Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2024 | May 03, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2024 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 | |
Trading Symbol | NTLA | |
Entity Registrant Name | INTELLIA THERAPEUTICS, INC. | |
Entity Central Index Key | 0001652130 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 96,475,421 | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-37766 | |
Entity Tax Identification Number | 36-4785571 | |
Entity Address, Address Line One | 40 Erie Street | |
Entity Address, Address Line Two | Suite 130 | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 857 | |
Local Phone Number | 285-6200 | |
Entity Incorporation, State or Country Code | DE | |
Title of 12(b) Security | Common Stock, par value $0.0001 per share | |
Security Exchange Name | NASDAQ | |
Document Quarterly Report | true | |
Document Transition Report | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 171,979 | $ 226,748 |
Marketable securities | 619,315 | 685,475 |
Accounts receivable | 36,427 | 36,456 |
Prepaid expenses and other current assets | 56,322 | 49,651 |
Total current assets | 884,043 | 998,330 |
Marketable securities - noncurrent | 162,090 | 99,864 |
Property and equipment, net | 32,075 | 32,760 |
Operating lease right-of-use assets | 110,424 | 115,375 |
Equity method investment | 0 | 11,765 |
Investments and other assets | 70,957 | 42,883 |
Total assets | 1,259,589 | 1,300,977 |
Current liabilities: | ||
Accounts payable | 13,689 | 7,452 |
Accrued expenses | 43,276 | 67,017 |
Current portion of operating lease liability | 18,763 | 18,599 |
Current portion of deferred revenue | 22,184 | 22,140 |
Total current liabilities | 97,912 | 115,208 |
Deferred revenue, net of current portion | 33,440 | 38,853 |
Long-term operating lease liability | 92,100 | 96,747 |
Total liabilities | 223,452 | 250,808 |
Commitments and contingencies (Note 6) | ||
Stockholders' equity: | ||
Common stock, $0.0001 par value; 240,000,000 shares authorized at March 31, 2024 and December 31, 2023; 96,333,373 and 92,997,158 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | 10 | 9 |
Additional paid-in capital | 2,802,889 | 2,710,797 |
Accumulated other comprehensive loss | (947) | (2,258) |
Accumulated deficit | (1,765,815) | (1,658,379) |
Total stockholders’ equity | 1,036,137 | 1,050,169 |
Total liabilities and stockholders' equity | $ 1,259,589 | $ 1,300,977 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2024 | Dec. 31, 2023 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 240,000,000 | 240,000,000 |
Common stock, shares issued | 96,333,373 | 92,997,158 |
Common stock, shares outstanding | 96,333,373 | 92,997,158 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Income Statement [Abstract] | ||
Collaboration revenue | $ 28,935 | $ 12,606 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Operating expenses: | ||
Research and development | $ 111,847 | $ 97,116 |
General and administrative | 31,091 | 27,448 |
Total operating expenses | 142,938 | 124,564 |
Operating loss | (114,003) | (111,958) |
Other income (expense), net: | ||
Interest income | 12,632 | 11,980 |
Change in fair value of investments, net | (6,065) | 0 |
Loss from equity method investment | 0 | (3,048) |
Change in fair value of contingent consideration | 0 | (100) |
Total other income (expense), net | 6,567 | 8,832 |
Net loss | $ (107,436) | $ (103,126) |
Net loss per share, basic | $ (1.12) | $ (1.17) |
Net loss per share, diluted | $ (1.12) | $ (1.17) |
Weighted average shares outstanding, basic | 95,502 | 87,772 |
Weighted average shares outstanding, diluted | 95,502 | 87,772 |
Other comprehensive (loss) gain: | ||
Unrealized gain (loss) on marketable securities | $ (821) | $ 2,989 |
Other comprehensive gain - equity method investment | 0 | 1,794 |
Comprehensive loss | $ (108,257) | $ (98,343) |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | At The Market Offerings [Member] | Common Stock | Common Stock At The Market Offerings [Member] | Additional Paid-In Capital | Additional Paid-In Capital At The Market Offerings [Member] | Accumulated Other Comprehensive Income | Retained Earnings |
Beginning balance at Dec. 31, 2022 | $ 1,235,584 | $ 9 | $ 2,420,223 | $ (7,461) | $ (1,177,187) | |||
Beginning balance, shares at Dec. 31, 2022 | 87,103,007 | |||||||
Issuance of common stock | $ 1,466 | $ 1,466 | ||||||
Issuance of common stock, shares | 35,349 | |||||||
Contingent consideration paid, shares | 567,045 | |||||||
Contingent consideration paid | 24,126 | 24,126 | ||||||
Exercise of stock options | 755 | 755 | ||||||
Exercise of stock options, shares | 48,353 | |||||||
Vesting of restricted stock units, shares | 342,025 | |||||||
Stock-based compensation | 27,255 | 27,255 | ||||||
Other comprehensive gain (loss) - unrealized gain (loss) on marketable securities | 2,989 | 2,989 | ||||||
Other comprehensive gain - equity method investment | 1,794 | 1,794 | ||||||
Net Income (Loss) | (103,126) | (103,126) | ||||||
Ending balance at Mar. 31, 2023 | 1,190,843 | $ 9 | 2,473,825 | (2,678) | (1,280,313) | |||
Ending balance, shares at Mar. 31, 2023 | 88,095,779 | |||||||
Beginning balance at Dec. 31, 2023 | $ 1,050,169 | $ 9 | 2,710,797 | (2,258) | (1,658,379) | |||
Beginning balance, shares at Dec. 31, 2023 | 92,997,158 | 92,997,158 | ||||||
Issuance of common stock | $ 55,959 | $ 1 | $ 55,958 | |||||
Issuance of common stock, shares | 2,209,938 | |||||||
Exercise of stock options | $ 1,958 | 1,958 | ||||||
Exercise of stock options, shares | 110,734 | 110,734 | ||||||
Vesting of restricted stock units, shares | 1,015,543 | |||||||
Stock-based compensation | $ 34,176 | 34,176 | ||||||
Other comprehensive gain (loss) - unrealized gain (loss) on marketable securities | (821) | (821) | ||||||
Other comprehensive gain - equity method investment | 0 | |||||||
Reclassification of other comprehensive loss - equity method investment | 2,132 | 2,132 | ||||||
Net Income (Loss) | (107,436) | (107,436) | ||||||
Ending balance at Mar. 31, 2024 | $ 1,036,137 | $ 10 | $ 2,802,889 | $ (947) | $ (1,765,815) | |||
Ending balance, shares at Mar. 31, 2024 | 96,333,373 | 96,333,373 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
At The Market Offerings [Member] | ||
Stock issuance cost, net | $ 210 | $ 62 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | ||
Net loss | $ (107,436) | $ (103,126) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 2,512 | 2,050 |
Stock-based compensation | 34,176 | 27,255 |
Accretion of investment discounts and premiums | (5,285) | (4,571) |
(Recognition) deferral of equity method investment intra-entity profit on sales | (20,967) | 2,812 |
Change in fair value of investments | 6,065 | 0 |
Loss from equity method investment | 0 | 3,048 |
Change in fair value of contingent consideration | 0 | 100 |
Changes in operating assets and liabilities: | ||
Accounts receivable | 29 | (1,138) |
Prepaid expenses and other current assets | (8,746) | (4,815) |
Operating lease right-of-use assets | 4,951 | 4,658 |
Other assets | 724 | (393) |
Accounts payable | 5,569 | 1,170 |
Accrued expenses | (22,403) | (17,392) |
Deferred revenue | (5,368) | (14,566) |
Operating lease liabilities | (4,483) | (4,420) |
Net cash used in operating activities | (120,662) | (109,328) |
CASH FLOWS FROM INVESTING ACTIVITIES: | ||
Purchases of property and equipment | (2,506) | (3,832) |
Purchases of marketable securities | (259,558) | (382,033) |
Maturities of marketable securities | 267,965 | 263,617 |
Net cash provided by (used in) investing activities | 5,901 | (122,248) |
CASH FLOWS FROM FINANCING ACTIVITIES: | ||
Proceeds from options exercised | 1,958 | 755 |
Net cash provided by financing activities | 59,992 | 2,221 |
Net decrease in cash, cash equivalents and restricted cash equivalents | (54,769) | (229,355) |
Cash, cash equivalents and restricted cash equivalents, beginning of period | 240,353 | 535,463 |
Cash, cash equivalents and restricted cash equivalents, end of period | 185,584 | 306,108 |
Reconciliation of cash, cash equivalents and restricted cash equivalents to condensed consolidated balance sheet: | ||
Cash and cash equivalents | 171,979 | 294,150 |
Restricted cash equivalents, included in investments and other assets | 13,605 | 11,958 |
Total cash, cash equivalents and restricted cash equivalents | 185,584 | 306,108 |
SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION: | ||
Purchases of property and equipment unpaid at period end | 847 | 3,525 |
Shares issued for Rewrite contingent consideration | 0 | 24,126 |
At The Market Offerings [Member] | ||
CASH FLOWS FROM FINANCING ACTIVITIES: | ||
Net proceeds from issuance of common stock through at-the-market offerings | $ 58,034 | $ 1,466 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Pay vs Performance Disclosure | ||
Net Income (Loss) | $ (107,436) | $ (103,126) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | Rule 10b5-1 Trading Plans The following table describes for the three months ended March 31, 2024 each trading arrangement under which the Company’s directors or officers adopted, materially modified , or terminated any contract, instruction, or written plan for the purchase or sale of Company securities that was intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) or any non-Rule 10b5-1 trading arrangement. Name (Title) Action Taken (Date of Action) Type of Trading Arrangement Nature of Trading Arrangement Duration of Trading Arrangement Aggregate Number of Securities David Lebwohl, M.D . ( Executive Vice President, Chief Medical Officer ) Adoption ( March 7, 2024 ) Rule 10b5-1 trading arrangement Sale Until the earlier of (a) March 7, 2025 ; (b) the first date on which all trades have been executed or expired without execution; and (c) the date on which the plan holder gives notice to terminate the plan. Up to 30,000 shares of the Company’s common stock Frank Verwiel, M.D . (Director ) Adoption ( March 7, 2024 ) Rule 10b5-1 trading arrangement Sale Until the earlier of (a) July 8, 2024 ; (b) the first date on which all trades have been executed or expired without execution; and (c) the date on which the plan holder gives notice to terminate the plan. Up to 1,504 shares of the Company’s common stock |
David Lebwohl, M.D [Member] | |
Trading Arrangements, by Individual | |
Name | David Lebwohl, M.D |
Title | Executive Vice President, Chief Medical Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | March 7, 2024 |
Rule 10b5-1 Arrangement Terminated | true |
Arrangement Duration | 1 year |
Aggregate Available | 30,000 |
Rule 10b5-1 Arrangement Modified | modified |
Trading Arrangement Expiration Date | March 7, 2025 |
Frank Verwiel, M.D [Member] | |
Trading Arrangements, by Individual | |
Name | Frank Verwiel, M.D |
Title | (Director |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | March 7, 2024 |
Rule 10b5-1 Arrangement Terminated | true |
Arrangement Duration | 4 months 1 day |
Aggregate Available | 1,504 |
Rule 10b5-1 Arrangement Modified | modified |
Trading Arrangement Expiration Date | July 8, 2024 |
Overview and Basis of Presentat
Overview and Basis of Presentation | 3 Months Ended |
Mar. 31, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Overview and Basis of Presentation | 1. Overview and Basis of Presentation Intellia Therapeutics, Inc. (“Intellia” or the “Company”) is a leading clinical-stage gene editing company, focused on developing potentially curative therapeutics using CRISPR/Cas9-based technologies. CRISPR/Cas9, an acronym for C lustered, R egularly I nterspaced S hort P alindromic R epeats (“CRISPR”)/CRISPR associated 9 (“Cas9”), is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid (“DNA”). To fully realize the transformative potential of CRISPR/Cas9-based technologies, Intellia is building a full-spectrum gene editing company, by leveraging its modular platform, to advance in vivo and ex vivo therapies for diseases with high unmet need by pursuing two primary approaches. For in vivo applications to address genetic diseases, the Company deploys CRISPR/Cas9 as the therapy. The Company’s in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body. In addition, the Company is advancing ex vivo applications to address immuno-oncology and autoimmune diseases, where it uses CRISPR/Cas9 as the tool to create the engineered cell therapy. For its ex vivo programs, CRISPR/Cas9 is used to engineer human cells outside the body. The Company’s deep scientific, technical and clinical development experience, along with its robust intellectual property (“IP”) portfolio, have enabled it to unlock broad therapeutic applications of CRISPR/Cas9 and related technologies to create new classes of genetic medicine. The condensed consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these condensed consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2023. The unaudited condensed consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its wholly- owned subsidiary, Intellia Securities Corp. All intercompany balances and transactions have been eliminated in consolidation. Comprehensive loss is comprised of net loss, unrealized gain/loss on marketable securities and other comprehensive gain/loss from equity method investment. The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates, judgments and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these condensed consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses, valuation of equity and fair value method investments, contingent consideration and stock-based compensation expense. The Company bases its estimates on historical experience and various other assumptions that management believes to be reasonable under the circumstances at the time such estimates are made. Actual results could differ from those estimates. The Company periodically reviews its estimates in light of changes in circumstances, facts and experience. The effects of material revisions in estimates, if any, would be reflected in the condensed consolidated financial statements prospectively from the date of the change in estimate. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period. Liquidity Since its inception through March 31, 2024 , the Company has raised an aggregate of $ 2,598.3 million to fund its operations through its initial public offering (“IPO”) and concurrent private placements, follow-on public offerings, at-the-market offerings and the sale of convertible preferred stock, as well as through its collaboration agreements. The Company expects that its cash, cash equivalents and marketable securities as of March 31, 2024 will enable the Company to fund its ongoing operating expenses and capital expenditure requirements for at least the twelve-month period following the issuance of these condensed consolidated financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies” to the consolidated financial statements included in the Annual Report for the year ended December 31, 2023. There have been no material changes to these policies during the three months ended March 31, 2024. Recent Issued Accounting Pronouncements Not Yet Effective In November 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2023-07, “Segment Reporting - Improvements to Reportable Segment Disclosures.” The ASU requires disclosure of incremental segment information on an annual and interim basis and also requires companies with a single reportable segment to provide all disclosures required by this ASU and all existing segment disclosures in Accounting Standard Codification (“ASC”) 280, “Segment Reporting.” The ASU is effective for fiscal years beginning after December 15, 2023, and interim periods beginning after December 15, 2024. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures. In December 2023, the FASB issued ASU No. 2023-09, “Income Taxes (Topic 740): Improvements to Income Tax Disclosures.” This ASU updates income tax disclosure requirements primarily by requiring specific categories and greater disaggregation within the rate reconciliation and disaggregation of income taxes paid by jurisdiction. This ASU is effective for annual periods beginning after December 15, 2024 and is applicable to the Company’s fiscal year beginning January 1, 2025, with early application permitted. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures. |
Marketable Securities
Marketable Securities | 3 Months Ended |
Mar. 31, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | 3. Marketable Securities The following table summarizes the Company’s available-for-sale marketable securities as of March 31, 2024 and December 31, 2023: March 31, 2024 Amortized Gross Unrealized Gross Unrealized Estimated Fair (In thousands) Marketable securities: U.S. Treasury and other government-backed securities $ 401,660 $ 40 $ ( 589 ) $ 401,111 Financial institution debt securities 214,001 56 ( 292 ) 213,765 Corporate debt securities 112,194 12 ( 129 ) 112,077 Other asset-backed securities 54,496 3 ( 47 ) 54,452 Total $ 782,351 $ 111 $ ( 1,057 ) $ 781,405 December 31, 2023 Amortized Gross Unrealized Gross Unrealized Estimated Fair (In thousands) Marketable securities: U.S. Treasury and other government-backed securities $ 382,260 $ 302 $ ( 254 ) $ 382,308 Financial institution debt securities 246,270 92 ( 243 ) 246,119 Corporate debt securities 97,490 53 ( 135 ) 97,408 Other asset-backed securities 59,453 75 ( 24 ) 59,504 Total $ 785,473 $ 522 $ ( 656 ) $ 785,339 The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. There were no material realized gains or losses in the three months ended March 31, 2024 or for the year ended December 31, 2023. The Company generally does not intend to sell any investments prior to recovery of their amortized cost basis for any investment in an unrealized loss position. As such, the Company has classified these losses as temporary in nature. The Company’s available-for-sale securities that are classified as short-term marketable securities in the condensed consolidated balance sheet mature within one year or less as of the balance sheet date. Available-for-sale securities that are classified as noncurrent in the condensed consolidated balance sheet are those that mature after one year but within five years from the balance sheet date and that the Company does not intend to dispose of within the next twelve months. At March 31, 2024 and December 31, 2023, the Company did no t hold any marketable securities that matured beyond five years of the balance sheet date. |
Fair Value Measurements
Fair Value Measurements | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 4. Fair Value Measurements The Company classifies fair value-based measurements using a three-level hierarchy that prioritizes the inputs used to measure fair value. This hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: Level 1, quoted market prices (unadjusted) in active markets for identical assets or liabilities; Level 2, observable inputs other than quoted market prices included in Level 1, such as quoted market prices for markets that are not active or other inputs that are observable or can be corroborated by observable market data; and Level 3, unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities, including certain pricing models, discounted cash flow methodologies and similar techniques that use significant unobservable inputs. T he Company’s financial assets recognized at fair value on a recurring basis consisted of the following: March 31, 2024 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 143,500 $ 143,500 $ - $ - Marketable securities: U.S. Treasury and other government-backed securities 401,111 179,434 221,677 - Financial institution debt securities 213,765 - 213,765 - Corporate debt securities 112,077 - 112,077 - Other asset-backed securities 54,452 - 54,452 - Total marketable securities 781,405 179,434 601,971 - Investment in Kyverna Therapeutics, Inc. 29,158 29,158 - - Total assets $ 954,063 $ 352,092 $ 601,971 $ - December 31, 2023 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 136,254 $ 136,254 $ - $ - Marketable securities: U.S. Treasury and other government-backed securities 382,308 120,556 261,752 - Financial institution debt securities 246,119 - 246,119 - Corporate debt securities 97,408 - 97,408 - Other asset-backed securities 59,504 - 59,504 - Total marketable securities 785,339 120,556 664,783 - Total assets $ 921,593 $ 256,810 $ 664,783 $ - Certain of the Company’s financial assets, including cash equivalents, restricted cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued at the end of each reporting period, utilizing third party pricing services or other observable market data. The pricing services utilize industry standard valuation models and observable market inputs to determine value. Other financial instruments, including accounts receivable, accounts payable and accrued expense, are carried at cost, which approximates fair value due to the short duration and term to maturity. The Company has determined that the estimated fair value of its investment in Kyverna Therapeutics, Inc. (“Kyverna”), a publicly traded company, is reported as Level 1 as it is valued at a quoted market price in an active market. The investment in Kyverna is classified within “investments and other assets” in the condensed consolidated balance sheets. Refer to Note 8 for further details. Other Investments The Company’s other investments include investments in AvenCell Therapeutics, Inc. (“AvenCell”) and SparingVision Sas (“SparingVision”). These investments are accounted for under ASC 321, “Investments in Equity Securities” (“ASC 321”) using the measurement alternative at cost minus impairment with adjustments for changes in observable prices . The Company previously accounted for the AvenCell investment under the equity method; refer to Note 8 for further details. The Company monitors any events or changes in circumstances that may have a significant effect on the fair value of investments, either due to impairment or based on observable price changes, and records adjustments as needed. These investments are classified as Level 3 assets and are not included in the fair value table above as they are not valued at fair value on a recurring basis. |
Accrued Expenses
Accrued Expenses | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 5. Accrued Expenses Accrued expenses consisted of the following: March 31, December 31, 2024 2023 (In thousands) Accrued research and development $ 21,197 $ 27,411 Employee compensation and benefits 10,386 26,615 Accrued legal and professional expenses 3,808 2,063 Accrued construction costs 3,995 6,891 Accrued other 3,890 4,037 Total accrued expenses $ 43,276 $ 67,017 |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Litigation There have been no material changes to any outstanding litigation, nor is the Company a party to any new litigation, since December 31, 2023. For further information please see the notes to the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2023. License Agreements The Company is party to license agreements, which may include contingent payments. These payments will become payable if and when certain development, regulatory and commercial milestones are achieved. As of March 31, 2024 , the satisfaction and timing of the contingent payments is uncertain and not reasonably estimable. |
Collaborations and Other Arrang
Collaborations and Other Arrangements | 3 Months Ended |
Mar. 31, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborations and Other Arrangements | 7. Collaborations and Other Arrangements To accelerate the development and commercialization of CRISPR/Cas9-based products in multiple therapeutic areas, the Company has formed, and intends to seek other opportunities to form, strategic alliances with collaborators who can augment its leadership in CRISPR/Cas9 therapeutic development. As of March 31, 2024, the Company’s accounts receivable were related to its collaborations with Regeneron Pharmaceuticals, Inc. (“Regeneron”), SparingVision, Kyverna and AvenCell, and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision. As of December 31, 2023, the Company’s accounts receivable were related to its collaborations with Regeneron, SparingVision, AvenCell and Kyverna and the Company’s contract liabilities were related to its collaborations with Regeneron and SparingVision. The following table presents changes in the Company’s accounts receivable and contract liabilities during the three months ended March 31, 2024 and 2023 (in thousands): Balance at Additions Deductions Balance at End Three months ended March 31, 2024 Accounts receivable $ 36,456 $ 5,872 $ ( 5,901 ) $ 36,427 Contract liabilities - deferred revenue $ 60,993 $ - $ ( 5,369 ) $ 55,624 Balance at Additions Deductions Balance at End Three months ended March 31, 2023 Accounts receivable $ 3,768 $ 4,488 $ ( 3,350 ) $ 4,906 Contract liabilities - deferred revenue $ 63,771 $ - $ ( 14,566 ) $ 49,205 During the three months ended March 31, 2024 and 2023, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Three Months Ended March 31, Revenue recognized in the period from: 2024 2023 Amounts included in the contract liability at the beginning of the period $ 5,369 $ 11,753 The Company has not incurred significant expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period. Regeneron Pharmaceuticals, Inc. In April 2016, the Company entered into a license and collaboration agreement with Regeneron (as amended from time to time, the “2016 Regeneron Agreement”). In October 2023, Regeneron exercised its one-time option to extend the technology collaboration term for an additional two years (the “2024 Technology Collaboration Extension”) , until April 2026 , in exchange for a nonrefundable payment of $ 30.0 million, which was received by the Company in April 2024. In September 2023, Regeneron and Intellia further expanded the research collaboration (the “2023 Regeneron Amendment”) to develop additional in vivo CRISPR-based gene editing therapies focused on neurological and muscular diseases. In 2018, the Company entered into a co-development and co-promotion (“Co/Co”) agreement with Regeneron for transthyretin (“ATTR”) amyloidosis (the “ATTR Co/Co”). In May 2020, the Company entered into co-development and co-funding agreements for the treatment of hemophilia A and hemophilia B (the “Hemophilia Co/Co”) agreements. In March 2024, the Company notified Regeneron that it is opting out of its hemophilia B Co/Co agreement. The Company will continue to have obligations under the hemophilia B Co/Co agreement until September 2024. In addition, after termination of the hemophilia B Co/Co agreement the Company will continue to support Regeneron with the development of gene editing products directed to hemophilia B, as applicable, under the 2016 Regeneron Agreement. That agreement will control the parties’ obligations to develop and commercialize gene editing products directed to hemophilia B. The Company may be eligible to receive up to $ 320.0 million in milestone payments and royalties in the high-single digits to low teens, which royalties are potentially subject to various reductions, offsets and upstream payment obligations. Since December 31, 2023, there have been no material changes to the key terms of the 2016 Regeneron Agreement, ATTR Co/Co, Hemophilia Co/Co, or 2023 Regeneron Amendment (the “Regeneron Agreements”), other than as described above. The Company recognized collaboration revenue of $ 7.4 million and $ 6.8 million during the three months ended March 31, 2024 and 2023, respectively . This includes $ 4.7 million and $ 4.2 million during the three months ended March 31, 2024 and 2023, respectively , primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement. These revenues are offset in part by contra-revenue related to the Hemophilia Co/Co agreements amounting to approximately $ 3.3 million and $ 2.9 million during the three months ended March 31, 2024 and 2023, respectively. As of March 31, 2024 , there was $ 41.9 million of the aggregate transaction price remaining to be recognized through April 2026, the remaining period of the collaboration . As of March 31, 2024 and December 31, 2023 , the Company had accounts receivable of $ 35.9 million and $ 35.7 million, respectively, and deferred revenue of $ 41.9 million and $ 47.1 million, respectively, related to the Regeneron Agreements. SparingVision SAS In October 2021, the Company and SparingVision, a genomic medicine company developing vision saving treatments for ocular diseases, entered into a license and collaboration agreement (the “SparingVision LCA”) to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. Since December 31, 2023, there have been no material changes to the key terms of the SparingVision LCA agreement. The Company recognized collaboration revenue of $ 0.5 million and $0 .4 million related to the SparingVision LCA for the three months ended March 31, 2024 and 2023, respectively. As of March 31, 2024 and December 31, 2023 , the Company had $ 0.3 million and $ 0.5 million in accounts receivable, respectively, related to the SparingVision LCA. As of March 31, 2024 and December 31, 2023 , the Company had deferred revenue of $ 13.7 million and $ 13.9 million, respectively, related to the SparingVision LCA, which is expected to be recognized over a six to nine year period from the signing of the agreement. ReCode Therapeutics, Inc. (“ReCode”) On February 14, 2024, the Company entered into a license, collaboration and option agreement with ReCode (the “ReCode LCA”), a clinical-stage genetic medicines company, to develop novel genomic medicines for the treatment of cystic fibrosis (“CF”). The ReCode LCA leverages the Company’s proprietary CRISPR-based gene editing platform, including its DNA writing technology, and ReCode’s proprietary Selective Organ Targeting (“SORT”) lipid nanoparticle delivery platform to precisely correct one or more CF disease-causing gene mutations. As part of the agreement, the companies will focus initial research efforts on therapeutic approaches that address CF for patients who have limited or no treatment options available, with the opportunity to expand the scope of the collaboration in later phases. The Company will be responsible for the design of the editing strategy and research-grade components for the investigational therapies. ReCode will lead the subsequent preclinical and clinical development and worldwide commercialization for certain programs arising from the collaboration. The Company also has an option to lead commercialization in the U.S. for certain programs (the “Co/Co option”). The ReCode LCA did not include an exchange of upfront consideration between the parties. The Company will be eligible to receive pre-specified development and commercial milestone payments, up to $ 262.0 million per product, as well as single digit royalties on potential sales. Certain milestone and royalty payments may be removed or reduced for a product if the Company exercises the Co/Co option. The Company did not recognize any revenue from the ReCode LCA during the three months ended March 31, 2024. Other Agreements The Company has existing license and collaboration agreements with AvenCell, Kyverna, and ONK Therapeutics, Ltd (“ONK”) . Since December 31, 2023, there have been no material changes to the key terms of the AvenCell, Kyverna and ONK license and collaboration agreements. During the three months ended March 31, 2024, the Company recognized $ 21.0 million of previously eliminated intra-entity profit related to the AvenCell agreement. See Note 8 for further details. The Company did not recognize material revenue from the Kyverna and ONK agreements during that period. During the three months ended March 31, 2023, the Company recognized $ 4.9 million, $ 0.4 million and $ 0.1 million in revenue related to AvenCell, Kyverna and ONK, respectively. |
Investments and Other Assets
Investments and Other Assets | 3 Months Ended |
Mar. 31, 2024 | |
Investments, All Other Investments [Abstract] | |
Investments and Other Assets | 8. Investments and Other Assets Investments and other assets consisted of the following: March 31, December 31, 2024 2023 (In thousands) Investment in Kyverna $ 29,158 $ 10,000 Other investments 24,213 14,760 Restricted cash, long-term 13,605 13,605 Prepaid expenses and other assets, long-term 3,981 4,518 Total investments and other assets $ 70,957 $ 42,883 Kyverna Therapeutics, Inc. In February 2024, Kyverna completed an initial public offering of its common stock (the “Kyverna IPO”). Prior to the Kyverna IPO, the Company accounted for its investment in Kyverna using the measurement alternative as Kyverna was a private company with no readily observable transaction price, and the investment was valued at $ 10.0 million as of December 31, 2023. As of March 31, 2024, the Company’s investment in Kyverna is valued at $ 29.2 million. The Company recognized an unrealized gain of $ 19.2 million recorded within “change in fair value of investments, net” in the condensed consolidated statement of operations and comprehensive loss during the three months ended March 31, 2024, associated with changes in the fair value of Kyverna’s common stock. AvenCell Therapeutics, Inc. As of December 31, 2023 and March 31, 2024, the Company holds a 33.33 % equity interest in AvenCell. As of December 31, 2023, the Company accounted for its investment using the equity method of accounting, as the Company had significant influence, but not control, over AvenCell, and the investment was valued at $ 11.8 million. During the first quarter of 2024, in conjunction with the completion of a debt financing, AvenCell increased the size of their board, with a single investor having control over AvenCell’s operational and financial decisions. As a result, the Company no longer has the ability to exercise significant influence over AvenCell, and therefore the Company’s investment in AvenCell is accounted for in accordance with ASC 321 as of March 31, 2024, and AvenCell is no longer considered to be a related party. The Company did no t recognize any revenue related to the AvenCell LCA during the three months ended March 31, 2024. During the three months ended March 31, 2023, the Company recognized $ 4.9 million in revenue related to the AvenCell LCA. The Company had $ 0.2 million in accounts receivable and $ 1.0 million in accrued expenses related to the AvenCell agreements as of both March 31, 2024 and December 31, 2023. The transition from equity method accounting to ASC 321 required the Company to reclassify $ 2.1 million from accumulated other comprehensive loss amounts previously recognized as a result of the investment in AvenCell to the carrying value of the investment in AvenCell and recognize $ 21.0 million of previously eliminated intra-entity profit as “collaboration revenue” in the condensed consolidated statement of operations and comprehensive loss during the three months ended March 31, 2024. The Company also considered whether there was an indication that the investment was impaired, and recorded an impairment of $ 25.3 million within “change in fair value of investments, net” in the condensed consolidated statement of operations and comprehensive loss for the three months ended March 31, 2024. This impairment was caused by AvenCell’s financial condition, indicators of fair value based on the completion of the debt financing, and an executed term sheet for further financing, which indicated that the fair value of the investment was less than its carrying value. The Company used a market approach to value the investment, which is a Level 3 measurement in the fair value hierarchy. As of March 31, 2024, the carrying value of the Company’s investment in AvenCell was $ 9.6 million. SparingVision SAS As of March 31, 2024, the fair value of the Company’s investment in SparingVision was $ 14.6 million. There have been no material changes in the valuation of the SparingVision investment as of March 31, 2024. |
Leases
Leases | 3 Months Ended |
Mar. 31, 2024 | |
Leases [Abstract] | |
Leases | 9. Leases The Company leases approximately 230,000 square feet of real estate, including laboratory and office space in Cambridge, Massachusetts, and the surrounding areas. The Company’s leases have remaining terms ranging from one to approximately nine years. Certain leases include options to renew, exercised at the Company’s sole discretion, with varying renewal terms that can extend the lease term for an additional three to five years. All of the Company’s leases qualify as operating leases. Separately, the Company entered into an agreement in February 2022, later amended in June 2023, to lease approximately 140,000 square feet of office, general laboratory and planned good manufacturing practice (“GMP”) manufacturing space at 840 Winter Street in Waltham , Massachusetts (the “840 Winter Lease”). The Company anticipates a phased move-in process during the second half of 2024. As of March 31, 2024, the Company had not taken control of the premises and therefore there are no right of use assets or liabilities recorded related to the 840 Winter Lease under ASC 842, “Leases (Topic 842)” (“ASC 842”). |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | 10. Stock-Based Compensation Stock-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, 2024 2023 (In thousands) Research and development $ 20,161 $ 16,931 General and administrative 14,015 10,324 Total $ 34,176 $ 27,255 Stock Option and Incentive Plan In April 2016, the Company adopted the Amended and Restated 2015 Stock Option and Incentive Plan (the “2015 Plan”). The 2015 Plan provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards (“RSAs”), restricted stock units (“RSUs”) and other stock-based awards. Recipients of incentive stock options and non-qualified stock options are eligible to purchase shares of the Company’s common stock at an exercise price equal to the fair value of such stock on the grant date. The Company maintains a retirement policy for equity awards granted to all employees, which applies to all equity awards granted after July 1, 2022 to employees who meet certain retirement eligibility criteria. As of March 31, 2024 , there were 4,365,763 shares available for future issuance under the 2015 Plan. The number of shares reserved for issuance under the 2015 Plan will be cumulatively increased on each January 1 st by four percent of the number of shares of stock issued and outstanding on the immediately preceding December 31 st or such lesser number of shares of stock as determined by the board of directors. Restricted Stock Units The following table summarizes the Company’s RSU activity for the three months ended March 31, 2024: Number of Weighted Unvested restricted stock units as of December 31, 2023 4,041,753 $ 50.40 Granted 2,939,011 34.52 Vested ( 1,015,543 ) 51.62 Cancelled ( 270,591 ) 46.14 Unvested restricted stock units as of March 31, 2024 5,694,630 $ 42.18 Restricted Stock Units - Service Awards The Company awards RSUs with a service condition to all employees upon hire and as part of their annual grant. RSUs with a service condition granted under the 2015 Plan in 2024 and 2023 generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting annually over the remaining two years. In March 2024, the Company granted 2,157,921 RSUs with a service condition to employees as part of their annual grant, which have the potential to vest over a period of three years . The weighted average grant date fair value of these RSUs was $ 32.66 and the vesting start date for these RSUs was January 1, 2024. In March 2023, the Company granted 2,195,135 RSUs with a service condition to employees as part of their annual grant, which have the potential to vest over a period of three years . The weighted average grant date fair value of these RSUs was $ 40.75 and the vesting start date for these RSUs was January 1, 2023. Unvested restricted stock units as of March 31, 2024 in the table above includes 4,733,350 RSUs that are service-based. Restricted Stock Units - Market Awards In March 2024, 2023 and 2022, market-based RSUs were granted to senior executives as part of their annual grant . These RSUs have the potential to vest after a period of three years , with a vesting start date of January 1, 2024, 2023 and 2022, respectively, and the number of shares to be delivered will depend on the Company ’s Total Shareholder Return (“TSR” ), a market condition, over that period relative to a defined group of biotechnology companies. The number of market-based RSUs granted in March 2024, 2023 and 2022 was 235,858 , 181,743 and 55,144 , respectively. The grant date fair value for the market-based RSUs, calculated using a Monte Carlo valuation model, was $ 51.12 , $ 68.55 and $ 126.49 , respectively. The following assumptions were used to determine the grant date fair value for the three years, respectively: risk free interest rate: 4.28 %, 4.60 % and 1.44 %; expected volatility: 77.2 %, 84.34 % and 82.53 %. The expected term for all grants was approximately 3.0 years; the expected dividend yield was 0.0 %. Unvested restricted stock units as of March 31, 2024 in the table above includes 472,745 RSUs that are market-based. Restricted Stock Units - Performance-Based Awards with TSR Multiplier Also in March 2024, 444,117 performance-based RSUs ( “PSUs”) with a relative TSR modifier were granted to senior executives as part of their annual grant . These PSUs, to the extent earned, shall vest on January 1, 2027, and the number of shares to be delivered will be determined based upon the achievement of certain performance goals, which can range from 0 % to 200 %. Following the determination of the achievement of performance criteria, the amount of shares awarded will be subject to adjustment based on the application of a TSR modifier, which can range from 75 % to 125 %. The grant date fair value for these PSUs, calculated using a Monte Carlo valuation model, was $ 36.16 . The following assumptions were used to determine the grant date fair value: risk free interest rate: 4.28 %; expected volatility: 77.2 %; expected term approximately 3.0 years; expected dividend yield: 0.0 %. The Company recognizes compensation expense ratably over the required service period based on its estimate of the number of shares that will vest based upon the probability of achieving the performance goals. Unvested restricted stock units as of March 31, 2024 in the table above includes 444,117 PSUs with a TSR multiplier. Restricted Stock Units - Performance-Based Awards In March 2022, the Company granted 66,296 performance-based RSUs to certain non-executive employees that would vest upon obtaining certain scientific milestones. There were two separate tranches, each attached to a different set of milestones. The milestone related to the first tranche, made up of 21,878 RSUs, was achieved in the first quarter of 2023 and these RSUs vested. The remaining performance milestones were considered not probable of achievement as of March 31, 2024 and, therefore, no related stock-based compensation was recorded during the period then ending. Unvested restricted stock units as of March 31, 2024 in the table above includes 44,418 performance-based RSUs. The weighted-average grant date fair value of all RSUs granted during the three months ended March 31, 2024 and 2023 was $ 34.52 and $ 42.78 , respectively. The total fair value of RSUs vested (measured on the date of vesting) for the three months ended March 31, 2024 and 2023 was $ 30.9 million and $ 12.1 million, respectively. As of March 31, 2024 , there was $ 191.3 million of unrecognized stock-based compensation expense related to all RSUs that are expected to vest. These costs are expected to be recognized over a weighted average remaining vesting period of 2.0 years . Stock Options The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $ 22.61 and $ 28.82 per option for those options granted during the three months ended March 31, 2024 and 2023 , respectively. Weighted average assumptions used to apply this pricing model were as follows: Three Months Ended March 31, 2024 2023 Risk-free interest rate 4.2 % 4.4 % Expected term 6.0 years 6.0 years Expected volatility of underlying stock 76.4 % 78.7 % Expected dividend yield 0.0 % 0.0 % Risk-free Interest Rate. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant with maturities approximately equal to the option’s expected term. Expected Term. The expected term represents the period that stock option awards are expected to be outstanding. For option grants that are considered to be “plain vanilla,” the Company determines the expected term using the simplified method. The simplified method deems the term to be the average of the time-to-vesting and the contractual life of the options. The Company uses the simplified method because it does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate the expected term. Expected Volatility. Expected volatility is estimated based on actual movements in the Company’s stock price over the most recent historical periods, over the expected term of their stock option grants. Expected Dividend Yield. The Company has not paid cash dividends and has no intention to pay cash dividends in the future. Stock options generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining two years, unless they contain specific vesting provisions. The maximum term of stock options granted under the 2015 Plan is ten years. The following is a summary of stock option activity for the three months ended March 31, 2024: Number of Weighted Weighted Aggregate (In years) (In thousands) Outstanding at December 31, 2023 5,458,999 $ 50.38 Granted 680,804 32.66 Exercised ( 110,734 ) 17.68 Forfeited ( 119,562 ) 85.00 Outstanding at March 31, 2024 5,909,507 $ 48.25 6.66 $ 27,623 Exercisable at March 31, 2024 4,261,853 $ 44.98 5.86 $ 27,477 The total intrinsic value (the amount by which the fair market value exceeded the exercise price) of stock options exercised during the three months ended March 31, 2024 and 2023 was $ 1.2 million and $ 1.1 million, respectively. As of March 31, 2024 , there was $ 55.9 million of unrecognized compensation cost related to stock options that have not yet vested which are expected to be recognized over a weighted average remaining vesting period of 1.41 years. Employee Stock Purchase Plan In May 2016, the Company adopted the 2016 Employee Stock Purchase Plan (the “2016 Plan”). The 2016 Plan allows eligible employees to purchase shares of the Company’s common stock on the last day of each predetermined six-month offering period at 85 % of the lower of the fair market value per share at the beginning or end of the applicable offering period. The 2016 Plan provides for six-month offering periods beginning in January and July of each year. As of March 31, 2024 , there were 1,077,487 shares available for future issuance under the 2016 Plan. The number of shares reserved for issuance under the 2016 Plan will be cumulatively increased on each January 1 st by the lesser of a) one percent of the number of shares of common stock issued and outstanding on the immediately preceding December 31 st , b) 500,000 shares of common stock, or c) such lesser number of shares of common stock as determined by the board of directors. During the three months ended March 31, 2024 and 2023 the Company did no t issue any shares of common stock under the 2016 Plan. The fair value of shares under the 2016 Plan was estimated at the beginning of the offering period using a Black-Scholes option-pricing model with the following assumptions: Three Months Ended March 31, 2024 2023 Risk-free interest rate 5.24 % 4.70 % Expected term 0.5 years 0.5 years Expected volatility of underlying stock 56.7 % 69.2 % Expected dividend yield 0.0 % 0.0 % |
Loss Per Share
Loss Per Share | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Loss Per Share | 11. Loss Per Share The Company calculates basic loss per share by dividing net loss for each respective period by the weighted average number of common shares outstanding for each respective period. The Company computes diluted loss per share after giving consideration to the dilutive effect of stock options and unvested restricted stock units that are outstanding during the period, except where such securities would be anti-dilutive. Basic and diluted loss per share was calculated as follows: Three Months Ended March 31, 2024 2023 (In thousands) Net loss $ ( 107,436 ) $ ( 103,126 ) Weighted average shares outstanding, basic and diluted 95,502 87,772 Net loss per share, basic and diluted $ ( 1.12 ) $ ( 1.17 ) The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2024 2023 (In thousands) Unvested restricted stock units 5,695 4,055 Stock options 5,910 5,905 11,605 9,960 |
Stockholders_ Equity
Stockholders’ Equity | 3 Months Ended |
Mar. 31, 2024 | |
Equity [Abstract] | |
Stockholders’ Equity | 12. Stockholders’ Equity At-the-Market Offering Programs 2022 Sale Agreement In March 2022, the Company entered into an Open Market Sale Agreement (the “2022 Sale Agreement”) with Jefferies LLC (“Jefferies”), under which Jefferies was able to offer and sell, from time to time in “at-the-market” offerings, shares of the Company’ s common stock having aggregate gross proceeds of up to $ 400.0 million. I n February 2024, the Company entered into an amendment to the 2022 Sale Agreement (the “2022 Sale Agreement, as amended”) to increase the size of the at-the-market offering program from $ 400.0 million to $ 750.0 million. The Company agreed to pay cash commissions of up to 3.0 % of the gross proceeds of sales of common stock under the 2022 Sale Agreement, as amended. Through March 31, 2024, the Company issued 9,728,101 shares of its common stock under the 2022 Sale Agreement, as amended. During the three months ended March 31, 2024 , the Company issued 2,209,938 shares of its common stock, in a series of sales, at an average price of $ 26.19 per share, in accordance with the 2022 Sale Agreement, as amended, for aggregate net proceeds of $ 56.0 million, after payment of cash commissions and approximately $ 0.2 million related to legal, accounting and other fees in connection with the sales. As of March 31, 2024 , $ 371.1 million in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Recent Accounting Pronouncements - Adopted | Recent Issued Accounting Pronouncements Not Yet Effective In November 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2023-07, “Segment Reporting - Improvements to Reportable Segment Disclosures.” The ASU requires disclosure of incremental segment information on an annual and interim basis and also requires companies with a single reportable segment to provide all disclosures required by this ASU and all existing segment disclosures in Accounting Standard Codification (“ASC”) 280, “Segment Reporting.” The ASU is effective for fiscal years beginning after December 15, 2023, and interim periods beginning after December 15, 2024. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures. In December 2023, the FASB issued ASU No. 2023-09, “Income Taxes (Topic 740): Improvements to Income Tax Disclosures.” This ASU updates income tax disclosure requirements primarily by requiring specific categories and greater disaggregation within the rate reconciliation and disaggregation of income taxes paid by jurisdiction. This ASU is effective for annual periods beginning after December 15, 2024 and is applicable to the Company’s fiscal year beginning January 1, 2025, with early application permitted. The Company is currently evaluating the impact of adopting this ASU on its consolidated financial statements and disclosures. |
Marketable Securities (Tables)
Marketable Securities (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available-for-sale Marketable Securities | The following table summarizes the Company’s available-for-sale marketable securities as of March 31, 2024 and December 31, 2023: March 31, 2024 Amortized Gross Unrealized Gross Unrealized Estimated Fair (In thousands) Marketable securities: U.S. Treasury and other government-backed securities $ 401,660 $ 40 $ ( 589 ) $ 401,111 Financial institution debt securities 214,001 56 ( 292 ) 213,765 Corporate debt securities 112,194 12 ( 129 ) 112,077 Other asset-backed securities 54,496 3 ( 47 ) 54,452 Total $ 782,351 $ 111 $ ( 1,057 ) $ 781,405 December 31, 2023 Amortized Gross Unrealized Gross Unrealized Estimated Fair (In thousands) Marketable securities: U.S. Treasury and other government-backed securities $ 382,260 $ 302 $ ( 254 ) $ 382,308 Financial institution debt securities 246,270 92 ( 243 ) 246,119 Corporate debt securities 97,490 53 ( 135 ) 97,408 Other asset-backed securities 59,453 75 ( 24 ) 59,504 Total $ 785,473 $ 522 $ ( 656 ) $ 785,339 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Recognized at Fair Value on Recurring Basis | he Company’s financial assets recognized at fair value on a recurring basis consisted of the following: March 31, 2024 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 143,500 $ 143,500 $ - $ - Marketable securities: U.S. Treasury and other government-backed securities 401,111 179,434 221,677 - Financial institution debt securities 213,765 - 213,765 - Corporate debt securities 112,077 - 112,077 - Other asset-backed securities 54,452 - 54,452 - Total marketable securities 781,405 179,434 601,971 - Investment in Kyverna Therapeutics, Inc. 29,158 29,158 - - Total assets $ 954,063 $ 352,092 $ 601,971 $ - December 31, 2023 Total Level 1 Level 2 Level 3 (In thousands) Assets Cash equivalents and restricted cash equivalents $ 136,254 $ 136,254 $ - $ - Marketable securities: U.S. Treasury and other government-backed securities 382,308 120,556 261,752 - Financial institution debt securities 246,119 - 246,119 - Corporate debt securities 97,408 - 97,408 - Other asset-backed securities 59,504 - 59,504 - Total marketable securities 785,339 120,556 664,783 - Total assets $ 921,593 $ 256,810 $ 664,783 $ - |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: March 31, December 31, 2024 2023 (In thousands) Accrued research and development $ 21,197 $ 27,411 Employee compensation and benefits 10,386 26,615 Accrued legal and professional expenses 3,808 2,063 Accrued construction costs 3,995 6,891 Accrued other 3,890 4,037 Total accrued expenses $ 43,276 $ 67,017 |
Collaborations and Other Arra_2
Collaborations and Other Arrangements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Changes in Accounts Receivable and Contract Liabilities | The following table presents changes in the Company’s accounts receivable and contract liabilities during the three months ended March 31, 2024 and 2023 (in thousands): Balance at Additions Deductions Balance at End Three months ended March 31, 2024 Accounts receivable $ 36,456 $ 5,872 $ ( 5,901 ) $ 36,427 Contract liabilities - deferred revenue $ 60,993 $ - $ ( 5,369 ) $ 55,624 Balance at Additions Deductions Balance at End Three months ended March 31, 2023 Accounts receivable $ 3,768 $ 4,488 $ ( 3,350 ) $ 4,906 Contract liabilities - deferred revenue $ 63,771 $ - $ ( 14,566 ) $ 49,205 |
Summary of Revenues Recognized Resulting from Changes in Contract Liability Balance | During the three months ended March 31, 2024 and 2023, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Three Months Ended March 31, Revenue recognized in the period from: 2024 2023 Amounts included in the contract liability at the beginning of the period $ 5,369 $ 11,753 |
Investments and Other Assets (T
Investments and Other Assets (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Investments, All Other Investments [Abstract] | |
Schedule of Investments and Other Assets | Investments and other assets consisted of the following: March 31, December 31, 2024 2023 (In thousands) Investment in Kyverna $ 29,158 $ 10,000 Other investments 24,213 14,760 Restricted cash, long-term 13,605 13,605 Prepaid expenses and other assets, long-term 3,981 4,518 Total investments and other assets $ 70,957 $ 42,883 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Schedule of Stock-Based Compensation Expense | Stock-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, 2024 2023 (In thousands) Research and development $ 20,161 $ 16,931 General and administrative 14,015 10,324 Total $ 34,176 $ 27,255 |
Summary of Restricted Stock Activity | The following table summarizes the Company’s RSU activity for the three months ended March 31, 2024: Number of Weighted Unvested restricted stock units as of December 31, 2023 4,041,753 $ 50.40 Granted 2,939,011 34.52 Vested ( 1,015,543 ) 51.62 Cancelled ( 270,591 ) 46.14 Unvested restricted stock units as of March 31, 2024 5,694,630 $ 42.18 |
Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted | Weighted average assumptions used to apply this pricing model were as follows: Three Months Ended March 31, 2024 2023 Risk-free interest rate 4.2 % 4.4 % Expected term 6.0 years 6.0 years Expected volatility of underlying stock 76.4 % 78.7 % Expected dividend yield 0.0 % 0.0 % |
Summary of Stock Option Activity | The following is a summary of stock option activity for the three months ended March 31, 2024: Number of Weighted Weighted Aggregate (In years) (In thousands) Outstanding at December 31, 2023 5,458,999 $ 50.38 Granted 680,804 32.66 Exercised ( 110,734 ) 17.68 Forfeited ( 119,562 ) 85.00 Outstanding at March 31, 2024 5,909,507 $ 48.25 6.66 $ 27,623 Exercisable at March 31, 2024 4,261,853 $ 44.98 5.86 $ 27,477 |
2016 Employee Stock Purchase Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted | The fair value of shares under the 2016 Plan was estimated at the beginning of the offering period using a Black-Scholes option-pricing model with the following assumptions: Three Months Ended March 31, 2024 2023 Risk-free interest rate 5.24 % 4.70 % Expected term 0.5 years 0.5 years Expected volatility of underlying stock 56.7 % 69.2 % Expected dividend yield 0.0 % 0.0 % |
Loss Per Share (Tables)
Loss Per Share (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Schedule of Basic and Diluted Net Loss Per Share | Basic and diluted loss per share was calculated as follows: Three Months Ended March 31, 2024 2023 (In thousands) Net loss $ ( 107,436 ) $ ( 103,126 ) Weighted average shares outstanding, basic and diluted 95,502 87,772 Net loss per share, basic and diluted $ ( 1.12 ) $ ( 1.17 ) |
Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share | The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2024 2023 (In thousands) Unvested restricted stock units 5,695 4,055 Stock options 5,910 5,905 11,605 9,960 |
Overview and Basis of Present_2
Overview and Basis of Presentation - Additional Information (Detail) $ in Millions | 3 Months Ended |
Mar. 31, 2024 USD ($) | |
IPO [Member] | |
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |
Proceeds from common stock offering | $ 2,598.3 |
Marketable Securities - Summary
Marketable Securities - Summary of Available -for-sale Marketable Securities (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Marketable Securities [Line Items] | ||
Amortized Cost | $ 782,351 | $ 785,473 |
Gross Unrealized Gains | 111 | 522 |
Gross Unrealized Losses | (1,057) | (656) |
Estimated Fair Value | 781,405 | 785,339 |
U.S. Treasury and Other Government - Backed Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 401,660 | 382,260 |
Gross Unrealized Gains | 40 | 302 |
Gross Unrealized Losses | (589) | (254) |
Estimated Fair Value | 401,111 | 382,308 |
Financial Institution Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 214,001 | 246,270 |
Gross Unrealized Gains | 56 | 92 |
Gross Unrealized Losses | (292) | (243) |
Estimated Fair Value | 213,765 | 246,119 |
Corporate Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 112,194 | 97,490 |
Gross Unrealized Gains | 12 | 53 |
Gross Unrealized Losses | (129) | (135) |
Estimated Fair Value | 112,077 | 97,408 |
Other Asset Backed Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 54,496 | 59,453 |
Gross Unrealized Gains | 3 | 75 |
Gross Unrealized Losses | (47) | (24) |
Estimated Fair Value | $ 54,452 | $ 59,504 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Marketable Securities [Line Items] | ||
Realized gains or losses on marketable securities | $ 0 | $ 0 |
Investments that matured beyond five years | $ 0 | $ 0 |
Minimum | ||
Marketable Securities [Line Items] | ||
Available-for-sales Securities, non-current, maturity period | 1 year | |
Maximum | ||
Marketable Securities [Line Items] | ||
Available-for-sales Securities, non-current, maturity period | 5 years |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets Recognized at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Marketable securities: | ||
Investment in Kyverna Therapeutics, Inc. | $ 29,158 | $ 10,000 |
Fair Value on Recurring Basis [Member] | ||
Assets | ||
Cash equivalents and restricted cash equivalents | 143,500 | 136,254 |
Marketable securities: | ||
Marketable securities | 781,405 | 785,339 |
Investment in Kyverna Therapeutics, Inc. | 29,158 | |
Total Assets | 954,063 | 921,593 |
Fair Value on Recurring Basis [Member] | U.S. Treasury and Other Government - Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 401,111 | 382,308 |
Fair Value on Recurring Basis [Member] | Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 213,765 | 246,119 |
Fair Value on Recurring Basis [Member] | Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 112,077 | 97,408 |
Fair Value on Recurring Basis [Member] | Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 54,452 | 59,504 |
Fair Value on Recurring Basis [Member] | Level 1 [Member] | ||
Assets | ||
Cash equivalents and restricted cash equivalents | 143,500 | 136,254 |
Marketable securities: | ||
Marketable securities | 179,434 | 120,556 |
Investment in Kyverna Therapeutics, Inc. | 29,158 | |
Total Assets | 352,092 | 256,810 |
Fair Value on Recurring Basis [Member] | Level 1 [Member] | U.S. Treasury and Other Government - Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 179,434 | 120,556 |
Fair Value on Recurring Basis [Member] | Level 2 [Member] | ||
Marketable securities: | ||
Marketable securities | 601,971 | 664,783 |
Total Assets | 601,971 | 664,783 |
Fair Value on Recurring Basis [Member] | Level 2 [Member] | U.S. Treasury and Other Government - Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 221,677 | 261,752 |
Fair Value on Recurring Basis [Member] | Level 2 [Member] | Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 213,765 | 246,119 |
Fair Value on Recurring Basis [Member] | Level 2 [Member] | Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 112,077 | 97,408 |
Fair Value on Recurring Basis [Member] | Level 2 [Member] | Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | $ 54,452 | $ 59,504 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Payables and Accruals [Abstract] | ||
Accrued research and development | $ 21,197 | $ 27,411 |
Employee compensation and benefits | 10,386 | 26,615 |
Accrued legal and professional expenses | 3,808 | 2,063 |
Accrued Construction Costs | 3,995 | 6,891 |
Accrued other | 3,890 | 4,037 |
Total accrued expenses | $ 43,276 | $ 67,017 |
Collaborations and Other Arra_3
Collaborations and Other Arrangements - Summary of Changes in Accounts Receivable and Contract Liabilities (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Accounts receivable: | ||
Accounts receivable, Balance at Beginning of Period | $ 36,456 | $ 3,768 |
Accounts receivable, Additions | 5,872 | 4,488 |
Accounts receivable, Deductions | (5,901) | (3,350) |
Accounts receivable, Balance at End of Period | 36,427 | 4,906 |
Contract liabilities: | ||
Deferred revenue, Balance at Beginning of Period | 60,993 | 63,771 |
Deferred revenue, Additions | 0 | 0 |
Deferred revenue, Deductions | (5,369) | (14,566) |
Deferred revenue, Balance at End of Period | $ 55,624 | $ 49,205 |
Collaborations and Other Arra_4
Collaborations and Other Arrangements - Summary of Revenues Recognized Resulting from Changes in Contract Liability Balance (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Revenue from Contract with Customer [Abstract] | ||
Amounts included in the contract liability at the beginning of the period | $ 5,369 | $ 11,753 |
Collaborations and Other Arra_5
Collaborations and Other Arrangements - Additional Information (Detail) - USD ($) | 1 Months Ended | 3 Months Ended | |||
Oct. 31, 2023 | Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | Dec. 31, 2022 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Costs to obtain or fulfill contract capitalized | $ 0 | ||||
Collaboration revenue | 28,935,000 | $ 12,606,000 | |||
Deferred revenue | 55,624,000 | 49,205,000 | $ 60,993,000 | $ 63,771,000 | |
Regeneron Pharmaceuticals Inc. [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Aggregate transaction price remaining to be recognized | 41,900,000 | ||||
Deferred revenue | 41,900,000 | 47,100,000 | |||
Development and commercial milestone payments | 320,000,000 | ||||
Regeneron Pharmaceuticals Inc. [Member] | Related Party [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Accounts receivable | 35,900,000 | 35,700,000 | |||
Regeneron Pharmaceuticals Inc. [Member] | Hemophilia Co Co Agreements | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | 3,300,000 | 2,900,000 | |||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Agreement [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | 7,400,000 | 6,800,000 | |||
Payments due | 4,700,000 | 4,200,000 | |||
Deferred revenue | $ 30,000,000 | ||||
Collaboration agreement extension period | two years | ||||
Collaboration term extension period | 2026-04 | ||||
Sparing Vision [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | 500,000 | 400,000 | |||
Deferred revenue | 13,700,000 | 13,900,000 | |||
Sparing Vision [Member] | Related Party [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Accounts receivable | 300,000 | $ 500,000 | |||
ReCode Therapeutics, Inc. | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Development and commercial milestone payments | 262,000,000 | ||||
AvenCell [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | 21,000,000 | 4,900,000 | |||
Accounts receivable | $ 200,000 | ||||
Revenue | 4,900,000 | ||||
Kyverna Therapeutics Inc [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Revenue | 400,000 | ||||
ONK Therapeutics [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Revenue | $ 100,000 |
Investments and Other Assets -
Investments and Other Assets - Schedule of Investments and Other Assets (Detail) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 | Mar. 31, 2023 |
Investments, All Other Investments [Abstract] | |||
Investment in Kyverna Therapeutics, Inc. | $ 29,158 | $ 10,000 | |
Other investments | 24,213 | 14,760 | |
Restricted cash, long-term | 13,605 | 13,605 | $ 11,958 |
Prepaid expenses and other assets, long-term | 3,981 | 4,518 | |
Total investments and other assets | $ 70,957 | $ 42,883 |
Investments and Other Assets _2
Investments and Other Assets - Additional Information (Detail) - USD ($) | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Schedule of Equity Method Investments [Line Items] | |||
Reclassification of other comprehensive loss - equity method investment | $ 2,132,000 | ||
Investments and other assets | 70,957,000 | $ 42,883,000 | |
Change in fair value of investments, net | (6,065,000) | $ 0 | |
Collaboration revenue | 28,935,000 | 12,606,000 | |
Accrued expenses | 43,276,000 | 67,017,000 | |
AvenCell Therapeutics, Inc. [Member] | |||
Schedule of Equity Method Investments [Line Items] | |||
Reclassification of other comprehensive loss - equity method investment | 2,100,000 | ||
Investments and other assets | 9,600,000 | ||
Kyverna Therapeutics Inc [Member] | |||
Schedule of Equity Method Investments [Line Items] | |||
Investment Owned, Balance, Principal Amount | 29,200,000 | ||
Change in fair value of investments, net | 19,200,000 | ||
Investment Owned, Fair Value | 10,000,000 | ||
AvenCell [Member] | |||
Schedule of Equity Method Investments [Line Items] | |||
Change in fair value of investments, net | 25,300,000 | ||
Investment Owned, Fair Value | $ 11,800,000 | ||
Collaboration revenue | 21,000,000 | 4,900,000 | |
Accounts receivable | 200,000 | ||
Accrued expenses | $ 1,000,000 | ||
AvenCell [Member] | AvenCell Therapeutics, Inc. [Member] | |||
Schedule of Equity Method Investments [Line Items] | |||
Equity method investment, ownership percentage | 33.33% | 33.33% | |
Collaboration revenue | $ 0 | ||
Sparing Vision [Member] | |||
Schedule of Equity Method Investments [Line Items] | |||
Investment Owned, Fair Value | 14,600,000 | ||
Collaboration revenue | $ 500,000 | $ 400,000 |
Rewrite Acquisition - Additiona
Rewrite Acquisition - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | |
Business Acquisition [Line Items] | |||
Research and development | $ 111,847 | $ 97,116 | |
Common stock, shares issued | 96,333,373 | 92,997,158 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | |
Feb. 28, 2022 ft² | Mar. 31, 2024 USD ($) ft² | Jun. 30, 2023 | Dec. 31, 2023 USD ($) | |
Lessee Lease Description [Line Items] | ||||
Operating lease right-of-use assets | $ | $ 110,424 | $ 115,375 | ||
Albany Lease [Member] | ||||
Lessee Lease Description [Line Items] | ||||
Operating lease, existence of option to extend | true | |||
840 Winter Street Lease [Member] | ||||
Lessee Lease Description [Line Items] | ||||
Area of space leased | 140,000 | |||
Operating lease, description | Separately, the Company entered into an agreement in February 2022, later amended in June 2023, to lease approximately 140,000 square feet of office, general laboratory and planned good manufacturing practice (“GMP”) manufacturing space at 840 Winter Street in Waltham | |||
Cambridge [Member] | ||||
Lessee Lease Description [Line Items] | ||||
Area of space leased | 230,000 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | $ 34,176 | $ 27,255 |
Research and Development [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | 20,161 | 16,931 |
General and Administrative [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | $ 14,015 | $ 10,324 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | |||||
Mar. 31, 2024 USD ($) $ / shares shares | Mar. 31, 2023 $ / shares shares | Mar. 31, 2022 Tranche $ / shares shares | May 31, 2016 | Mar. 31, 2024 USD ($) $ / shares shares | Mar. 31, 2023 USD ($) $ / shares shares | Dec. 31, 2023 shares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Common stock, shares issued | 96,333,373 | 96,333,373 | 92,997,158 | ||||
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | $ 34.52 | ||||||
Number of Shares, Granted | 2,939,011 | ||||||
Unvested restricted stock units | 5,694,630 | 5,694,630 | 4,041,753 | ||||
Risk-free interest rate | 4.20% | 4.40% | |||||
Expected dividend yield | 0% | 0% | |||||
Expected volatility | 76.40% | 78.70% | |||||
Expected term | 6 years | 6 years | |||||
Employee Stock Option | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Remaining vesting period | 1 year 4 months 28 days | ||||||
Weighted average grant date fair value per share | $ / shares | $ 22.61 | $ 28.82 | |||||
Total intrinsic value of stock options exercised | $ | $ 1.2 | $ 1.1 | |||||
Unrecognized compensation cost related to stock options | $ | $ 55.9 | $ 55.9 | |||||
2015 Plan [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Description of stock options granted under the Plan | Stock options generally vest as to one-third on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining two years, unless they contain specific vesting provisions. The maximum term of stock options granted under the 2015 Plan is ten years. | ||||||
Shares available for future issuance | 4,365,763 | 4,365,763 | |||||
Percentage of cumulative increase in number of shares for future issuance | 4% | ||||||
2016 Employee Stock Purchase Plan [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Description of stock options granted under the Plan | The 2016 Plan allows eligible employees to purchase shares of the Company’s common stock on the last day of each predetermined six-month offering period at 85% of the lower of the fair market value per share at the beginning or end of the applicable offering period. | ||||||
Percentage of fair market value of common stock | 85% | ||||||
Shares available for future issuance | 1,077,487 | 1,077,487 | |||||
Common stock, shares issued | 0 | 0 | 0 | 0 | |||
Risk-free interest rate | 5.24% | 4.70% | |||||
Expected dividend yield | 0% | 0% | |||||
Expected volatility | 56.70% | 69.20% | |||||
Expected term | 6 months | 6 months | |||||
Service Awards [Member] | RSUs [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Remaining vesting period | 3 years | 3 years | |||||
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | $ 32.66 | $ 40.75 | |||||
Number of Shares, Granted | 2,157,921 | 2,195,135 | |||||
Unvested restricted stock units | 4,733,350 | 4,733,350 | |||||
Market Awards [Member] | RSUs [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Unvested restricted stock units | 472,745 | 472,745 | |||||
Market Awards [Member] | RSUs [Member] | Senior Executives [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Remaining vesting period | 3 years | 3 years | 3 years | ||||
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | $ 51.12 | $ 68.55 | $ 126.49 | ||||
Number of Shares, Granted | 235,858 | 181,743 | 55,144 | ||||
Risk-free interest rate | 4.28% | 4.60% | 1.44% | ||||
Expected dividend yield | 0% | 0% | 0% | ||||
Expected volatility | 77.20% | 84.34% | 82.53% | ||||
Expected term | 3 years | 3 years | 3 years | ||||
Performance Based Awards with TSR Multiplier [Member] | RSUs [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Unvested restricted stock units | 444,117 | 444,117 | |||||
Performance Based Awards with TSR Multiplier [Member] | RSUs [Member] | Senior Executives [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | $ 36.16 | ||||||
Number of Shares, Granted | 444,117 | ||||||
Risk-free interest rate | 4.28% | ||||||
Expected dividend yield | 0% | ||||||
Expected volatility | 77.20% | ||||||
Expected term | 3 years | ||||||
Performance Based Awards [Member] | RSUs [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | $ 34.52 | $ 42.78 | |||||
Total fair value amount, Vested | $ | $ 30.9 | $ 12.1 | |||||
Unvested restricted stock units | 44,418 | 44,418 | |||||
Unrecognized equity-based compensation expense related to restricted stock | $ | $ 191.3 | $ 191.3 | |||||
Weighted average period of unrecognized compensation costs | 2 years | ||||||
Performance Based Awards [Member] | RSUs [Member] | Non-executive Employees [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of Shares, Granted | 66,296 | ||||||
Number of tranches | Tranche | 2 | ||||||
Performance Based Awards [Member] | First Anniversary of Original Vesting Date [Member] | RSUs [Member] | Non-executive Employees [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of Shares, Granted | 21,878 | ||||||
Maximum | 2016 Employee Stock Purchase Plan [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Common stock, shares issued | 500,000 | 500,000 | |||||
Maximum | Performance Based Awards with TSR Multiplier [Member] | RSUs [Member] | Senior Executives [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Share Based Compensation Arrangements by Share Based Payment Award, Performance Shares | 200% | ||||||
TSR modifier | 125% | ||||||
Minimum | Performance Based Awards with TSR Multiplier [Member] | RSUs [Member] | Senior Executives [Member] | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Share Based Compensation Arrangements by Share Based Payment Award, Performance Shares | 0% | ||||||
TSR modifier | 75% |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Restricted Stock Activity (Detail) | 3 Months Ended |
Mar. 31, 2024 $ / shares shares | |
Share-Based Payment Arrangement [Abstract] | |
Number of Shares, Unvested, Beginning balance | shares | 4,041,753 |
Number of Shares, Granted | shares | 2,939,011 |
Number of Shares, Vested | shares | (1,015,543) |
Number of Shares, Cancelled | shares | (270,591) |
Number of Shares, Unvested, Ending balance | shares | 5,694,630 |
Weighted Average Grant Date Fair Value per Share, Unvested, Beginning balance | $ / shares | $ 50.4 |
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | 34.52 |
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares | 51.62 |
Weighted Average Grant Date Fair Value per Share, Cancelled | $ / shares | 46.14 |
Weighted Average Grant Date Fair Value per Share, Unvested, Ending balance | $ / shares | $ 42.18 |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted (Detail) | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||
Risk-free interest rate | 4.20% | 4.40% |
Expected term | 6 years | 6 years |
Expected volatility of underlying stock | 76.40% | 78.70% |
Expected dividend yield | 0% | 0% |
2016 Employee Stock Purchase Plan [Member] | ||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||
Risk-free interest rate | 5.24% | 4.70% |
Expected term | 6 months | 6 months |
Expected volatility of underlying stock | 56.70% | 69.20% |
Expected dividend yield | 0% | 0% |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of Stock Option Activity (Detail) $ / shares in Units, $ in Thousands | 3 Months Ended |
Mar. 31, 2024 USD ($) $ / shares shares | |
Share-Based Payment Arrangement [Abstract] | |
Number of Options, Outstanding, Beginning Balance | shares | 5,458,999 |
Number of options, Granted | shares | 680,804 |
Number of options, Exercised | shares | (110,734) |
Number of options, Forfeited | shares | (119,562) |
Number of Options, Outstanding, Ending Balance | shares | 5,909,507 |
Number of Options, Exercisable | shares | 4,261,853 |
Weighted Average Exercise Price per Share, Outstanding, Beginning Balance | $ / shares | $ 50.38 |
Weighted Average Exercise Price per Share, Granted | $ / shares | 32.66 |
Weighted Average Exercise Price per Share, Exercised | $ / shares | 17.68 |
Weighted Average Exercise Price per Share, Forfeited | $ / shares | 85 |
Weighted Average Exercise Price per Share, Outstanding, Ending Balance | $ / shares | 48.25 |
Weighted Average Exercise Price per Share, Exercisable | $ / shares | $ 44.98 |
Weighted Average Remaining Contractual Term, Outstanding | 6 years 7 months 28 days |
Weighted Average Remaining Contractual Term, Exercisable | 5 years 10 months 9 days |
Aggregate Intrinsic Value, Outstanding | $ | $ 27,623 |
Aggregate Intrinsic Value, Exercisable | $ | $ 27,477 |
Loss Per Share - Schedule of Ba
Loss Per Share - Schedule of Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Earnings Per Share [Abstract] | ||
Net Income (Loss) | $ (107,436) | $ (103,126) |
Weighted average shares outstanding, basic | 95,502 | 87,772 |
Weighted average shares outstanding, diluted | 95,502 | 87,772 |
Net loss per share, basic | $ (1.12) | $ (1.17) |
Net loss per share, diluted | $ (1.12) | $ (1.17) |
Loss Per Share - Potential Dilu
Loss Per Share - Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share (Detail) - shares shares in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 11,605 | 9,960 |
Unvested Restricted Stock Units [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 5,695 | 4,055 |
Employee Stock Option | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 5,910 | 5,905 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | ||
Mar. 31, 2024 | Feb. 29, 2024 | Mar. 31, 2022 | Mar. 31, 2024 | |
2022 Sales Agreement [Member] | Maximum | ||||
Class Of Stock [Line Items] | ||||
Proceeds from common stock offering | $ 400 | |||
Amended 2022 Sales Agreement [Member] | ||||
Class Of Stock [Line Items] | ||||
Proceeds from common stock offering | $ 56 | |||
Percentage of gross proceeds from common stock as sales agent cash commission | 3% | |||
Stock Issued During Period, Shares, New Issues | 9,728,101 | 2,209,938 | ||
Common stock price per share | $ 26.19 | $ 26.19 | ||
Legal accounting and other fees | $ 0.2 | |||
Proceeds from common stock offering | $ 371.1 | $ 371.1 | ||
Amended 2022 Sales Agreement [Member] | Maximum | ||||
Class Of Stock [Line Items] | ||||
Proceeds from common stock offering | $ 750 | |||
Amended 2022 Sales Agreement [Member] | Minimum | ||||
Class Of Stock [Line Items] | ||||
Proceeds from common stock offering | $ 400 |