs). Revenue is also recognized when the Company has incurred qualified research and development costs that are reimbursable from its collaboration partners and when there is reasonable assurance that such costs will be reimbursed. Any payments received from a collaboration partner in advance of the completion of the relevant performance obligation are recorded as deferred revenue. Cash, Cash Equivalents, and Restricted Cash Sangamo considers all highly liquid investments purchased with original maturities of three months or less at the purchase date to be cash equivalents. Cash and cash equivalents consist of cash, deposits in demand money market accounts and U.S. government-sponsored entity debt securities. Restricted cash consists of a letter of credit for $1.5 million, representing a deposit for the lease of the corporate headquarters in Brisbane, California. A reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets to the amounts reported within the accompanying Condensed Consolidated Statements of Cash Flows is as follows (in thousands): September 30, December 31, September 30, December 31, Cash and cash equivalents $ 94,570 $ 178,872 $ 176,954 $ 131,329 Non-current restricted cash 1,500 1,500 1,500 1,500 Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows $ 96,070 $ 180,372 $ 178,454 $ 132,829 Recently Adopted Accounting Pronouncements None. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | FAIR VALUE MEASUREMENTS The Company measures certain financial assets and liabilities at fair value on a recurring basis, including cash equivalents, marketable securities, and the free shares asset. Fair value is determined based on a three-tier hierarchy under the authoritative guidance for fair value measurements and disclosures that prioritizes the inputs used in measuring fair value as follows: Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities; Level 2: Quoted prices in markets that are not active or inputs which are observable, either directly or indirectly, for substantially the full term of the asset or liability; and Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurements and unobservable (i.e., supported by little or no market activity). The fair value measurements of the Company’s cash equivalents and marketable securities are identified at the following levels within the fair value hierarchy (in thousands): September 30, 2022 Fair Value Measurements Total Level 1 Level 2 Level 3 Assets: Cash equivalents: Money market funds $ 44,911 $ 44,911 $ — $ — Total 44,911 44,911 — — Marketable securities: U.S. government-sponsored entity debt securities 23,392 — 23,392 — Commercial paper securities 120,386 — 120,386 — Corporate debt securities 14,888 — 14,888 — Asset-backed securities 37,773 — 37,773 — U.S. treasury bills 16,922 — 16,922 — Certificates of deposit 42,337 — 42,337 — Total 255,698 — 255,698 — Total cash equivalents and marketable securities $ 300,609 $ 44,911 $ 255,698 $ — December 31, 2021 Fair Value Measurements Total Level 1 Level 2 Level 3 Assets: Cash equivalents: Money market funds $ 119,919 $ 119,919 $ — $ — Total 119,919 119,919 — — Marketable securities: U.S. government-sponsored entity debt securities 30,614 — 30,614 — Commercial paper securities 105,757 — 105,757 — Corporate debt securities 33,682 — 33,682 — Asset-backed securities 70,701 — 70,701 — Certificates of deposit 45,091 — 45,091 — Total 285,845 — 285,845 — Total cash equivalents and marketable securities $ 405,764 $ 119,919 $ 285,845 $ — Cash Equivalents and Marketable Securities The Company generally classifies its marketable securities as Level 2. Instruments are classified as Level 2 when observable market prices for identical securities that are traded in less active markets are used. When observable market prices for identical securities are not available, such instruments are priced using benchmark curves, benchmarking of like securities, sector groupings, matrix pricing and valuation models. These valuation models are proprietary to the pricing providers or brokers and incorporate a number of inputs, including in approximate order of priority: benchmark yields, reported trades, broker/dealer quotes, issuer spreads, two-sided markets, benchmark securities, bids, offers and reference data including market research publications. For certain security types, additional inputs may be used, or some of the standard inputs may not be applicable. Evaluators may prioritize inputs differently on any given day for any security based on market conditions, and not all inputs listed are available for use in the evaluation process for each security evaluation on any given day. |
CASH EQUIVALENTS AND MARKETABLE
CASH EQUIVALENTS AND MARKETABLE SECURITIES | 9 Months Ended |
Sep. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
CASH EQUIVALENTS AND MARKETABLE SECURITIES | CASH EQUIVALENTS AND MARKETABLE SECURITIES The table below summarizes the Company’s cash equivalents and marketable securities (in thousands): Amortized Gross Gross Estimated September 30, 2022 Assets Cash equivalents: Money market funds $ 44,911 $ — $ — $ 44,911 Total 44,911 — — 44,911 Marketable securities: U.S. government-sponsored entity debt securities 23,832 — (440) 23,392 Commercial paper securities 120,750 11 (375) 120,386 Corporate debt securities 15,050 — (162) 14,888 Asset-backed securities 38,039 2 (268) 37,773 U.S. treasury bills 16,943 — (21) 16,922 Certificate of deposits 42,478 5 (146) 42,337 Total 257,092 18 (1,412) 255,698 Total cash equivalents and marketable securities $ 302,003 $ 18 $ (1,412) $ 300,609 December 31, 2021 Assets Cash equivalents: Money market funds $ 119,919 $ — $ — $ 119,919 Total 119,919 — — 119,919 Marketable securities: U.S. government-sponsored entity debt securities 30,700 1 (87) 30,614 Commercial paper securities 105,792 7 (42) 105,757 Corporate debt securities 33,723 1 (42) 33,682 Asset-backed securities 70,807 1 (107) 70,701 Certificate of deposits 45,116 1 (26) 45,091 Total 286,138 11 (304) 285,845 Total cash equivalents and marketable securities $ 406,057 $ 11 $ (304) $ 405,764 The fair value of marketable securities by contractual maturity were as follows (in thousands): September 30, December 31, Maturing in one year or less $ 218,998 $ 197,676 Maturing after one year through five years 36,700 88,169 Total $ 255,698 $ 285,845 There were no realized gains and losses on the sales of investments during the three and nine months ended September 30, 2022. Realized gains and losses on the sales of investments were insignificant during the three and nine months ended September 30, 2021. The Company manages credit risk associated with its investment portfolio through its investment policy, which limits purchases to high-quality issuers and also limits the amount of its portfolio that can be invested in a single issuer. The Company did not record an allowance for credit losses or other impairment charges related to its marketable securities for the three and nine months ended September 30, 2022 and 2021. The Company had unrealized losses related to its marketable securities for the three and nine months ended September 30, 2022 and 2021. The Company had no material unrealized losses, individually and in the aggregate, for marketable |
BASIC AND DILUTED NET LOSS PER
BASIC AND DILUTED NET LOSS PER SHARE | 9 Months Ended |
Sep. 30, 2022 | |
Earnings Per Share [Abstract] | |
BASIC AND DILUTED NET LOSS PER SHARE | BASIC AND DILUTED NET LOSS PER SHARE Basic net loss per share attributable to Sangamo Therapeutics, Inc. stockholders has been computed by dividing net loss attributable to Sangamo Therapeutics, Inc. stockholders by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders is calculated by dividing net loss attributable to Sangamo Therapeutics, Inc. stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration of any potential dilutive securities, as their effect would be anti-dilutive. The total number of shares subject to stock options and restricted stock units (“RSUs”) outstanding and the employee stock purchase plan (“ESPP”) shares reserved for issuance, which are all anti-dilutive, were excluded from consideration in the calculation of diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders. Stock options and RSUs outstanding and ESPP shares reserved for issuance as of September 30, 2022 and 2021 totaled 18,524,016 and 15,838,002, respectively. |
MAJOR CUSTOMERS, PARTNERSHIPS A
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES | 9 Months Ended |
Sep. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES | MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES Novartis Institutes for BioMedical Research, Inc. On July 27, 2020, the Company entered into a collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc. (“Novartis”) for the research, development and commercialization of gene regulation therapies to treat three neurodevelopmental disorders. Under the agreement, which was effective upon execution, the Company granted Novartis an exclusive, royalty bearing and worldwide license, under its relevant patents and know-how, to develop, manufacture and commercialize certain of its zinc finger protein (“ZFP”) transcription factors (“ZFP-TFs”) targeted to three undisclosed genes that are associated with certain neurodevelopmental disorders, including autism spectrum disorder and intellectual disability. The Company is performing early research activities over the collaboration period for each gene target and manufacture the ZFP-TFs required for such research, costs of which are funded by Novartis. Novartis is responsible for additional research activities, studies enabling investigational new drug applications (“INDs”), clinical development, regulatory approvals, manufacturing of preclinical, clinical and approved products, and global commercialization. Subject to certain exceptions set forth in the agreement, the Company is prohibited from developing, manufacturing or commercializing any therapeutic product targeting any of the three genes that are the subject of the collaboration. Novartis also has the option to license certain of the Company’s proprietary adeno-associated viruses (“AAVs”) for the sole purpose of developing, manufacturing and commercializing licensed products arising from the collaboration. Under the agreement, Novartis paid the Company a $75.0 million upfront license fee in August 2020. In addition to this fee and the cost reimbursements for early research activities, the Company is eligible to earn from Novartis up to $420.0 million in development milestones and up to $300.0 million in commercial milestones. The Company is also eligible to earn from Novartis tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of licensed products arising from the collaboration. These royalty payments will be subject to reduction due to patent expiration, loss of market exclusivity and payments made under certain licenses for third-party intellectual property. The agreement will continue, on a product-by-product and country-by-country basis, until the expiration of the applicable royalty term. Novartis has the right to terminate the agreement, in its entirety or on a target-by-target basis, for any reason after a specified notice period. Each party also has the right to terminate the agreement on account of the other party’s bankruptcy or material, uncured breach. All payments received under the agreement, when earned, are non-refundable and non-creditable. The transaction price of $95.1 million includes the upfront license fee of $75.0 million and estimated research costs of $20.1 million to be provided over the estimated research period. All clinical or regulatory milestone amounts were considered fully constrained at inception of the agreement. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at this time is uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will re-evaluate the transaction price, including the estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. The Company assessed the agreement with Novartis in accordance with ASC Topic 606 and concluded that Novartis is a customer. The Company has identified a single performance obligation within this arrangement as a license to the technology and ongoing research services. The Company concluded that the license is not discrete as it does not have stand-alone value to Novartis apart from the research services to be performed pursuant to the agreement. As a result, the Company recognizes revenue from the upfront payment based on proportional performance of the ongoing research services through the estimated research period. The estimation of progress towards the satisfaction of performance obligation and project cost is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its performance obligation. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $2.0 million and $1.9 million, respectively, and deferred revenue of $17.7 million and $40.9 million, respectively, related to this agreement. Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Novartis agreement: Recognition of upfront license fee $ 7,582 $ 9,093 $ 23,222 $ 22,852 Research services 2,028 2,421 6,211 6,104 Total $ 9,610 $ 11,514 $ 29,433 $ 28,956 The Company paid $1.5 million for financial advisory fees during the year ended December 31, 2020, equal to 2% of $75.0 million received for the upfront license fee related to the collaboration and license agreement with Novartis. The Company recognized $1.5 million as a contract asset as such amount represents a cost of obtaining the agreement. This balance is amortized and included in general and administrative expenses on a systematic basis consistent with the transfer of the services to Novartis in accordance with ASC Topic 340, Other Assets and Deferred Costs (“ASC Topic 340”). The Company amortized $0.2 million and $0.5 million during the three and nine months ended September 30, 2022, respectively, and $0.2 million and $0.5 million during the three and nine months ended September 30, 2021, respectively. Biogen MA, Inc. In February 2020, the Company entered into a collaboration and license agreement with Biogen MA, Inc. (“BIMA”) and Biogen International GmbH (together with BIMA, “Biogen”) for the research, development and commercialization of gene regulation therapies for the treatment of neurological diseases. The companies plan to leverage the Company’s proprietary ZFP technology delivered via AAV to modulate expression of key genes involved in neurological diseases. Concurrently with the execution of the collaboration agreement, the Company entered into a stock purchase agreement with BIMA, pursuant to which BIMA agreed to purchase 24,420,157 shares of the Company’s common stock (the “Biogen Shares”), at a price per share of $9.2137, for an aggregate purchase price of approximately $225.0 million. The collaboration agreement became effective in April 2020 following the termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and satisfaction of other customary closing conditions, including the payment of $225.0 million for the purchase of the Biogen Shares. Under the collaboration agreement, Biogen paid the Company an upfront license fee of $125.0 million in May 2020. The Company is also eligible to receive research, development, regulatory and commercial milestone payments that could total up to approximately $2.4 billion if Biogen selects all of the targets allowed under the agreement and all the specified milestones set forth in the agreement are achieved, which includes up to $925.0 million in pre-approval milestone payments and up to $1.5 billion in first commercial sale and other sales-based milestone payments. In addition, the Company is eligible to receive tiered high single-digit to sub-teen royalties on potential net commercial sales of licensed products arising from the collaboration. These royalty payments are subject to reduction due to patent expiration, entry of biosimilar products to the market and payments made under certain licenses for third-party intellectual property. Under the collaboration agreement, the Company granted to Biogen an exclusive, royalty bearing and worldwide license, under its relevant patents and know-how, to develop, manufacture and commercialize certain ZFP and/or AAV-based products directed to certain neurological disease gene targets selected by Biogen. Biogen has already selected four of these: ST-501 to treat tauopathies, ST-502 to treat synucleinopathies including Parkinson’s disease, a third product candidate targeting DM1, a neuromuscular disease, and a fourth undisclosed neurological disease gene target. Biogen has exclusive rights to nominate up to seven additional targets over the remaining period of five years from the effective date of the collaboration agreement. For each gene target selected by Biogen, the Company performs early research activities, costs of which are shared by the companies, aimed at the development of the combination of proprietary central nervous system delivery vectors and ZFP-TFs (or potential other ZFP products) targeting therapeutically relevant genes. Biogen has assumed responsibility and costs for the IND-enabling studies, clinical development, related regulatory interactions, and global commercialization. The Company is responsible for manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity, where appropriate, which is currently in development. Biogen is responsible for manufacturing activities beyond the first clinical trial for each of the first three products. The Company’s research activities for any targets will be performed over the period not to exceed seven years from the effective date of the agreement (i.e., through April 2027). Subject to certain exceptions set forth in the collaboration agreement, the Company is prohibited from developing, manufacturing or commercializing any therapeutic product directed to the targets selected by Biogen. The collaboration agreement continues on a product-by-product and country-by-country basis until the expiration of all applicable royalty terms. Biogen has the right to terminate the collaboration agreement, in its entirety or on a target-by-target basis, for any reason after a specified notice period, and also has the right to replace up to ten targets. Each party has the right to terminate this agreement on account of the other party’s bankruptcy or material, uncured breach. In addition, the Company may terminate the collaboration agreement if Biogen challenges any patents licensed by the Company to Biogen. Pursuant to the terms of the stock purchase agreement, Biogen has agreed not to, without the Company’s prior written consent and subject to specified conditions and exceptions, directly or indirectly acquire shares of the Company’s outstanding common stock, seek or propose a tender or exchange offer or merger between the parties, solicit proxies or consents with respect to any matter, or undertake other specified actions related to the potential acquisition of additional equity interests in the Company. Such standstill restrictions expire on the earlier of the three-year anniversary of the effectiveness of the collaboration agreement and the date that Biogen beneficially owns less than 5% of the Company’s common stock. The Company assessed the collaboration agreement with Biogen in accordance with ASC Topic 606 and concluded that Biogen is a customer. The transaction price of $204.6 million includes the upfront license fee of $125.0 million and the excess consideration from the stock purchase of $79.6 million, which represents the difference between the $225.0 million received for the purchase of the Biogen Shares and the $145.4 million estimated fair value of the equity issued. The equity issued to Biogen was valued using an option pricing model to reflect certain holding period restrictions. None of the target selection fees and clinical or regulatory milestones have been included in the transaction price, as all such amounts are fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that nomination of additional targets and achievement of the milestones at this time is uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will re-evaluate the transaction price as uncertain events are resolved or other changes in circumstances occur. The Company has identified a single performance obligation within the Biogen collaboration agreement, which is a stand-ready obligation consisting of a series of distinct days of research services, during which Biogen obtains access to the Company’s license and research resources. Revenue from the upfront license fee relates to access to the license and Company’s obligation to stand-ready to perform such research services corresponding to the targets selected by Biogen. As a result of this obligation to perform research services when and if requested throughout the duration of the contract, the upfront license fee and the excess consideration from the stock purchase will be recognized over time on a straight-line basis consistent with the resources expected to be dedicated to providing the research services through April 2027, the estimated period of the obligation. The estimated period of performance is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverable. Revenue from the reimbursement by Biogen of shared costs of early research activities performed by Sangamo is recognized as the research services are performed. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $1.7 million and $2.8 million, respectively, and deferred revenue of $132.1 million and $154.0 million, respectively, related to this agreement. Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Biogen agreement: Recognition of license and stand-ready fee $ 7,306 $ 7,306 $ 21,918 $ 21,918 Research services 2,107 3,661 8,740 10,266 Total $ 9,413 $ 10,967 $ 30,658 $ 32,184 The Company paid $7.0 million for financial advisory fees during the year ended December 31, 2020, equal to 2% of $225.0 million received for the sale of shares and 2% of $125.0 million received for the upfront fee. The fees incurred related to both the collaboration agreement with Biogen and the stock purchase agreement for the sale of shares. The Company believes that the allocation of fees on a relative fair value basis between the two agreements is reasonable. The Company recognized $4.1 million, which represents 2% of the transaction price of $204.6 million, as a contract asset. This balance is amortized and included in general and administrative expenses on a systematic basis consistent with the transfer of the services to Biogen in accordance with ASC Topic 340. The Company amortized $0.1 million and $0.4 million during the three and nine months ended September 30, 2022, respectively, and $0.1 million and $0.4 million during the three and nine months ended September 30, 2021, respectively. The Company recognized $2.9 million, which represented 2% of the $145.4 million estimated fair value of the equity issued, as a share issuance cost and recorded this amount in equity as a reduction in net proceeds during the year ended December 31, 2020. Kite Pharma, Inc. In February 2018, the Company entered into a global collaboration and license agreement with Kite Pharma, Inc. (“Kite”), a Gilead Sciences, Inc. subsidiary, which became effective on April 5, 2018 (“Effective Date”), and was amended and restated in September 2019, for the research, development, and commercialization of potential engineered cell therapies for cancer. In this collaboration, Sangamo is working together with Kite on a research program under which the companies are designing zinc finger nucleases (“ZFNs”) and viral vectors to disrupt and insert certain genes in T-cells and natural killer cells (“NK-cells”) including the insertion of genes that encode chimeric antigen receptors (“CARs”), T-cell receptors (“TCRs”), and NK-cell receptors (“NKRs”) directed to mutually agreed targets. Kite is responsible for all clinical development, manufacturing and commercialization of any resulting products. Subject to the terms of this agreement, the Company granted Kite an exclusive, royalty-bearing, worldwide sublicensable license under the Company’s relevant patents and know-how to develop, manufacture and commercialize, for the purpose of treating cancer, specific cell therapy products that may result from the research program and that are engineered ex vivo using selected ZFNs and viral vectors developed under the research program to express CARs, TCRs or NKRs directed to candidate targets. During the research program term and subject to certain exceptions, the Company is prohibited from researching, developing, manufacturing and commercializing, for the purpose of treating cancer, any cell therapy product that, as a result of ex vivo genome editing, expresses a CAR, TCR or NKR that is directed to a target expressed on or in a human cancer cell. After the research program term concludes and subject to certain exceptions, the Company will be prohibited from developing, manufacturing and commercializing, for the purpose of treating cancer, any cell therapy product that, as a result of ex vivo genome editing, expresses a CAR, TCR or NKR that is directed to a candidate target. Following the Effective Date, the Company received a $150.0 million upfront payment from Kite. In addition, Kite reimburses the Company’s direct costs to conduct the joint research program. Sangamo is also eligible to receive contingent development- and sales-based milestone payments that could total up to $3.0 billion if all of the specified milestones set forth in this agreement are achieved. Of this amount, approximately $1.3 billion relates to the achievement of specified research, clinical development, regulatory and first commercial sale milestones, and approximately $1.8 billion relates to the achievement of specified sales-based milestones if annual worldwide net sales of licensed products reach specified levels. Each development- and sales-based milestone payment is payable (i) only once for each licensed product, regardless of the number of times that the associated milestone event is achieved by such licensed product, and (ii) only for the first 10 times that the associated milestone event is achieved regardless of the number of licensed products that may achieve such milestone event. In addition, the Company is entitled to receive escalating, tiered royalty payments with a percentage in the single digits based on future annual worldwide net sales of licensed products. These royalty payments are subject to reduction due to patent expiration, entry of biosimilar products to the market and payments made under certain licenses for third-party intellectual property. The initial research term in the agreement is six years from the Effective Date. Kite has an option to extend the research term for up to two additional one-year periods for a separate upfront fee of $10.0 million per year. All contingent payments under the agreement, when earned, will be non-refundable and non-creditable. In connection with the amendment and restatement of the agreement in September 2019, the Company entered into a new research plan with Kite, with estimated reimbursable service cost of approximately $3.4 million, which is included in the total transaction price. The Company concluded the total transaction price under this agreement is $189.3 million and includes the upfront license fee of $150.0 million and $39.3 million estimated reimbursable service costs for identified research projects over the estimated performance period. Further, the Company concluded the estimated fees for the presumed exercise of the research term extension options and all milestone amounts are fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at this time is uncertain and contingent upon future events which are uncertain at this time. The Company will re-evaluate the transaction price including the estimated variable consideration included in the transaction price and all constrained amounts in each reporting period and as uncertain events are resolved or other changes in circumstances occur. None of the development and sales-based milestone payments have been included in the transaction price. The Company assessed the agreement with Kite in accordance with ASC Topic 606 and concluded that Kite is a customer. Kite has the right to terminate this agreement in its entirety or on a per licensed product or per candidate target basis for any reason after a specified notice period. Each party has the right to terminate this agreement on account of the other party’s bankruptcy or material, uncured breach. The Company has identified the primary performance obligations within the Kite agreement as: (1) a license to the technology along with the stand-ready obligation to perform research services, and (2) the ongoing research services. Revenue from the upfront license fee relates to access to the license and Company’s obligation to stand-ready to perform such research services as additional targets are selected by Kite. As a result of this obligation to perform research services when and if requested throughout the duration of the contract, the fee for the license and the stand-ready obligation will be recognized over time on a straight-line basis through April 2024, the estimated period of the stand-ready obligation. Revenue from the reimbursable costs related to the integrated service deliverable is recognized as the research services are performed. Related costs and expenses under these arrangements have historically approximated the revenues recognized. The estimated period of performance and project cost is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverables. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $0.6 million and $0.1 million, respectively, and deferred revenue of $37.8 million and $56.5 million, respectively, related to this agreement. Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Kite agreement: Recognition of license and stand-ready fee $ 6,296 $ 6,296 $ 18,682 $ 18,682 Research services 619 113 875 339 Total $ 6,915 $ 6,409 $ 19,557 $ 19,021 Sanofi S.A. In January 2014, the Company entered into an exclusive worldwide collaboration and license agreement (“2014 Collaboration Agreement”) to develop therapeutics for hemoglobinopathies, focused on beta thalassemia and sickle cell disease (“SCD”). The 2014 Collaboration Agreement was originally signed with BIMA, who subsequently assigned it to Bioverativ Inc., which was later acquired by Sanofi. Under the 2014 Collaboration Agreement, the Company was originally jointly conducting two research programs: a beta thalassemia program, which was discontinued in the third quarter of 2021, and the SCD program, which resulted in the development of SAR445136 (now known as BIVV003), a ZFN, gene-edited cell therapy product candidate for the treatment of SCD, which remains active. In December 2021, Sanofi notified the Company of its termination for convenience, effective June 28, 2022 (the “Termination Date”), of the 2014 Collaboration Agreement. A termination and transition agreement (the “Termination and Transition Agreement”) was executed by the parties on September 6, 2022. In the SCD program, the Company and Sanofi were jointly responsible for research and development activities prior to filing of an IND, but Sanofi was responsible for subsequent worldwide clinical development, manufacturing and commercialization of licensed products developed under the agreement. Subject to the terms of the agreement, the Company had granted Sanofi an exclusive, royalty-bearing license, with the right to grant sublicenses, to use certain ZFP and other technology controlled by the Company for the purpose of researching, developing, manufacturing and commercializing licensed products developed under the agreement. The Company had also granted Sanofi a non-exclusive worldwide, royalty-free fully paid license with the right to grant sublicenses, under the Company’s interest in certain other intellectual property developed pursuant to the agreement. During the term of the agreement, the Company was not permitted to research, develop, manufacture or commercialize, outside of the agreement, certain gene therapy products that target genes relevant to the licensed products. Under the agreement, the Company received an upfront license fee of $20.0 million and was eligible to receive additional payments upon the achievement of specified clinical development, regulatory and sales milestones, as well as royalty payments for each licensed product based on net sales of such product. Sanofi was also to reimburse Sangamo for agreed upon costs incurred in connection with research and development activities conducted by Sangamo. Through the Termination Date, a total of $13.5 million has been received based upon achievement of clinical development milestones. No products have been approved and therefore no royalty fees have been or will be earned under the 2014 Collaboration Agreement. In its termination notice to the Company, Sanofi indicated that its termination relates to Sanofi’s change in strategic direction to focus on allogeneic universal genomic medicine approaches rather than autologous personalized cell therapies. As of the Termination Date, the 2014 Collaboration Agreement was terminated in its entirety and following the Termination Date, the Company will not be entitled to receive any further milestone payments or royalties from Sanofi. As of the Termination Date, Sanofi has no further obligations under the 2014 Collaboration Agreement to develop or to fund the development of any collaboration research programs under the 2014 Collaboration Agreement. The licenses granted to Sanofi under the 2014 Collaboration Agreement have been terminated, and the license rights have reverted to the Company. As part of the Termination and Transition Agreement, Sanofi granted to the Company exclusive, worldwide, fully paid, royalty-free, perpetual, irrevocable licenses, with the right to grant sublicenses through multiple tiers, to certain of its intellectual property, to develop, manufacture, use, sell and otherwise commercialize BIVV003, the product candidate in development under the SCD program. The Company has agreed to take on responsibilities for all clinical trials related to BIVV003, including completion of the ongoing clinical trial and the related long-term follow-up study. The Company also assumed all regulatory responsibilities related to BIVV003. Sanofi has agreed to provide post-transition assistance to the Company for up to 90 days after the Termination Date, and transferred or assigned to the Company all documentation, materials, contracts with third parties, and the right to use certain Sanofi-owned equipment related to BIVV003. Sanofi has also agreed to reimburse the Company for the costs of conducting the ongoing clinical trial of BIVV003 and the costs of the long-term follow-up study through December 31, 2023, up to $7.0 million. In addition, should the Company elect not to continue the development of BIVV003 past December 31, 2023, Sanofi will become obligated to reimburse the Company for the costs of the long-term follow-up study incurred after 2023, up to $5.3 million. Sanofi’s reimbursement obligations will terminate upon certain triggering events, including the Company’s entering into a contract with a third party for collaboration, partnership, sale, licensing, or divestiture of BIVV003, or if the FDA permits early closure of the clinical trial and/or the long-term follow-up study. The Company assessed the 2014 Collaboration Agreement with Sanofi in accordance with ASC Topic 606 and concluded that Sanofi was a customer, under that arrangement. The Company identified the performance obligation within this arrangement as a license to the technology combined with ongoing research services activities. The Company concluded that the license was not distinct as it did not have value to Sanofi without the research services. As a result, the Company recognized revenue from the upfront payment and the milestones based on progress of performance of the ongoing research services. The estimation of progress towards the satisfaction of the performance obligation and project cost was reviewed quarterly and adjusted, as needed, to reflect the Company’s then-current assumptions regarding the timing of its deliverables. Related costs and expenses under these arrangements have historically approximated the revenues recognized. Sanofi’s December 2021 notice of termination of the 2014 Collaboration Agreement represented a modification that reduced the expected scope of the Company’s services and the estimated transaction price and shortened the remaining performance timeline. Consistent with this change, all services under the 2014 Collaboration Agreement were completed by June 28, 2022, and all amounts ultimately included in the transaction price were recognized by such date. The final transaction price of $96.3 million includes the upfront license fee of $20.0 million, two milestone payments in the aggregate amount of $13.5 million and reimbursement of research costs of $62.8 million. As of September 30, 2022 and December 31, 2021, the Company had a receivable of zero and $0.6 million, respectively, related to the 2014 Collaboration Agreement. Deferred revenue related to the 2014 Collaboration Agreement was zero and $1.1 million as of September 30, 2022 and December 31, 2021, respectively. The Company concluded that Sanofi is not a customer under the Termination and Transition Agreement as Sanofi is not entitled to receive and cannot use the results of the ongoing clinical trial or the long-term follow-up study. This relationship is also not a collaboration in the scope of ASC Topic 808, Collaborative Arrangements . The Company concluded that the assets acquired from Sanofi do not represent a business, as substantially all of their value is concentrated in the acquired or re-acquired licenses to intellectual property. The Company has no obligation to repay Sanofi for its ongoing funding of the clinical trial or long-term follow-up study costs. Therefore, the Company will recognize Sanofi reimbursements as reductions of its research and development expense. During the three and nine months ended September 30, 2022, the Company decreased its research and development expense by $0.9 million, which is included within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022. Revenues recognized under the 2014 Collaboration Agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Sanofi agreement: Recognition of upfront fee $ — $ (1,125) $ 677 $ (692) Research services — 1,125 2,126 2,417 Milestone achievement — (759) 457 (467) Total $ — $ (759) $ 3,260 $ 1,258 In September 2021, the Company recorded an adjustment to revenue related to a change in estimate in connection with the collaboration agreement with Sanofi. This adjustment was a result of an increase in the project costs for its beta thalassemia program and the increase in project scope and the corresponding costs for its SCD program, both of which resulted in a decrease in the measure of proportional cumulative performance. This adjustment decreased revenue by $2.5 million, increased net loss by $2.5 million and increased the Company's basic and diluted net loss per share by $0.02 for the three and nine months ended September 30, 2021. Pfizer Inc. Giroctocogene Fitelparvovec Global Collaboration and License Agreement In May 2017, the Company entered into an exclusive global collaboration and license agreement with Pfizer Inc. (“Pfizer”), pursuant to which it established a collaboration for the research, development |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 9 Months Ended |
Sep. 30, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | STOCK-BASED COMPENSATION The following table shows total stock-based compensation expense included in the Condensed Consolidated Statements of Operations (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Research and development $ 4,395 $ 4,935 $ 13,656 $ 14,645 General and administrative 3,398 2,938 9,746 10,235 Total stock-based compensation expense $ 7,793 $ 7,873 $ 23,402 $ 24,880 |
STOCKHOLDERS' EQUITY
STOCKHOLDERS' EQUITY | 9 Months Ended |
Sep. 30, 2022 | |
Equity [Abstract] | |
STOCKHOLDERS' EQUITY | STOCKHOLDERS’ EQUITY At-the-Market Offering Agreement In August 2020, the Company entered into an Open Market Sale Agreement℠ with Jefferies LLC (“Jefferies”) with respect to an at-the-market offering program under which the Company may offer and sell, from time to time at its sole discretion, shares of the Company’s common stock having an aggregate offering price of up to $150.0 million through Jefferies as the Company’s sales agent or principal. The Company is not obligated to sell any shares under the sales agreement. During the three and nine months ended September 30, 2022, the Company sold 8,483,104 and 14,711,770 shares of its common stock for net proceeds of approximately $42.2 million and $66.4 million, respectively, of which approximately $0.6 million was received in October 2022 and recorded within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022. During the three and nine months ended September 30, 2021, the Company sold 202,705 and 2,007,932 shares of its common stock for net proceeds of approximately $2.4 million, and $27.1 million, respectively. |
INCOME TAXES
INCOME TAXES | 9 Months Ended |
Sep. 30, 2022 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | INCOME TAXES The Company’s provision for income taxes for interim periods is determined using an estimate of its annual effective tax rate, adjusted for discrete items, if any, that arise during the period. Each quarter, the Company updates its estimate of the annual effective tax rate, and if the estimated annual effective tax rate changes, the Company makes a cumulative adjustment in such period. During the three and nine months ended September 30, 2022, the Company recorded income tax expense of $0.1 million and $0.2 million, respectively. During the three and nine months ended September 30, 2021, the Company recorded income tax expense of $0.1 million and $0.4 million, respectively. The Company continues to maintain a full valuation allowance on its U.S. federal and state net deferred tax assets and on the Sangamo France net deferred tax assets, as the Company believes it is not more likely than not that these benefits will be realized. The tax expense for the three and nine months ended September 30, 2022 was primarily due to foreign income tax expense. The Coronavirus Aid, Relief, and Economic Security Act, enacted on March 27, 2020, provided an Employee Retention Credit (“ERC”), which is a refundable payroll tax credit that encouraged businesses to keep employees on the payroll during the COVID-19 pandemic. Eligible employers could qualify for up to $5,000 of credit for each employee based on certain wages paid after March 12, 2020 and before January 1, 2021 and extended to September 30, 2021. Based on the Company’s evaluation of this provision and the significant pandemic-related impacts on the operations, the Company recognized zero and $3.0 million of ERC for the three and nine months ended September 30, 2022, respectively, within interest and other income, net in Condensed Consolidated Statement of Operations upon filing for the refund in January and April 2022. No refund has been received during the three and nine months ended September 30, 2022 and the amount has been recorded within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022. |
ORGANIZATION, BASIS OF PRESEN_2
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - (Policies) | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited Condensed Consolidated Financial Statements have been prepared in accordance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) for interim financial information and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Accordingly, they do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. In the opinion of management, all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation of these financial statements for the periods presented have been included. Operating results for the three and nine months ended September 30, 2022 are not necessarily indicative of the results that may be expected for the year ending December 31, 2022. The Condensed Consolidated Balance Sheet data at December 31, 2021 was derived from the audited Consolidated Financial Statements included in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2021 (the “2021 Annual Report”) as filed with the SEC on February 24, 2022. The accompanying Condensed Consolidated Financial Statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in the Condensed Consolidated Financial Statements. For consolidated entities where the Company owns or is exposed to less than 100% of the economics, the Company records net loss attributable to non-controlling interests on its Condensed Consolidated Statements of Operations equal to the percentage of the economic or ownership interest retained in such entities by the respective non-controlling parties. The accompanying Condensed Consolidated Financial Statements and related financial information should be read together with the audited Consolidated Financial Statements and footnotes for the year ended December 31, 2021, included in the 2021 Annual Report. |
Use of Estimates | Use of Estimates The preparation of Condensed Consolidated Financial Statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the Condensed Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates including critical accounting policies or estimates related to revenue recognition, clinical trial accruals, income taxes, fair value of assets and liabilities, including from acquisitions, and stock-based compensation. Estimates are based on historical experience and on various other market specific and other relevant assumptions that the Company believes to be reasonable under the circumstances, the results of which form the basis for |
Revenue Recognition | Revenue Recognition The Company accounts for its revenues pursuant to the provisions of Accounting Standards Codification (“ASC”) Topic 606, Revenue from Contracts with Customers (“ASC Topic 606”). The Company’s contract revenues are derived from collaboration agreements including licensing arrangements and research activity grants. Research and licensing agreements typically include upfront signing or license fees, cost reimbursements for research services, minimum sublicense fees, milestone payments and royalties on future licensees’ product sales. The Company has agreements with both fixed and variable consideration. Non-refundable upfront fees and funding of research and development activities are considered fixed, while milestone payments are generally identified as variable consideration. Sangamo’s research grants are typically multi-year agreements and provide for the reimbursement of qualified expenses for research and development as defined under the terms of the grant agreement. Revenues under research grant agreements are generally recognized when the related qualified research expenses are incurred. Deferred revenue primarily represents the portion of research or license payments received but not earned. In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under its agreements, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC Topic 606. The Company’s performance obligations include license rights, development services and services associated with regulatory submission and approval processes. Revenues from research services earned under collaboration agreements are generally recognized as revenue as the related services are provided. Revenues from non-refundable upfront fees are recognized over time either by measuring progress towards satisfaction of the relevant performance obligation, using the input method (i.e., cumulative actual costs incurred relative to total estimated costs) or on a straight-line basis when a performance obligation is expected to be satisfied evenly over a period of time (or when the entity has a stand-ready obligation). Significant management judgment is required to determine the level of effort required under an arrangement, and the period over which the Company expects to complete its performance obligations under the arrangement, which may include total internal personnel costs and external costs to be incurred as well as, in certain cases, the estimated stand-ready obligation period. Changes in these estimates can have a material effect on revenue recognized. If the Company cannot reasonably estimate when its performance obligations either are completed or become inconsequential, then revenue recognition is deferred until the Company can reasonably make such estimates. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint and, if necessary, adjusts its estimate of the overall transaction price. Revenue is then recognized over the remaining estimated period of performance using the cumulative catch-up method. The estimated period of performance and project costs, such as personnel and manufacturing cost, are reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverables. As part of the accounting for these arrangements, the Company must develop assumptions that require judgment to determine the stand-alone selling price of each performance obligation identified in the contract. The Company uses key assumptions to determine the stand-alone selling price, which may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Related costs and expenses under these arrangements have historically approximated the revenues recognized. Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows: Three Months Ended Nine Months Ended 2022 2021 2022 2021 Novartis Institutes for BioMedical Research, Inc. 36 % 40 % 35 % 35 % Biogen MA, Inc. 36 % 38 % 36 % 39 % Kite Pharma, Inc. 26 % 22 % 23 % 23 % Sanofi S.A. — % (3) % 4 % 2 % Funds received from the Company’s collaboration partners are generally not refundable and are recorded as revenue as the Company fulfills its performance obligations, which are satisfied over time (i.e., stand ready obligations) or by using the input method (i.e., cumulative actual costs incurred relative to total estimated costs). Revenue is also recognized when the Company has incurred qualified research and development costs that are reimbursable from its collaboration partners and when there is reasonable assurance that such costs will be reimbursed. Any payments received from a collaboration partner in advance of the completion of the relevant performance obligation are recorded as deferred revenue. |
Cash, Cash Equivalents and Restricted Cash | Cash, Cash Equivalents, and Restricted Cash Sangamo considers all highly liquid investments purchased with original maturities of three months or less at the purchase date to be cash equivalents. Cash and cash equivalents consist of cash, deposits in demand money market accounts and U.S. government-sponsored entity debt securities. Restricted cash consists of a letter of credit for $1.5 million, representing a deposit for the lease of the corporate headquarters in Brisbane, California. |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements None. |
Fair Value Measurements | FAIR VALUE MEASUREMENTS The Company measures certain financial assets and liabilities at fair value on a recurring basis, including cash equivalents, marketable securities, and the free shares asset. Fair value is determined based on a three-tier hierarchy under the authoritative guidance for fair value measurements and disclosures that prioritizes the inputs used in measuring fair value as follows: Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities; Level 2: Quoted prices in markets that are not active or inputs which are observable, either directly or indirectly, for substantially the full term of the asset or liability; and Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurements and unobservable (i.e., supported by little or no market activity). |
ORGANIZATION, BASIS OF PRESEN_3
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Schedule of Revenue from Strategic Partnering Collaboration Agreements and Research Activity Grants as a Percentage of Total Revenues | Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows: Three Months Ended Nine Months Ended 2022 2021 2022 2021 Novartis Institutes for BioMedical Research, Inc. 36 % 40 % 35 % 35 % Biogen MA, Inc. 36 % 38 % 36 % 39 % Kite Pharma, Inc. 26 % 22 % 23 % 23 % Sanofi S.A. — % (3) % 4 % 2 % |
Schedule of Cash and Cash Equivalents | A reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets to the amounts reported within the accompanying Condensed Consolidated Statements of Cash Flows is as follows (in thousands): September 30, December 31, September 30, December 31, Cash and cash equivalents $ 94,570 $ 178,872 $ 176,954 $ 131,329 Non-current restricted cash 1,500 1,500 1,500 1,500 Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows $ 96,070 $ 180,372 $ 178,454 $ 132,829 |
Restrictions on Cash and Cash Equivalents | A reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets to the amounts reported within the accompanying Condensed Consolidated Statements of Cash Flows is as follows (in thousands): September 30, December 31, September 30, December 31, Cash and cash equivalents $ 94,570 $ 178,872 $ 176,954 $ 131,329 Non-current restricted cash 1,500 1,500 1,500 1,500 Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows $ 96,070 $ 180,372 $ 178,454 $ 132,829 |
FAIR VALUE MEASUREMENTS - (Tabl
FAIR VALUE MEASUREMENTS - (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Summary of Fair Value Measurements of Cash Equivalents and Marketable Securities | The fair value measurements of the Company’s cash equivalents and marketable securities are identified at the following levels within the fair value hierarchy (in thousands): September 30, 2022 Fair Value Measurements Total Level 1 Level 2 Level 3 Assets: Cash equivalents: Money market funds $ 44,911 $ 44,911 $ — $ — Total 44,911 44,911 — — Marketable securities: U.S. government-sponsored entity debt securities 23,392 — 23,392 — Commercial paper securities 120,386 — 120,386 — Corporate debt securities 14,888 — 14,888 — Asset-backed securities 37,773 — 37,773 — U.S. treasury bills 16,922 — 16,922 — Certificates of deposit 42,337 — 42,337 — Total 255,698 — 255,698 — Total cash equivalents and marketable securities $ 300,609 $ 44,911 $ 255,698 $ — December 31, 2021 Fair Value Measurements Total Level 1 Level 2 Level 3 Assets: Cash equivalents: Money market funds $ 119,919 $ 119,919 $ — $ — Total 119,919 119,919 — — Marketable securities: U.S. government-sponsored entity debt securities 30,614 — 30,614 — Commercial paper securities 105,757 — 105,757 — Corporate debt securities 33,682 — 33,682 — Asset-backed securities 70,701 — 70,701 — Certificates of deposit 45,091 — 45,091 — Total 285,845 — 285,845 — Total cash equivalents and marketable securities $ 405,764 $ 119,919 $ 285,845 $ — |
CASH EQUIVALENTS AND MARKETAB_2
CASH EQUIVALENTS AND MARKETABLE SECURITIES - (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | The table below summarizes the Company’s cash equivalents and marketable securities (in thousands): Amortized Gross Gross Estimated September 30, 2022 Assets Cash equivalents: Money market funds $ 44,911 $ — $ — $ 44,911 Total 44,911 — — 44,911 Marketable securities: U.S. government-sponsored entity debt securities 23,832 — (440) 23,392 Commercial paper securities 120,750 11 (375) 120,386 Corporate debt securities 15,050 — (162) 14,888 Asset-backed securities 38,039 2 (268) 37,773 U.S. treasury bills 16,943 — (21) 16,922 Certificate of deposits 42,478 5 (146) 42,337 Total 257,092 18 (1,412) 255,698 Total cash equivalents and marketable securities $ 302,003 $ 18 $ (1,412) $ 300,609 December 31, 2021 Assets Cash equivalents: Money market funds $ 119,919 $ — $ — $ 119,919 Total 119,919 — — 119,919 Marketable securities: U.S. government-sponsored entity debt securities 30,700 1 (87) 30,614 Commercial paper securities 105,792 7 (42) 105,757 Corporate debt securities 33,723 1 (42) 33,682 Asset-backed securities 70,807 1 (107) 70,701 Certificate of deposits 45,116 1 (26) 45,091 Total 286,138 11 (304) 285,845 Total cash equivalents and marketable securities $ 406,057 $ 11 $ (304) $ 405,764 |
Fair value of investments available-for-sale | The fair value of marketable securities by contractual maturity were as follows (in thousands): September 30, December 31, Maturing in one year or less $ 218,998 $ 197,676 Maturing after one year through five years 36,700 88,169 Total $ 255,698 $ 285,845 |
MAJOR CUSTOMERS, PARTNERSHIPS_2
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Novartis Institutes for BioMedical Research, Inc. | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |
Revenues Recognized under Agreement | Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Novartis agreement: Recognition of upfront license fee $ 7,582 $ 9,093 $ 23,222 $ 22,852 Research services 2,028 2,421 6,211 6,104 Total $ 9,610 $ 11,514 $ 29,433 $ 28,956 |
Biogen MA, Inc. | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |
Revenues Recognized under Agreement | Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Biogen agreement: Recognition of license and stand-ready fee $ 7,306 $ 7,306 $ 21,918 $ 21,918 Research services 2,107 3,661 8,740 10,266 Total $ 9,413 $ 10,967 $ 30,658 $ 32,184 |
Kite Pharma, Inc. | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |
Revenues Recognized under Agreement | Revenues recognized under the agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Kite agreement: Recognition of license and stand-ready fee $ 6,296 $ 6,296 $ 18,682 $ 18,682 Research services 619 113 875 339 Total $ 6,915 $ 6,409 $ 19,557 $ 19,021 |
Sanofi S.A. | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |
Revenues Recognized under Agreement | Revenues recognized under the 2014 Collaboration Agreement were as follows (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Revenue related to Sanofi agreement: Recognition of upfront fee $ — $ (1,125) $ 677 $ (692) Research services — 1,125 2,126 2,417 Milestone achievement — (759) 457 (467) Total $ — $ (759) $ 3,260 $ 1,258 |
STOCK-BASED COMPENSATION - (Tab
STOCK-BASED COMPENSATION - (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation Expense | The following table shows total stock-based compensation expense included in the Condensed Consolidated Statements of Operations (in thousands): Three Months Ended Nine Months Ended 2022 2021 2022 2021 Research and development $ 4,395 $ 4,935 $ 13,656 $ 14,645 General and administrative 3,398 2,938 9,746 10,235 Total stock-based compensation expense $ 7,793 $ 7,873 $ 23,402 $ 24,880 |
ORGANIZATION, BASIS OF PRESEN_4
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | 102 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Jun. 28, 2022 | |
Change in Accounting Estimate [Line Items] | |||||
Decreased revenue | $ (26,460) | $ (28,563) | $ (84,069) | $ (82,715) | |
Increased net loss | $ 53,155 | $ 47,688 | $ 140,305 | $ 140,801 | |
Increased earnings per share, basic (in dollars per share) | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) | |
Increased earnings per share, diluted (in dollars per share) | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) | |
Letter of credit established as a deposit | $ 1,500 | $ 1,500 | |||
Sanofi S.A. | |||||
Change in Accounting Estimate [Line Items] | |||||
Decreased revenue | $ 0 | $ 759 | $ (3,260) | $ (1,258) | $ (13,500) |
Sanofi S.A. | Change In Collaboration Agreement Scope | Collaboration agreement | |||||
Change in Accounting Estimate [Line Items] | |||||
Decreased revenue | 2,500 | 2,500 | |||
Increased net loss | $ 2,500 | $ 2,500 | |||
Increased earnings per share, basic (in dollars per share) | $ 0.02 | $ 0.02 | |||
Increased earnings per share, diluted (in dollars per share) | $ 0.02 | $ 0.02 |
ORGANIZATION, BASIS OF PRESEN_5
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Revenues from Strategic Partnering Collaboration Agreements and Research Activity Grants (Detail) - Revenue from contract with customer - Customer concentration risk | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Novartis Institutes for BioMedical Research, Inc. | ||||
Concentration Risk [Line Items] | ||||
Percentage of revenues | 36% | 40% | 35% | 35% |
Biogen MA, Inc. | ||||
Concentration Risk [Line Items] | ||||
Percentage of revenues | 36% | 38% | 36% | 39% |
Kite Pharma, Inc. | ||||
Concentration Risk [Line Items] | ||||
Percentage of revenues | 26% | 22% | 23% | 23% |
Sanofi S.A. | ||||
Concentration Risk [Line Items] | ||||
Percentage of revenues | 0% | (3.00%) | 4% | 2% |
ORGANIZATION, BASIS OF PRESEN_6
ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Reconciliation of Cash and Cash Equivalents (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 | Sep. 30, 2021 | Dec. 31, 2020 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||||
Cash and cash equivalents | $ 94,570 | $ 178,872 | $ 176,954 | $ 131,329 |
Non-current restricted cash | 1,500 | 1,500 | 1,500 | 1,500 |
Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows | $ 96,070 | $ 180,372 | $ 178,454 | $ 132,829 |
FAIR VALUE MEASUREMENTS (Detail
FAIR VALUE MEASUREMENTS (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | $ 255,698 | $ 285,845 |
Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 44,911 | 119,919 |
U.S. government-sponsored entity debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 23,392 | 30,614 |
Commercial paper securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 120,386 | 105,757 |
Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 14,888 | 33,682 |
Asset-backed securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 37,773 | 70,701 |
U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 16,922 | |
Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 42,337 | 45,091 |
Fair value on recurring basis | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 44,911 | 119,919 |
Total marketable securities | 255,698 | 285,845 |
Total cash equivalents and marketable securities and free shares asset | 300,609 | 405,764 |
Fair value on recurring basis | Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 44,911 | 119,919 |
Fair value on recurring basis | U.S. government-sponsored entity debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 23,392 | 30,614 |
Fair value on recurring basis | Commercial paper securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 120,386 | 105,757 |
Fair value on recurring basis | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 14,888 | 33,682 |
Fair value on recurring basis | Asset-backed securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 37,773 | 70,701 |
Fair value on recurring basis | U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 16,922 | |
Fair value on recurring basis | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 42,337 | 45,091 |
Fair value on recurring basis | Level 1 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 44,911 | 119,919 |
Total marketable securities | 0 | 0 |
Total cash equivalents and marketable securities and free shares asset | 44,911 | 119,919 |
Fair value on recurring basis | Level 1 | Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 44,911 | 119,919 |
Fair value on recurring basis | Level 1 | U.S. government-sponsored entity debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 1 | Commercial paper securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 1 | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 1 | Asset-backed securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 1 | U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | |
Fair value on recurring basis | Level 1 | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 2 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 0 | 0 |
Total marketable securities | 255,698 | 285,845 |
Total cash equivalents and marketable securities and free shares asset | 255,698 | 285,845 |
Fair value on recurring basis | Level 2 | Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 0 | 0 |
Fair value on recurring basis | Level 2 | U.S. government-sponsored entity debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 23,392 | 30,614 |
Fair value on recurring basis | Level 2 | Commercial paper securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 120,386 | 105,757 |
Fair value on recurring basis | Level 2 | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 14,888 | 33,682 |
Fair value on recurring basis | Level 2 | Asset-backed securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 37,773 | 70,701 |
Fair value on recurring basis | Level 2 | U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 16,922 | |
Fair value on recurring basis | Level 2 | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 42,337 | 45,091 |
Fair value on recurring basis | Level 3 | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 0 | 0 |
Total marketable securities | 0 | 0 |
Total cash equivalents and marketable securities and free shares asset | 0 | 0 |
Fair value on recurring basis | Level 3 | Money market funds | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total cash equivalents | 0 | 0 |
Fair value on recurring basis | Level 3 | U.S. government-sponsored entity debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 3 | Commercial paper securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 3 | Corporate debt securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 3 | Asset-backed securities | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | 0 |
Fair value on recurring basis | Level 3 | U.S. treasury bills | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | 0 | |
Fair value on recurring basis | Level 3 | Certificates of deposit | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total marketable securities | $ 0 | $ 0 |
CASH EQUIVALENTS AND MARKETAB_3
CASH EQUIVALENTS AND MARKETABLE SECURITIES - Summary of Cash Equivalents and Available-for-Sale Securities (Detail) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 | Sep. 30, 2021 | Dec. 31, 2020 |
ASSETS | ||||
Cash equivalents, Amortized Cost | $ 94,570 | $ 178,872 | $ 176,954 | $ 131,329 |
Available-for-sale securities, Amortized Cost | 257,092 | 286,138 | ||
Available-for-sale securities, Gross Unrealized Gains | 18 | 11 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (1,412) | (304) | ||
Available-for-sale securities, Estimated Fair Value | 255,698 | 285,845 | ||
Total cash equivalents and available-for-sale securities, Amortized Cost | 302,003 | 406,057 | ||
Total cash equivalents and available-for-sale securities, Gross Unrealized Gains | 18 | 11 | ||
Total cash equivalents and available-for-sale securities, Gross Unrealized (Losses) | (1,412) | (304) | ||
Total cash equivalents and available-for-sale securities, Estimated Fair Value | 300,609 | 405,764 | ||
Money market funds | ||||
ASSETS | ||||
Cash equivalents, Amortized Cost | 44,911 | 119,919 | ||
Cash equivalents, Gross Unrealized Gains | 0 | 0 | ||
Cash equivalents, Gross Unrealized (Losses) | 0 | 0 | ||
Cash equivalents, Estimated Fair Value | 44,911 | 119,919 | ||
Cash equivalents | ||||
ASSETS | ||||
Cash equivalents, Amortized Cost | 44,911 | 119,919 | ||
Cash equivalents, Gross Unrealized Gains | 0 | 0 | ||
Cash equivalents, Gross Unrealized (Losses) | 0 | 0 | ||
Cash equivalents, Estimated Fair Value | 44,911 | 119,919 | ||
U.S. government-sponsored entity debt securities | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 23,832 | 30,700 | ||
Available-for-sale securities, Gross Unrealized Gains | 0 | 1 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (440) | (87) | ||
Available-for-sale securities, Estimated Fair Value | 23,392 | 30,614 | ||
Commercial paper securities | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 120,750 | 105,792 | ||
Available-for-sale securities, Gross Unrealized Gains | 11 | 7 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (375) | (42) | ||
Available-for-sale securities, Estimated Fair Value | 120,386 | 105,757 | ||
Corporate debt securities | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 15,050 | 33,723 | ||
Available-for-sale securities, Gross Unrealized Gains | 0 | 1 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (162) | (42) | ||
Available-for-sale securities, Estimated Fair Value | 14,888 | 33,682 | ||
Asset-backed securities | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 38,039 | 70,807 | ||
Available-for-sale securities, Gross Unrealized Gains | 2 | 1 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (268) | (107) | ||
Available-for-sale securities, Estimated Fair Value | 37,773 | 70,701 | ||
U.S. treasury bills | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 16,943 | |||
Available-for-sale securities, Gross Unrealized Gains | 0 | |||
Available-for-sale securities, Gross Unrealized (Losses) | (21) | |||
Available-for-sale securities, Estimated Fair Value | 16,922 | |||
Certificates of deposit | ||||
ASSETS | ||||
Available-for-sale securities, Amortized Cost | 42,478 | 45,116 | ||
Available-for-sale securities, Gross Unrealized Gains | 5 | 1 | ||
Available-for-sale securities, Gross Unrealized (Losses) | (146) | (26) | ||
Available-for-sale securities, Estimated Fair Value | $ 42,337 | $ 45,091 |
CASH EQUIVALENTS AND MARKETAB_4
CASH EQUIVALENTS AND MARKETABLE SECURITIES - Summary of cost and estimated fair value of short-term investments (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Investments, Debt and Equity Securities [Abstract] | ||
Maturing in one year or less | $ 218,998 | $ 197,676 |
Maturing after one year through five years | 36,700 | 88,169 |
Total | $ 255,698 | $ 285,845 |
CASH EQUIVALENTS AND MARKETAB_5
CASH EQUIVALENTS AND MARKETABLE SECURITIES - Additional Information (Details) - USD ($) | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Dec. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |||||
Realized investment gains (losses) | $ 0 | $ 0 | $ 0 | $ 0 | |
Allowance for credit loss related to marketable securities, not previously recorded | 0 | 0 | 0 | 0 | |
Other impairment charges related to marketable securities | 0 | $ 0 | 0 | $ 0 | |
Allowance for credit loss related to marketable securities | $ 0 | $ 0 | $ 0 |
BASIC AND DILUTED NET LOSS PE_2
BASIC AND DILUTED NET LOSS PER SHARE - Additional Information (Detail) - shares | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Earnings Per Share [Abstract] | ||
Stock options and RSUs outstanding (in shares) | 18,524,016 | 15,838,002 |
MAJOR CUSTOMERS, PARTNERSHIPS_3
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Novartis Institutes for BioMedical Research, Inc. (Detail) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | 9 Months Ended | 12 Months Ended | ||||
Jul. 27, 2020 | Aug. 31, 2020 | Sep. 30, 2022 | Sep. 30, 2021 | Jun. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Dec. 31, 2020 | Dec. 31, 2021 | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||||||
Accounts receivable | $ 4,522 | $ 4,522 | $ 6,013 | ||||||
Novartis Institutes for BioMedical Research, Inc. | |||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||||||
Contract asset | $ 1,500 | ||||||||
Novartis Institutes for BioMedical Research, Inc. | Collaboration And License Agreement | |||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||||||
Proceeds from collaborators | $ 75,000 | ||||||||
Collaborative arrangement transaction price | $ 95,100 | ||||||||
License fee | 75,000 | ||||||||
Collaborative arrangement estimated reimbursable service costs | 20,100 | ||||||||
Accounts receivable | 2,000 | 2,000 | 1,900 | ||||||
Deferred revenue | 17,700 | 17,700 | $ 40,900 | ||||||
Financial advisory fees | $ 1,500 | ||||||||
Percent of initial recognition | 2% | ||||||||
Amortization | $ 200 | $ 200 | $ 500 | $ 500 | |||||
Novartis Institutes for BioMedical Research, Inc. | Collaboration And License Agreement | Achievement Of Specified Preclinical Development Clinical Development And First Commercial Sale Milestones | |||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||||||
Development and sales-based milestone payments to be received | 420,000 | ||||||||
Novartis Institutes for BioMedical Research, Inc. | Collaboration And License Agreement | Achievement Of Commercial Milestones | |||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||||||
Development and sales-based milestone payments to be received | $ 300,000 |
MAJOR CUSTOMERS, PARTNERSHIPS_4
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Revenues Recognized under Agreement (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 102 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Jun. 28, 2022 | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | $ 26,460 | $ 28,563 | $ 84,069 | $ 82,715 | |
Novartis Institutes for BioMedical Research, Inc. | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 9,610 | 11,514 | 29,433 | 28,956 | |
Novartis Institutes for BioMedical Research, Inc. | Recognition of upfront fee | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 7,582 | 9,093 | 23,222 | 22,852 | |
Novartis Institutes for BioMedical Research, Inc. | Research services | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 2,028 | 2,421 | 6,211 | 6,104 | |
Biogen MA, Inc. | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 9,413 | 10,967 | 30,658 | 32,184 | |
Biogen MA, Inc. | Recognition of upfront fee | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 7,306 | 7,306 | 21,918 | 21,918 | |
Biogen MA, Inc. | Research services | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 2,107 | 3,661 | 8,740 | 10,266 | |
Kite Pharma, Inc. | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 6,915 | 6,409 | 19,557 | 19,021 | |
Kite Pharma, Inc. | Recognition of upfront fee | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 6,296 | 6,296 | 18,682 | 18,682 | |
Kite Pharma, Inc. | Research services | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 619 | 113 | 875 | 339 | |
Sanofi | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 0 | (759) | 3,260 | 1,258 | $ 13,500 |
Sanofi | Recognition of upfront fee | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 0 | (1,125) | 677 | (692) | |
Sanofi | Research services | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | 0 | 1,125 | 2,126 | 2,417 | |
Sanofi | Milestone achievement | |||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||||
Revenues | $ 0 | $ (759) | $ 457 | $ (467) |
MAJOR CUSTOMERS, PARTNERSHIPS_5
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Biogen MA, Inc. (Detail) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||||
May 31, 2020 USD ($) | Apr. 30, 2020 USD ($) product_target $ / shares shares | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Dec. 31, 2020 USD ($) | Dec. 31, 2021 USD ($) | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Accounts receivable | $ 4,522 | $ 4,522 | $ 6,013 | |||||
Biogen MA, Inc. | Stock purchase agreement | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Number of shares issued in transaction (in shares) | shares | 24,420,157 | |||||||
Sale of stock, price per share (in dollars per share) | $ / shares | $ 9.2137 | |||||||
Consideration received on transaction | $ 225,000 | |||||||
Standstill restriction period | 3 years | |||||||
Standstill restriction, ownership threshold percentage ownership percentage | 5% | |||||||
Collaborative arrangement transaction price | $ 204,600 | |||||||
Excess consideration received on transaction | 79,600 | |||||||
Collaboration agreement, equity issued | 145,400 | |||||||
Accounts receivable | 1,700 | 1,700 | 2,800 | |||||
Deferred revenue | 132,100 | 132,100 | $ 154,000 | |||||
Recognized portion of equity issued | $ 2,900 | |||||||
Biogen MA, Inc. | Collaboration And License Agreement | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Consideration received on transaction | 204,600 | |||||||
Proceeds from collaborators | $ 125,000 | 125,000 | ||||||
Maximum milestone payment receivable | $ 2,400,000 | |||||||
Number of additional product targets | product_target | 7 | |||||||
Remaining period | 5 years | |||||||
Number of product targets selected | product_target | 3 | |||||||
Research period | 7 years | |||||||
Maximum number of product targets replaced | product_target | 10 | |||||||
Financial advisory fees | $ 7,000 | |||||||
Percent of initial recognition | 2% | |||||||
Portion of contract asset recognized | 4,100 | 4,100 | ||||||
Amortization | $ 100 | $ 100 | $ 400 | $ 400 | ||||
Biogen MA, Inc. | Collaboration And License Agreement | Pre-approval milestone | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Maximum milestone payment receivable | $ 925,000 | |||||||
Biogen MA, Inc. | Collaboration And License Agreement | Sales-based milestone | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Maximum milestone payment receivable | $ 1,500,000 |
MAJOR CUSTOMERS, PARTNERSHIPS_6
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Kite Pharma, Inc. (Detail) $ in Thousands | 1 Months Ended | |||
Sep. 30, 2019 USD ($) | Apr. 30, 2018 USD ($) milestone option | Sep. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||
Accounts receivable | $ 4,522 | $ 6,013 | ||
Kite Pharma, Inc. | Collaboration And License Agreement | ||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||
Milestone payments received | $ 150,000 | |||
Development and sales-based milestone payments to be received | $ 3,000,000 | |||
Initial research term of agreement | 6 years | |||
Number of options to extend initial research term | option | 2 | |||
Extended research term of agreement | 1 year | |||
Separate upfront fee | $ 10,000 | |||
Collaborative arrangement estimated reimbursable service costs for new research plan | $ 3,400 | |||
Collaborative arrangement transaction price | 189,300 | |||
Revenues under agreement | 150,000 | |||
Collaborative arrangement estimated reimbursable service costs | $ 39,300 | |||
Accounts receivable | 600 | 100 | ||
Deferred revenue | $ 37,800 | $ 56,500 | ||
Kite Pharma, Inc. | Collaboration And License Agreement | Achievement of specified research, clinical development, regulatory and first commercial sale milestones | ||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||
Contingent development - and sales-based milestone payments to be received | 1,300,000 | |||
Kite Pharma, Inc. | Collaboration And License Agreement | Achievement of specified sales-based milestones if annual worldwide net sales of licensed products reach specified levels | ||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||
Contingent development - and sales-based milestone payments to be received | $ 1,800,000 | |||
Maximum amount of achieved milestones to receive payment | milestone | 10 |
MAJOR CUSTOMERS, PARTNERSHIPS_7
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Sanofi S.A. (Detail) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 102 Months Ended | ||||
Sep. 06, 2022 USD ($) | Jan. 31, 2014 USD ($) program | Sep. 30, 2022 USD ($) $ / shares | Sep. 30, 2021 USD ($) $ / shares | Sep. 30, 2022 USD ($) product milestone $ / shares | Sep. 30, 2021 USD ($) $ / shares | Jun. 28, 2022 USD ($) | Dec. 31, 2021 USD ($) | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Revenues | $ 26,460 | $ 28,563 | $ 84,069 | $ 82,715 | ||||
Accounts receivable | 4,522 | 4,522 | $ 6,013 | |||||
Decrease in research and development expense | 900 | 900 | ||||||
Increased net loss | $ 53,155 | $ 47,688 | $ 140,305 | $ 140,801 | ||||
Increased earnings per share, diluted (in dollars per share) | $ / shares | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) | ||||
Increased earnings per share, basic (in dollars per share) | $ / shares | $ (0.34) | $ (0.33) | $ (0.93) | $ (0.98) | ||||
Sanofi S.A. | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Number of research programs | program | 2 | |||||||
Revenues under agreement | $ 20,000 | |||||||
Revenues | $ 0 | $ (759) | $ 3,260 | $ 1,258 | $ 13,500 | |||
Number of products approved | product | 0 | |||||||
Post transition assistance period | 90 days | |||||||
Expenses reimbursable through December 31, 2023 (up to) | $ 7,000 | |||||||
Expenses reimbursable after December 31, 2023 (up to) | $ 5,300 | |||||||
Sanofi S.A. | Collaboration And License Agreement | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Revenues under agreement | 20,000 | |||||||
Number of milestones included in transaction price | milestone | 0 | |||||||
Collaborative arrangement transaction price | 96,300 | |||||||
Collaborative arrangement estimated reimbursable service costs | 62,800 | |||||||
Accounts receivable | 0 | $ 0 | 600 | |||||
Deferred revenue | $ 0 | $ 0 | $ 1,100 | |||||
Sanofi S.A. | Collaboration And License Agreement | Milestone three | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Milestone revenue receivable | $ 13,500 | |||||||
Sanofi S.A. | Collaboration agreement | Change In Collaboration Agreement Scope | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Revenues | (2,500) | (2,500) | ||||||
Increased net loss | $ 2,500 | $ 2,500 | ||||||
Increased earnings per share, diluted (in dollars per share) | $ / shares | $ 0.02 | $ 0.02 | ||||||
Increased earnings per share, basic (in dollars per share) | $ / shares | $ 0.02 | $ 0.02 |
MAJOR CUSTOMERS, PARTNERSHIPS_8
MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Pfizer Inc. (Detail) | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | 52 Months Ended | 65 Months Ended | ||||
Sep. 30, 2020 USD ($) | Dec. 31, 2017 USD ($) | May 31, 2017 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Dec. 31, 2020 USD ($) | Mar. 31, 2022 USD ($) milestone product | Sep. 30, 2022 USD ($) product milestone | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Revenues | $ 26,460,000 | $ 28,563,000 | $ 84,069,000 | $ 82,715,000 | ||||||
Pfizer Inc. | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Agreement termination, term | 15 years | |||||||||
Pfizer Inc. | S B Five Two Five And Other Products | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Revenues under agreement | $ 70,000,000 | |||||||||
Potential amount to be funded for achievement of specified commercialized and sales milestones | 266,500,000 | |||||||||
Milestone revenue receivable | 300,000,000 | |||||||||
Pfizer Inc. | S B Five Two Five And Other Products | Achievement of specified clinical development intellectual property and regulatory milestones | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Development and sales-based milestone payments to be received | 208,500,000 | |||||||||
Pfizer Inc. | S B Five Two Five And Other Products | Achievement of first commercial sale milestones | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Development and sales-based milestone payments to be received | 475,000,000 | |||||||||
Pfizer Inc. | Other products | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Milestone revenue receivable | 175,000,000 | |||||||||
Pfizer Inc. | C9ORF72 | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Revenues under agreement | $ 5,000,000 | $ 5,000,000 | ||||||||
Development and sales-based milestone payments to be received | $ 5,000,000 | |||||||||
Collaborative arrangement transaction price | $ 17,000,000 | |||||||||
Pfizer SB-525 | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Revenues under agreement | $ 55,000,000 | |||||||||
Collaborative arrangement transaction price | 134,000,000 | |||||||||
Research service fees | $ 79,000,000 | |||||||||
Revenues | 0 | 0 | 0 | 0 | ||||||
Pfizer SB-525 | S B Five Two Five And Other Products | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Collaborative arrangement, number of milestones achieved | milestone | 2 | |||||||||
Milestone payments received | $ 55,000,000 | |||||||||
Number of products approved | product | 0 | |||||||||
Number of milestones included in transaction price | milestone | 0 | |||||||||
Pfizer C9ORF72 | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Agreement termination, term | 15 years | |||||||||
Milestone payments received | $ 12,000,000 | $ 5,000,000 | ||||||||
Number of products approved | product | 0 | |||||||||
Number of milestones included in transaction price | milestone | 0 | |||||||||
Pfizer C9ORF72 | C9ORF72 | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Revenues under agreement | 12,000,000 | |||||||||
Revenues | $ 0 | $ 0 | $ 0 | $ 0 | ||||||
Pfizer C9ORF72 | C9ORF72 | Achievement Of Specified Preclinical Development Clinical Development And First Commercial Sale Milestones | Maximum | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Development and sales-based milestone payments to be received | 60,000,000 | |||||||||
Pfizer C9ORF72 | C9ORF72 | Achievement Of Commercial Milestones | Maximum | ||||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||||
Development and sales-based milestone payments to be received | $ 90,000,000 |
STOCK-BASED COMPENSATION - Stoc
STOCK-BASED COMPENSATION - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | $ 7,793 | $ 7,873 | $ 23,402 | $ 24,880 |
Research and development | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | 4,395 | 4,935 | 13,656 | 14,645 |
General and administrative | ||||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||||
Total stock-based compensation expense | $ 3,398 | $ 2,938 | $ 9,746 | $ 10,235 |
STOCKHOLDERS' EQUITY (Detail)
STOCKHOLDERS' EQUITY (Detail) - Jefferies LLC - At-The-Market Offering Agreement - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | |||
Oct. 31, 2022 | Aug. 31, 2020 | Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Stock offering program, maximum value | $ 150 | |||||
Issuance of common stock under public offering, net of issuance costs (in shares) | 8,483,104 | 202,705 | 14,711,770 | 2,007,932 | ||
Consideration received on transaction | $ 42.2 | $ 2.4 | $ 66.4 | $ 27.1 | ||
Subsequent Event | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Consideration received on transaction | $ 0.6 |
INCOME TAXES (Details)
INCOME TAXES (Details) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Income Tax Disclosure [Abstract] | ||||
Income tax expense | $ 30,000 | $ 86,000 | $ 170,000 | $ 373,000 |
Employee retention credit, CARES Act | 0 | 3,000,000 | ||
Proceeds from employee retention credit, CARES Act | $ 0 | $ 0 |