Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2024 | May 03, 2024 | |
Document Information [Line Items] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2024 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q1 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity Registrant Name | 4D Molecular Therapeutics, Inc. | |
Entity Central Index Key | 0001650648 | |
Entity Current Reporting Status | Yes | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | false | |
Entity Shell Company | false | |
Entity Interactive Data Current | Yes | |
Entity File Number | 001-39782 | |
Entity Tax Identification Number | 47-3506994 | |
Entity Address, Address Line One | 5858 Horton Street #455 | |
Entity Address, City or Town | Emeryville | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 94608 | |
City Area Code | (510) | |
Local Phone Number | 505-2680 | |
Entity Incorporation, State or Country Code | DE | |
Title of 12(b) Security | Common Stock, par value $0.0001 per share | |
Trading Symbol | FDMT | |
Security Exchange Name | NASDAQ | |
Pre-funded Warrants [Member] | ||
Document Information [Line Items] | ||
Entity Common Stock, Shares Outstanding | 3,583,476 | |
Common Stock [Member] | ||
Document Information [Line Items] | ||
Entity Common Stock, Shares Outstanding | 51,703,412 |
Condensed Balance Sheets (Unaud
Condensed Balance Sheets (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 275,594 | $ 249,108 |
Marketable securities | 250,317 | 39,124 |
Prepaid expenses and other current assets | 9,678 | 8,356 |
Total current assets | 535,589 | 296,588 |
Marketable securities, long-term | 62,942 | 10,954 |
Property and equipment, net | 19,464 | 20,126 |
Operating lease right-of-use assets, net | 11,118 | 11,539 |
Other assets | 771 | 684 |
Total assets | 629,884 | 339,891 |
Current liabilities | ||
Accounts payable | 4,217 | 3,515 |
Accrued and other current liabilities | 9,013 | 12,014 |
Deferred revenue | 0 | 273 |
Operating lease liabilities, current portion | 3,173 | 3,149 |
Total current liabilities | 16,403 | 18,951 |
Deferred revenue, net of current portion | 1,322 | 972 |
Derivative liability | 369 | 369 |
Operating lease liabilities, long-term portion | 10,998 | 11,522 |
Other liabilities | 231 | 248 |
Total liabilities | 29,323 | 32,062 |
Commitments and contingencies (Note 9) | ||
Stockholders’ equity | ||
Preferred stock, $0.0001 par value, 10,000,000 shares authorized at March 31, 2024 and December 31, 2023; no shares issued and outstanding at March 31, 2024 and December 31, 2023 | 0 | 0 |
Common stock, $0.0001 par value, 300,000,000 shares authorized at March 31, 2024 and December 31, 2023; 51,157,388 and 43,075,218 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | 5 | 4 |
Additional paid-in-capital | 1,048,393 | 723,136 |
Accumulated other comprehensive gain (loss) | (109) | 16 |
Accumulated deficit | (447,728) | (415,327) |
Total stockholders’ equity | 600,561 | 307,829 |
Total liabilities and stockholders’ equity | $ 629,884 | $ 339,891 |
Condensed Balance Sheets (Una_2
Condensed Balance Sheets (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares, issued | 51,157,388 | 43,075,218 |
Common stock, shares, outstanding | 51,157,388 | 43,075,218 |
Condensed Statements of Operati
Condensed Statements of Operations (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Revenue: | ||
Collaboration and license revenue | $ 28 | $ 298 |
Operating expenses: | ||
Research and development | 27,870 | 22,412 |
General and administrative | 10,294 | 7,992 |
Total operating expenses | 38,164 | 30,404 |
Loss from operations | (38,136) | (30,106) |
Other income (expense): | ||
Interest income | 5,745 | 1,443 |
Other expense, net | (10) | (19) |
Total other income, net | 5,735 | 1,424 |
Net loss | $ (32,401) | $ (28,682) |
Net loss per share, basic | $ (0.66) | $ (0.88) |
Net loss per share, diluted | $ (0.66) | $ (0.88) |
Weighted-average shares outstanding used in computing net loss per share, basic | 49,271,984 | 32,723,530 |
Weighted-average shares outstanding used in computing net loss per share, diluted | 49,271,984 | 32,723,530 |
Condensed Statements of Compreh
Condensed Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (32,401) | $ (28,682) |
Other comprehensive loss: | ||
Net unrealized gain (loss) on marketable securities | (125) | 926 |
Total comprehensive loss | $ (32,526) | $ (27,756) |
Condensed Statements of Stockho
Condensed Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | ATM Offering Program | February / March 2024 Public Offering Sale | Common Stock | Common Stock ATM Offering Program | Common Stock February / March 2024 Public Offering Sale | Additional Paid-in Capital | Additional Paid-in Capital ATM Offering Program | Additional Paid-in Capital February / March 2024 Public Offering Sale | Accumulated Other Comprehensive Loss | Accumulated Deficit |
Stockholders' Equity, beginning balances at Dec. 31, 2022 | $ 231,337 | $ 3 | $ 547,020 | $ (1,196) | $ (314,490) | ||||||
Stockholders' Equity, beginning balances (in shares) at Dec. 31, 2022 | 32,626,627 | ||||||||||
Issuance of common stock upon exercise of stock options | 1,135 | 1,135 | |||||||||
Issuance of common stock upon exercise of stock options (in shares) | 122,207 | ||||||||||
Issuance of common stock, net of issuance costs | $ 9,647 | $ 9,647 | |||||||||
Issuance of common stock, net of issuance costs (in shares) | 487,934 | ||||||||||
Stock-based compensation expense | 4,221 | 4,221 | |||||||||
Vesting of common stock warrants issued for services | 22 | 22 | |||||||||
Net unrealized gain (loss) on marketable securities | 926 | 926 | |||||||||
Net loss | (28,682) | (28,682) | |||||||||
Stockholders' Equity, ending balances at Mar. 31, 2023 | 218,606 | $ 3 | 562,045 | (270) | (343,172) | ||||||
Stockholders' Equity, ending balances (in shares) at Mar. 31, 2023 | 33,236,768 | ||||||||||
Stockholders' Equity, beginning balances at Dec. 31, 2023 | $ 307,829 | $ 4 | 723,136 | 16 | (415,327) | ||||||
Stockholders' Equity, beginning balances (in shares) at Dec. 31, 2023 | 43,075,218 | ||||||||||
Issuance of common stock upon exercise of stock options (in shares) | 225,860 | ||||||||||
Issuance of common stock upon exercise of stock options and vesting of RSUs | $ 3,113 | 3,113 | |||||||||
Issuance of common stock upon exercise of stock options and vesting of RSUs (in shares) | 236,856 | ||||||||||
Issuance of common stock, net of issuance costs | $ 316,149 | $ 1 | $ 316,148 | ||||||||
Issuance of common stock, net of issuance costs (in shares) | 7,845,314 | ||||||||||
Stock-based compensation expense | 5,974 | 5,974 | |||||||||
Vesting of common stock warrants issued for services | 22 | 22 | |||||||||
Net unrealized gain (loss) on marketable securities | (125) | (125) | |||||||||
Net loss | (32,401) | (32,401) | |||||||||
Stockholders' Equity, ending balances at Mar. 31, 2024 | $ 600,561 | $ 5 | $ 1,048,393 | $ (109) | $ (447,728) | ||||||
Stockholders' Equity, ending balances (in shares) at Mar. 31, 2024 | 51,157,388 |
Condensed Statements of Cash Fl
Condensed Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Cash flows from operating activities | ||
Net loss | $ (32,401) | $ (28,682) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Stock-based compensation expense | 5,974 | 4,221 |
Vesting of common stock warrant in return for services | 22 | 22 |
Change in fair value of derivative liability | 0 | 7 |
Depreciation and amortization | 1,173 | 1,029 |
Amortization of right-of-use assets | 421 | 383 |
Net amortization (accretion) of premium (discount) on marketable securities | (757) | (395) |
Changes in operating assets and liabilities | ||
Prepaid expenses and other current assets | (1,322) | 90 |
Other assets | (88) | (110) |
Accounts payable | 703 | (333) |
Accrued and other liabilities | (2,389) | (3,397) |
Deferred revenue | 77 | (298) |
Operating lease liabilities | (501) | (443) |
Net cash used in operating activities | (29,088) | (27,906) |
Cash flows from investing activities | ||
Purchase of marketable securities | (277,324) | (14,729) |
Maturities of marketable securities | 14,776 | 58,871 |
Acquisition of property and equipment | (710) | (1,012) |
Net cash provided by (used in) investing activities | (263,258) | 43,130 |
Cash flows from financing activities | ||
Payment of offering costs | 0 | (69) |
Issuance of common stock upon the exercise of stock options | 3,113 | 1,416 |
Net cash provided by financing activities | 318,832 | 10,994 |
Net increase in cash and cash equivalents | 26,486 | 26,218 |
Cash and cash equivalents, beginning of period | 249,108 | 52,351 |
Cash and cash equivalents, end of period | 275,594 | 78,569 |
Supplemental disclosures of noncash investing and financing information | ||
Unpaid offering costs | 430 | 0 |
Purchases of property and equipment in accounts payable and accrued and other liabilities | 0 | 357 |
Public offering | ||
Cash flows from financing activities | ||
Issuance of common stock for the offering program, net of issuance costs | 315,719 | 0 |
ATM Offering Program | ||
Cash flows from financing activities | ||
Issuance of common stock for the offering program, net of issuance costs | $ 0 | $ 9,647 |
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) | $ (32,401) | $ (28,682) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Mar. 31, 2024 shares | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | During the fiscal quarter ended March 31, 2024, the following officers and directors, as defined in Rule 16a-1(f) adopted and terminated “Rule 10b5-1 trading arrangements,” as defined in Regulation S-K Item 408, as follows: On January 4, 2024 , Fariborz Kamal , our President and Chief Operating Officer , adopted a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 15,000 shares of our common stock. The trading arrangement is intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement is until December 31, 2024 , or earlier if all transactions under the trading arrangement are completed. On March 19, 2024 , Robert Kim , our Chief Medical Officer , terminated a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 25,595 shares of our common stock. The trading arrangement was intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement was until March 31, 2024 , or earlier if all transactions under the trading arrangement were completed. On March 19, 2024 , Robert Kim , our Chief Medical Officer also adopted a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 16,000 shares of our common stock. The trading arrangement is intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement is until June 30, 2026 , or earlier if all transactions under the trading arrangement are completed. On March 21, 2024 , David Kirn , our Chief Executive Officer , terminated a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 200,000 shares of our common stock. The trading arrangement was intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement was until November 29, 2024 , or earlier if all transactions under the trading arrangement were completed. On March 21, 2024 , David Kirn , our Chief Executive Officer also adopted a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 305,083 shares of our common stock. The trading arrangement is intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement is until May 15, 2025 , or earlier if all transactions under the trading arrangement are completed. On March 22, 2024 , Jacob Chacko , a member of our board of directors , adopted a Rule 10b5-1 trading arrangement providing for the sale from time to time of up to 32,500 shares of our common stock. The trading arrangement is intended to satisfy the affirmative defense in Rule 10b5-1(c). The duration of the trading arrangement is until June 30, 2025 , or earlier if all transactions under the trading arrangement are completed. None of our other directors or officers (as defined in Section 16 of the Securities Exchange Act of 1934, as amended) adopted or terminated any contract, instruction or written plan for the purchase or sale of our securities that was intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) or any “non-Rule 10b5-1 trading arrangement,” as defined in Item 408(a) of Regulation S-K. |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
Rule 10b5-1 Trading Plan | Fariborz Kamal | |
Trading Arrangements, by Individual | |
Name | Fariborz Kamal |
Title | President and Chief Operating Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | January 4, 2024 |
Arrangement Duration | 11 months 28 days |
Aggregate Available | 15,000 |
Expiration Date | Dec. 31, 2024 |
Rule 10b5-1 Trading Plan | Robert Kim | |
Trading Arrangements, by Individual | |
Name | Robert Kim |
Title | Chief Medical Officer |
Rule 10b5-1 Arrangement Terminated | true |
Termination Date | March 19, 2024 |
Arrangement Duration | 13 days |
Aggregate Available | 25,595 |
Expiration Date | Mar. 31, 2024 |
Rule 10b5-1 Trading Plan | David Kirn | |
Trading Arrangements, by Individual | |
Name | David Kirn |
Title | Chief Executive Officer |
Rule 10b5-1 Arrangement Terminated | true |
Termination Date | March 21, 2024 |
Arrangement Duration | 8 months 9 days |
Aggregate Available | 200,000 |
Expiration Date | Nov. 29, 2024 |
Rule 10b5-1 Trading Plan | Jacob Chacko | |
Trading Arrangements, by Individual | |
Name | Jacob Chacko |
Title | member of our board of directors |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | March 22, 2024 |
Arrangement Duration | 15 months 9 days |
Aggregate Available | 32,500 |
Expiration Date | Jun. 30, 2025 |
Rule 10b5-1 Trading Plan One | Robert Kim | |
Trading Arrangements, by Individual | |
Name | Robert Kim |
Title | Chief Medical Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | March 19, 2024 |
Arrangement Duration | 27 months 12 days |
Aggregate Available | 16,000 |
Expiration Date | Jun. 30, 2026 |
Rule 10b5-1 Trading Plan One | David Kirn | |
Trading Arrangements, by Individual | |
Name | David Kirn |
Title | Chief Executive Officer |
Rule 10b5-1 Arrangement Adopted | true |
Adoption Date | March 21, 2024 |
Arrangement Duration | 13 months 25 days |
Aggregate Available | 305,083 |
Expiration Date | May 15, 2025 |
The Company
The Company | 3 Months Ended |
Mar. 31, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
The Company | 1. The Company 4D Molecular Therapeutics, Inc. (the “Company”) was formed as a limited liability company in September 2013 under the name 4D Molecular Therapeutics, LLC. The Company changed its name and converted into a corporation which was incorporated in the state of Delaware in March 2015. The Company is a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines. Initial Public Offering In December 2020, the Company sold and issued 9,660,000 shares of common stock at a price to the public of $ 23.00 per share, which included shares sold upon the underwriters’ exercise of their overallotment option to purchase 1,260,000 additional shares. The Company received an aggregate of $ 204.7 million in net proceeds, after deducting underwriting discounts and commissions and offering costs. Upon the closing of the Company's initial public offering in December 2020 (the "IPO"), all outstanding shares of redeemable convertible preferred stock automatically converted into 11,575,984 shares of common stock. Subsequent to the closing of the IPO, there were no shares of redeemable convertible preferred stock outstanding. 2021 Follow On Public Offering In November 2021, the Company completed an underwritten public offering ("2021 Offering") in which 4,750,000 shares of the Company's common stock were sold at an offering price of $ 25.00 per share pursuant to an effective Registration Statement on Form S-1. The net proceeds from the 2021 Offering were $ 111.1 million, after deducting underwriting discounts and commissions and offering expenses. 2023 Follow On Public Offering In May 2023, the Company completed its third underwritten public offering (the “2023 Offering”) in which 8,625,000 shares of the Company’s common stock were sold at an offering price of $ 16.00 per share pursuant to an effective Registration Statement on Form S-3. The net proceeds from the 2023 Offering were $ 129.2 million, after deducting underwriting discounts and commissions and offering expenses. 2024 Follow On Public Offering In February 2024, the Company completed an underwritten public offering (the “2024 Offering”) in which 6,586,015 shares of the Company’s common stock were sold at an offering price of $ 29.50 per share, as well as pre-funded warrants to purchase 3,583,476 shares of the Company’s common stock at an offering price of $ 29.4999 per underlying share pursuant to an effective Registration Statement on Form S-3. The net proceeds from the 2024 Offering were $ 281.2 million, after deducting underwriting discounts and commissions and other offering expenses. The Company also granted the underwriters the option to purchase up to 1,525,423 additional shares of common stock in connection with the offering. In March 2024, the underwriters exercised their option to purchase 1,259,299 additional shares of common stock resulting in net proceeds of $ 34.9 million, after deducting commissions. Liquidity The Company has incurred significant losses and negative cash flows from operations and had an accumulated deficit of $ 447.7 million as of March 31, 2024. The Company believes that its cash and cash equivalents and marketable securities as of March 31, 2024 are sufficient for the Company to fund planned operations for at least one year from the issuance date of these unaudited condensed financial statements. The Company has historically financed its operations primarily through the sale of equity securities, and to a lesser extent, from cash received pursuant to its collaboration and license agreements. To date, none of the Company’s product candidates have been approved for sale, and therefore, the Company has not generated any revenue from product sales. Management expects operating losses and negative cash flows from operations to continue for the foreseeable future. The Company plans to raise additional funding as required based on the status of its clinical trials and projected cash flows. There can be no assurance that, in the event the Company requires additional financing, such financing will be available on terms acceptable to the Company, if at all. Failure to generate sufficient cash flows from operations, raise additional capital and reduce discretionary spending should additional capital not become available could have a material adverse effect on the Company’s ability to achieve its business objectives. |
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 Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with United States Generally Accepted Accounting Principles (“U.S. GAAP”) and the rules and regulations of the United States Securities and Exchange Commission (“SEC”) for interim reporting. Certain information and note disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been condensed or omitted. Accordingly, the unaudited condensed financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC. The accompanying financial information for the three months ended March 31, 2024 and 2023 is unaudited. The unaudited condensed financial statements have been prepared on the same basis as the annual audited financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s financial position as of March 31, 2024 and December 31, 2023 and its results of operations and cash flows for the three months ended March 31, 2024 and 2023. The results for interim periods are not necessarily indicative of the results expected for the full fiscal year or any other periods. Use of Estimates and Judgments The preparation of condensed financial statements in conformity with U.S. GAAP requires management to make estimates and judgments that affect the reported amounts of assets, liabilities, revenue and expenses; and disclosure of contingent assets and liabilities as of the date of the financial statements. Such estimates include the determination of useful lives for property and equipment, the contract term, transaction price and costs of collaboration agreements, as well as estimates of the fair value of common stock (prior to the IPO), stock options and the derivative instrument and income tax uncertainties. Actual results could differ from those estimates. Due to the war in Ukraine, conflicts in the Middle East, rising interest rates and inflation, and the lingering impacts of the coronavirus (“COVID-19”) pandemic, there has been uncertainty and disruption in the global economy and financial markets. The Company is not aware of any specific event or circumstance that would require an update to its estimates or judgments or a revision of the carrying value of its assets or liabilities as of March 31, 2024. While there was not a material impact to the Company’s unaudited condensed financial statements as of March 31, 2024 , these estimates may change, as new events occur and additional information is obtained, as well as other factors that could result in material impacts to the unaudited condensed financial statements in future reporting periods. Segment Information The Company operates and manages its business as one reportable and operating segment. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on a company-wide basis for purposes of allocating resources and assessing financial performance. Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, marketable securities and accounts receivable. The Company’s cash is held at two financial institutions in the United States of America. The Company’s cash equivalents are invested in money market funds. The Company also invests in U.S. Treasuries, U.S. government sponsored agencies, commercial paper, corporate bonds and certificates of deposit. The Company has not experienced any losses on its deposits of cash and cash equivalents. Such deposits may, at times, exceed federally insured limits. The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Three Months Ended March 31, 2024 2023 Customer A 0 % 99 % Customer B 100 % * Total 100 % 99 % * Less than 10 % The Company did no t have accounts receivable from its partners in collaboration and license agreements as of March 31, 2024 and December 31, 2023. The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Three Months Ended March 31, 2024 2023 Netherlands $ — $ 295 United States 28 3 Total revenue $ 28 $ 298 Other Risks and Uncertainties The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, suppliers for key raw materials, contract manufacturing organizations (“CMOs”) and contract research organizations (“CROs”), compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies, clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance and reporting. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third parties (including for clinical trials and some aspects of research and preclinical testing). Recently Issued Accounting Pronouncements Not Yet Adopted On December 14, 2023, the Financial Accounting Standards Board issued Accounting Standards Update (“ASU”) No. 2023-09, Improvements to Income Tax Disclosures. The final guidance adds clarifications related to the presentation of rate reconciliation for public business entities and definitions of specific categories in rate reconciliation. These amendments are effective for public business entities for annual periods beginning after December 15, 2024. For other entities, amendments are effective for annual periods beginning after December 15, 2025. The Company is not early adopting ASU 2023-09. The Company expects to make additional disclosures for income taxes upon adoption of this ASU. |
Fair Value Measurements
Fair Value Measurements | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements The following tables represent the Company’s fair value hierarchy for financial assets and financial liabilities measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023 (in thousands): Basis for Fair Value Measurements Fair Value as of Level 1 Level 2 Level 3 March 31, 2024 Assets Money market funds $ 245,111 $ — $ — $ 245,111 Commercial paper — 64,624 — 64,624 Corporate bonds — 169,106 — 169,106 U.S. Treasuries — 26,262 — 26,262 U.S. government sponsored agencies — 1,398 — 1,398 Certificates of deposit — 51,869 — 51,869 Total $ 245,111 $ 313,259 $ — $ 558,370 Liabilities Derivative liability $ — $ — $ 369 $ 369 Total $ — $ — $ 369 $ 369 Basis for Fair Value Measurements Fair Value as of Level 1 Level 2 Level 3 December 31, 2023 Assets Money market funds $ 217,644 $ — $ — $ 217,644 Commercial paper — 18,243 — 18,243 Corporate bonds — 28,939 — 28,939 U.S. government sponsored agencies — 2,896 — 2,896 Total $ 217,644 $ 50,078 $ — $ 267,722 Liabilities Derivative liability $ — $ — $ 369 $ 369 Total $ — $ — $ 369 $ 369 Level 3 Inputs The fair value of the derivative liability is based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. The fair value of the derivative liability was determined using a present value analysis with multiple scenarios. In determining the fair value of the derivative liability, the inputs impacting fair value include the change of control payment to Cystic Fibrosis Foundation (CFF), the probability of a change of control event, the product status at time of a change of control event and the discount rate. See Note 14 for further discussion on the embedded derivative. There were no transfers between Level 1, 2 and 3 during the periods presented. The following tables set forth a summary of the changes in the fair value of the Company’s Level 3 financial instrument during the three-month periods ended March 31, 2024 and 2023 (in thousands): Derivative Balance as of December 31, 2023 $ 369 Change in fair value included in other income (expense), net — Balance as of March 31, 2024 $ 369 Derivative Balance as of December 31, 2022 $ 212 Change in fair value included in other income (expense), net 7 Balance as of March 31, 2023 $ 219 |
Marketable Securities
Marketable Securities | 3 Months Ended |
Mar. 31, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | 4. Marketable Securities Marketable securities, which are classified as available-for-sale, consisted of the following as of March 31, 2024 and December 31, 2023 (in thousands): Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of March 31, 2024 Short-term marketable securities: Commercial paper $ 61,428 $ 5 $ ( 46 ) $ 61,387 Corporate bonds 109,432 16 ( 47 ) 109,401 U.S. Treasuries 26,262 1 ( 1 ) 26,262 U.S. government sponsored agencies 1,400 — ( 2 ) 1,398 Certificates of deposit 51,875 3 ( 9 ) 51,869 Total short-term marketable securities $ 250,397 $ 25 $ ( 105 ) $ 250,317 Long-term marketable securities: Commercial paper $ 3,234 $ 3 $ — $ 3,237 Corporate bonds 59,737 14 ( 46 ) 59,705 Total long-term marketable securities $ 62,971 $ 17 $ ( 46 ) $ 62,942 Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of December 31, 2023 Short-term marketable securities: Commercial paper $ 17,603 $ 1 $ ( 26 ) $ 17,578 Corporate bonds 18,641 12 ( 3 ) 18,650 U.S. government sponsored agencies 2,900 — ( 4 ) 2,896 Total short-term marketable securities $ 39,144 $ 13 $ ( 33 ) $ 39,124 Long-term marketable securities: Corporate bonds $ 661 $ 4 $ — $ 665 Commercial paper 10,257 32 — 10,289 Total long-term marketable securities $ 10,918 $ 36 $ — $ 10,954 All marketable securities held as of March 31, 2024 had contractual maturities of less than two years . There have been no material realized gains or losses on marketable securities for the periods presented. Aggregate fair values of marketable securities with unrealized losses and gains were as follows as of March 31, 2024 and December 31, 2023 (in thousands): March 31, December 31, 2024 2023 Aggregate fair value of marketable securities in a continuous loss position for less than twelve months $ 209,564 $ 20,494 Aggregate fair value of marketable securities in a continuous loss position for more than twelve months — 4,986 Aggregate fair value of marketable securities in unrealized gain position 103,695 24,598 Total marketable securities $ 313,259 $ 50,078 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 no t record an allowance for credit losses or other impairment charges related to its marketable securities for any period presented. The Company has determined that (i) it does not have the intent to sell any of these investments, and (ii) it is not more likely than not that it will be required to sell any of these investments before recovery of the entire amortized cost basis. The Company further considered the maximum unrealized loss amounts both at the individual instrument level, $ 9 thousand, as well as in aggregate, $ 151 thousand, as of March 31, 2024, as immaterial. These unrealized losses were not attributed to credit risk and were associated with changes in market conditions. The Company periodically reviews its marketable securities for indications of credit losses. The Company anticipates that it will recover the entire amortized cost basis of such securities, and therefore, no credit loss existed as of March 31, 2024. |
Property and Equipment, Net
Property and Equipment, Net | 3 Months Ended |
Mar. 31, 2024 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net, consisted of the following (in thousands): March 31, December 31, Machinery and equipment $ 13,605 $ 12,996 Leasehold improvements 17,325 16,902 Furniture and fixtures 566 566 Office equipment 239 239 Computer equipment and software 835 805 Construction in progress — 550 Total property and equipment 32,570 32,058 Less: Accumulated depreciation and amortization ( 13,106 ) ( 11,932 ) Property and equipment, net $ 19,464 $ 20,126 All property and equipment are maintained in the United States. Depreciation expense was $ 1.2 million and $ 1.0 million for the three months ended March 31 , 2024 and 2023, respectively. |
Accrued and Other Current Liabi
Accrued and Other Current Liabilities | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
Accrued and Other Current Liabilities | 6. Accrued and Other Current Liabilities Accrued and other current liabilities consisted of the following (in thousands): March 31, December 31, Payroll and related expenses $ 4,516 $ 8,149 Accrued clinical and preclinical study costs 2,908 2,180 Consulting and professional 1,447 1,581 Other accrued expenses 142 104 Total accrued and other current liabilities $ 9,013 $ 12,014 |
Research and Collaboration Arra
Research and Collaboration Arrangements | 3 Months Ended |
Mar. 31, 2024 | |
Research And Collaboration Arrangements [Abstract] | |
Research and Collaboration Arrangements | 7. Research and Collaboration Arrangements Collaboration and license revenue for each period was as follows (in thousands): Three Months Ended March 31, 2024 2023 uniQure $ — $ 295 CFF 28 3 Total revenue $ 28 $ 298 Deferred revenue is summarized as follows (in thousands): March 31, December 31, CFF $ 1,322 $ 1,245 Total deferred revenue $ 1,322 $ 1,245 The total amount of revenue in the three months ended March 31, 2024 which was included in deferred revenue at January 1, 2024 was zero . The total amount of revenue in the three months ended March 31, 2023 which was included in deferred revenue at January 1, 2023 was $ 0.3 million. uniQure In January 2014, the Company and uniQure biopharma B.V. (“uniQure”) entered into a Collaboration and License Agreement (the “uniQure Agreement”) to collaborate on the discovery and non-clinical research activities related to the Company’s Therapeutic Vector Evolution platform in order to generate and validate vectors for gene delivery to treat diseases within the central nervous system and liver (together, the “uniQure Field”). The uniQure Agreement provided uniQure with a research license as well as an exclusive development and commercialization license for each project variant selected for further development. The initial research term is three years with an option for uniQure to extend the research term one time for an additional year. Once the Company’s research plan has concluded, uniQure is solely responsible for the continued development, manufacturing and commercialization of the project variants as potential product candidates. In October 2016, uniQure exercised its option to extend the research term for an additional year to January 2018. The Company was also required to work exclusively with uniQure in the uniQure Field (the “uniQure Exclusivity Clause”). Pursuant to the uniQure Agreement, the Company received upfront payments of $ 0.2 million, and was entitled to receive (i) contingent payments for the achievement of research and development milestones of up to $ 5.0 million for each licensed product selected under the arrangement, and (ii) royalties in the single digit range on future sales of the potential product candidates and sublicense consideration in the low teens to low thirties range on any future sublicensing arrangements. The Company also received capped research and development service fees based on contractual full-time employee rates per year. In connection with the performance obligations under the uniQure Agreement, the founders of 4D Molecular Therapeutics, LLC received equity options to purchase an aggregate of 609,744 of uniQure ordinary shares that vested over the initial three-year term of the agreement. The upfront payment of $ 0.2 million was recorded as deferred revenue and was recognized on a ratable basis over the estimated performance period of four years . Payments and reimbursements for research costs were recognized on an as-incurred basis. The options to purchase uniQure shares were deemed to be a noncash component of the arrangement consideration, as the vesting of options is linked to the uniQure Agreement and there is a requirement for the holders of the options to provide services under the agreement. The fair value of the uniQure options, which was estimated to be $ 10.6 million, was recognized ratably as revenue over the estimated performance period of four years and the associated compensation expense related to the stock options was recorded as research and development expense. In August 2019, the Company and uniQure entered into an Amended and Restated Collaboration and License Agreement (the “Amended uniQure Agreement”), which amended and restated the uniQure Agreement, and a separate Collaboration and License Agreement (the “Second uniQure Agreement”). Under these agreements, the Company agreed to transfer incremental rights and services to uniQure in exchange for uniQure eliminating the uniQure Exclusivity Clause and transferring other rights back to the Company. Under the Amended uniQure Agreement, uniQure continues to have an exclusive license to select AAV capsid variants (the “Selected Variants”) in the uniQure Field. uniQure continues to be solely responsible, at its cost, to develop and commercialize the compounds and products containing the Selected Variants. The amended uniQure Agreement eliminated the uniQure Exclusivity Clause in the uniQure Agreement. Furthermore, the contingent payments that the Company was entitled to from uniQure for the achievement of research and development milestones of up to $ 5.0 million for each licensed product selected under the uniQure Agreement were eliminated and sublicense consideration on any future sublicensing arrangements was reduced from the low teens to low thirties percentages to mid-single digit to mid-twenties percentages. Under the Second uniQure Agreement, the parties agreed to research and develop new AAV capsid variants (the “New Variants”) that are not Selected Variants that affect certain targets selected by uniQure (the “uniQure Targets”) in the uniQure Field. The Company is solely responsible, at its cost, for the research of the New Variants. The Company granted uniQure an exclusive license to a certain number of the New Variants (the “uniQure New Variants”) that affect the uniQure Targets. uniQure is solely responsible, at its cost, to develop and commercialize the compounds and products containing the uniQure New Variants that affect the uniQure Targets (the “Licensed Products”). The Company retains all rights to New Variants in the uniQure Field that affect targets other than the uniQure Targets. Under both the Amended uniQure Agreement and the Second uniQure Agreement, uniQure will be required to pay the Company royalties on worldwide annual net sales of Licensed Products at a mid-single digit percentage rate, subject to certain specified reductions. uniQure will also be required to pay the Company sublicensing consideration for sublicensing the Company’s intellectual property rights licensed under the Amended uniQure Agreement or the Second uniQure Agreement to third parties at a rate between the mid-single digit to mid-twenties. The Company has reciprocal obligations, at the same percentage rates as uniQure, to pay uniQure royalties and sublicensing consideration for sublicensing certain intellectual property rights licensed under the Amended uniQure Agreement or the Second uniQure Agreement to third parties. The Company concluded that the Amended uniQure Agreement and the Second uniQure Agreement should be accounted for as one combined contract that should be accounted for as a separate contract from the uniQure Agreement given that the incremental licensed intellectual property rights and research and development services are distinct from the rights and services previously transferred to uniQure under the uniQure Agreement and the transaction price increased by an amount that equals the standalone selling price of the incremental rights and services to be transferred to uniQure under the Amended uniQure Agreement and Second uniQure Agreement. Neither party was required to pay monetary consideration in connection with the execution of the Amended uniQure Agreement or the Second uniQure Agreement or for subsequent performance by the parties under those agreements, notwithstanding the potential future royalty and sublicense consideration described above. The fair value of the non-monetary consideration given by uniQure to the Company, for the intellectual property right is $ 5.1 million. This intellectual property right is considered to be an in-process research and development asset with no alternative future use and, accordingly, was written off as acquired in-process research and development expense in the year ended December 31, 2019. The incremental transaction price described in the paragraph above was recorded as deferred revenue given that the Company identified one single combined performance obligation under ASC 606, which includes the licenses to the New Variants, research services and participation in the joint steering committee (“JSC”). Revenue is being recognized using the input method based on actual costs incurred as a percentage of total budgeted costs as the Company completes its performance obligation. The Company completed its performance obligation during the third quarter of 2023 and the deferred revenue was recognized as revenue in the same period. The Company determined the transaction price using the risk adjusted net present value analysis (“rNPV”) methodology to value the elimination of the uniQure exclusivity clause and other material rights received by the Company, including the potential royalties the Company would receive from uniQure. The rNPVs incorporate estimates and assumptions including the number of products the Company and uniQure would develop, the risk-adjusted probability of successfully developing a biopharmaceutical product, the probability that uniQure will develop a product, the research and development costs, the potential worldwide sales and associated commercialization costs, corporate tax rate, and discount rate. During the three months ended March 31, 2024 and 2023, the Company recognized zero and $ 0.3 million, respectively, under both of the Amended uniQure Agreement and the Second uniQure Agreement. There were no amounts due from uniQure under the uniQure Agreement, Amended uniQure Agreement or Second uniQure Agreement as of March 31, 2024 and December 31, 2023. As of March 31, 2024 and December 31, 2023, deferred revenue relating to uniQure was zero and the aggregate amount of the transaction price allocated to the remaining performance obligation was zero . Cystic Fibrosis Foundation (CFF) In September 2016, the Company entered into an award agreement for the Optimized Adeno-Associated Virus for Lung Epithelia Gene Delivery Development Program with CFF, a non-profit organization dedicated to finding a cure for cystic fibrosis, an inherited disorder that causes disease in the pulmonary airways leading to morbidity and mortality. Under this agreement, CFF contributes funding to help advance the Company’s CF research program. The September 2016 grant award agreement was incorporated into a new grant award agreement with the CFF in September 2017 with the same objectives, which was subsequently amended in August 2018 and February 2021. In August 2023, the Company executed a third amendment to the agreement (the “August 2023 Amendment”), which modified the research plan, increased the aggregate milestone payments from $ 3.5 million to $ 6.3 million and extended the estimated project completion date. The aforementioned September 2017 agreement and three amendments are collectively referred to as the “CFF Agreement”. The August 2023 Amendment represents a contract modification to an existing contract under Topic 606, given the amendment did not include any additional goods or services, and the remaining research activities are not distinct from those previously provided. The August 2023 Amendment did not impact the transaction price, given the increased award amount relates to variable consideration for future milestones that are fully constrained. Accordingly, the contract modification did not result in a revenue adjustment. As of March 31, 2024 and December 31, 2023, the Company had achieved milestones totaling $ 1.8 million and $ 1.7 million, respectively, under the CFF Agreement. The remaining award amount will be paid by CFF based on achievement of certain development milestones by the Company. The Company expects to make payments to CFF equal to six times the actual award received by the Company in three installments within the first four years of the first commercial sale of a product developed under this agreement. The Company also has agreed to make future sales-based milestone payments to CFF of up to three times the actual award received upon achieving specified commercialization milestones with respect to the first of any product developed utilizing any compound covered under the CFF Agreement. The CFF Agreement also requires the Company to pay to CFF royalties of a mid-single digit percentage, up to six times the actual award received, on any amounts received by the Company from the sale, license or transfer to a third-party of rights in the technology developed as a result of this collaboration. Any such royalty payments shall be credited against the payments owed by the Company upon first commercial sale. In the event of a change of control of the Company, CFF will receive certain payments, depending on the timing of the change of control and the size of the transaction. To date, the Company has not developed a commercial product in connection with the CFF Agreement, and it has not licensed, sold or otherwise transferred to another party the product developed under the CFF Agreement or the underlying technology. If at any time prior to the first commercial sale of a product developed as a result of the CFF Agreement, the Company ceases to use commercially reasonable efforts to develop or commercialize any product under the CFF Agreement for a continuous period of 180 consecutive days and fails to present a reasonable plan to resume commercially reasonable efforts, the Company will grant to CFF an irrevocable, exclusive worldwide interruption license under all of the Company’s interest in the research plan technology to exploit such product. Any third-party license granted by the Company shall be subject to such interruption license. The Company identified one performance obligation within the CFF grant agreement for research activities. The Company’s contract with CFF does not include a significant financing component. The Company concluded that the transaction price should not include the variable consideration related to future research milestones as they were considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal of cumulative revenue in the future. The Company re-evaluates the transaction price and estimated period of performance at each reporting period. Revenue recognized during the three months ended March 31, 2024 and 2023 was immaterial. As of March 31, 2024 and December 31, 2023, deferred revenue relating to the CFF Agreement was $ 1.3 million and $ 1.2 million, respectively. There were no accounts receivable from CFF under the CFF Agreement as of March 31, 2024 and December 31, 2023. As of March 31, 2024 and December 31, 2023, the aggregate amount of the transaction price allocated to the remaining performance obligation was $ 1.3 million and $ 1.2 million, respectively. Based on current timelines, the deferred revenue is expected to be recognized as revenue over the next one to three years as the Company performs research services through the completion of IND-enabling studies. The obligation to make payments to CFF upon a change of control meets the definition of an embedded derivative that is required to be bifurcated and separately accounted for as a derivative liability. See Note 14 for further discussion of the embedded derivative. Arbor Biotechnologies, Inc. On December 20, 2023 (the “Effective Date”), the Company entered into a co-development and co-commercialization agreement (the “Arbor Agreement”) with Arbor Biotechnologies, Inc. (“Arbor”), pursuant to which the Company and Arbor agreed to co-develop and co-commercialize on a fifty-fifty cost-and-profit-sharing basis up to six genetic medicine products to treat central nervous system (“CNS”) indications, based on combining the Company’s CNS-targeting vectors and Arbors’ Cas enzymes and Guide RNAs. The first product candidate under the Arbor Agreement will address a molecular target implicated in amyotrophic lateral sclerosis (ALS). The Company will account for its costs related to the co-development and co-commercialization of product candidates under the Arbor Agreement as research and development expenses in the periods in which they are incurred. As of March 31, 2024, no Shared Product has been designated by the JSC (each as defined in the Arbor Agreement) under the Arbor Agreement, and no research and development expenses were incurred related to the Arbor Agreement during the three months ended March 31, 2024. |
License Arrangements
License Arrangements | 3 Months Ended |
Mar. 31, 2024 | |
License Arrangements [Abstract] | |
License Arrangements | 8. License Arrangements Astellas Gene Therapies, Inc. On July 5, 2023, the Company entered into a licensing agreement (the “License Agreement”) with Astellas Gene Therapies, Inc. (“AGT”), pursuant to which the Company granted to AGT a license to utilize its intravitreal R100 vector (“4D Vector”) to develop and commercialize licensed compounds and licensed products for one genetic target implicated in rare monogenic ophthalmic disease(s), with options to add up to two additional targets implicated in rare monogenic ophthalmic diseases after paying additional option exercise fees. Under the terms of the License Agreement, the Company has provided its 4D vector technology to AGT to deliver AGT's genetic payloads for the treatment of rare monogenic diseases. AGT will conduct all subsequent research, development, manufacturing, and commercialization activities. As partial consideration for the rights and licenses granted to AGT by the Company under this Agreement, AGT paid the Company an upfront amount of $ 20 million, which was received in July 2023. The Company may receive potential future option fees and milestones of up to $ 942.5 million including potential near-term development milestones of $ 15 million for the initial target. In addition, the Company is entitled to receive mid-single digit to double-digit, sub-teen royalties on net sales of all licensed products. Under the License Agreement, the Company’s performance obligation is to grant and make available to AGT the Licensed IP and Licensed Know-How (each as defined in the License Agreement) with respect to the 4D Vector. In connection with the grant of the license, in July 2023, the Company delivered to AGT the Transferred Material (as defined in the License Agreement). As of March 31, 2024, there was no deferred revenue and no accounts receivable relating to the License Agreement. Aevitas Therapeutics, Inc. and The Trustees of the University of Pennsylvania On April 21, 2023, the Company entered into an agreement (the “Agreement”) with Aevitas Therapeutics, Inc. (“Aevitas”), pursuant to which the Company acquired all of Aevitas’ worldwide rights to short-form human complement factor H ("sCFH"), which the Company plans to use for its 4D-175 product candidate research program. The asset purchase was accounted for as an asset acquisition. As consideration for the Agreement, the Company shall pay Aevitas up to approximately $ 144 million in cash upon certain late-stage development, regulatory and sales milestones being achieved plus royalties in the low single digits range on sales of 4D-175. In addition, as part of the Agreement, the Company was assigned a License Agreement for sCFH with the Trustees of the University of Pennsylvania, under which the Company shall pay the University of Pennsylvania up to approximately $ 42 million in cash upon certain late-stage development, regulatory and sales milestones being achieved plus royalties in the low single digits range on sales of 4D-175. No upfront consideration was paid under the Agreement. As of March 31, 2024, the Company has no t recorded a liability related to contingent consideration for future milestone and royalty payments to either Aevitas or the University of Pennsylvania, as the achievement of such milestones has not occurred and was not deemed probable and product sales have not commenced. Regents of the University of California The Company has exclusive, worldwide license agreements (the “UC Agreements”) with the Regents of the University of California (the “UC Regents”) relating to the use of certain patents and intellectual property surrounding its core technologies, including Therapeutic Vector Evolution. Pursuant to each of the UC Agreements executed prior to January 2019, the Company was obligated to pay a (i) non-refundable license fee of $ 5,000 upon execution, (ii) a non-refundable license fee of $ 5,000 each year thereafter, until sales of a licensed product are made and royalties are paid to the UC Regents, (iii) reimbursement of domestic and foreign patent filing, prosecution and maintenance fees, and (iv) either $ 50,000 or issuance of a 3 % equity interest in the Company upon the closing of the first qualified financing at the option of the UC Regents. The Company’s first qualified financing occurred in 2015 and at the election of the UC Regents, the Company issued the UC Regents in January 2016 an amount of common stock equal to 6 % of the equity interests in the Company pursuant to the applicable clause in each of the UC Agreements. Pursuant to an agreement with the UC Regents executed in January 2019 the Company paid a non-refundable license fee of $ 50,000 to the UC Regents upon execution of the agreement. The Company is obligated to pay a non-refundable license fee of $ 5,000 on the one-year anniversary of the contract effective date and each year thereafter, until sales of a licensed product are made and royalties are paid to the UC Regents. In addition, the Company is obligated to make certain contingent payments including (i) development milestones up to $ 3.1 million, (ii) low single digit royalties on the net sales of its developed products that consists of a minimum annual royalty of up to $ 0.1 million per year for the term of the agreement beginning in the first calendar year after the year in which net sales first occurred, and (iii) sublicense consideration in the mid-teens to the mid-twenties-range on any future sublicensing arrangements the Company may enter into with third-party licensees. Regents of the University of California and The Trustees of the University of Pennsylvania In July 2021, the Company entered into an exclusive license agreement with the UC Regents and the Trustees of the University Pennsylvania to license intellectual property related to certain vectors. In July 2021, the Company paid a non-refundable license fee of $ 100,000 to the UC Regents upon execution of the agreement. The Company is obligated to pay a non-refundable license maintenance fee of $ 10,000 on the one-year anniversary of the contract effective date and each year thereafter, except years for which the Company has paid royalties on the net sales of a licensed product. In addition, the Company is obligated to make certain contingent payments including (i) development milestones up to $ 3.9 million, (ii) low single digit percentage rate royalties on the net sales of its licensed products that consists of a minimum annual royalty of up to $ 0.1 million per year for the term of the agreement beginning in the first calendar year after the year in which net sales first occurred and (iii) sublicense consideration in the mid-single digits to the low twenties percentage rate range on any future sublicensing arrangements the Company may enter into with third-party licensees. On February 22, 2023, the Company provided 90 days written notice to the UC Regents and the Trustees of the University Pennsylvania to terminate said agreement in its entirety at-will. Accordingly, the effective termination date of this agreement is May 23, 2023. The Company did no t incur any expense under the provisions of the outstanding license agreements during each of the three months ended March 31, 2024 and 2023. |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies Operating Lease Commitments 5980 Horton Street Building Lease In May 2015, the Company executed a lease agreement for office and laboratory space in Emeryville, California. In January 2016, the Company executed the first amendment to the lease agreement for additional rentable office and laboratory space which extended the lease term to March 31, 2023 and updated the option to renew the lease at market rent from the prior additional term of three years to five years . In October 2018, the Company executed a second amendment to extend the lease to September 2026 . Additionally, the second amendment provided a tenant improvement allowance of $ 0.2 million, which was paid to the Company in November 2018. The 5980 Horton Street Building Lease is considered an operating lease under ASC 842 as it does not meet the criteria of a finance lease. As of March 31, 2024, the operating lease right-of-use asset and operating lease liability were $ 1.1 million and $ 1.3 million, respectively. As of December 31, 2023, the operating lease right-of-use asset and operating lease liability were $ 1.2 million and $ 1.4 million, respectively. The discount rate used to determine the lease liability was 7.5 %. 5858 Horton Street Building Lease and Expansion In October 2018, the Company executed a second lease agreement for additional office and laboratory space in Emeryville, California. The new lease has an initial term of 87 months beginning on the rent commencement date with the option to renew the lease for one additional term of five years at market rent. The Company did not have to pay rent until October 2019. This lease agreement also provided for a tenant improvement allowance of $ 0.4 million, which was paid to the Company in December 2019. The Company amortizes the tenant improvement allowance on a straight-line basis over the remaining term of the lease as a reduction of rent expense. In May 2019, the Company amended the second lease agreement executed in October 2018 to add additional office, manufacturing and laboratory space (the “Expansion”). The amendment extended the term of the lease to December 31, 2029. The Company did not have to pay rent until December 2019. The lease agreement also included a tenant improvement allowance, which was increased to $ 2.3 million in February 2021 pursuant to a second amendment to the second lease agreement. The Company fully received the $ 2.3 million tenant improvement allowance in 2023. The tenant improvement allowance funds improvements to the additional office, manufacturing and laboratory space, which the Company has determined to be lessee owned. The allowance is treated as a reduction of lease payments used to measure the lease liability. The 5858 Horton Street Building Lease and Expansion are considered operating leases under ASC 842 as they do not meet the criteria of a finance lease. As of March 31, 2024, the operating lease right-of-use asset and operating lease liability was $ 10.0 million and $ 12.9 million, respectively. As of December 31, 2023, the operating lease right-of-use asset and operating lease liability was $ 10.3 million and $ 13.2 million, respectively. The discount rate used to determine the lease liability was 7.8 %. Neither of the leases include a general option for the Company to terminate the leases. Emeryville, California Warehouse Lease In January 2024, the Company entered into a new lease for warehouse space in Emeryville, California (the “Warehouse Lease Agreement”), with a projected lease commencement date of August 31, 2024 . The lease consists of approximately 7,800 square feet of warehouse space and has a lease term of five years and four months . Future minimum lease payments under the Warehouse Lease Agreement from its projected commencement date through lease expiration in the fourth quarter of 2029 are approximately $ 1.5 million. As of March 31, 2024, the Company has no t recorded a right-of-use asset or lease liability on its condensed balance sheets as the Company has not yet obtained control of the warehouse space. The following table summarizes the components of lease expense for the three months ended March 31, 2024 and 2023, which are included in operating expenses in the Company’s condensed statements of operations (in thousands): Three Months Ended March 31, 2024 2023 Operating lease cost $ 698 $ 697 Variable lease cost 354 486 Total $ 1,052 $ 1,183 Variable lease payments include amounts relating to common area maintenance and are recognized in the condensed statements of operations as incurred. The following table summarizes supplemental information related to operating leases: March 31, December 31, Weighted-average remaining lease term (in years): 5.5 5.7 Weighted-average discount rate: 7.8 % 7.8 % The following table summarizes the maturities of lease liabilities as of March 31, 2024 (in thousands): 2024 (remaining nine months) $ 2,371 2025 3,244 2026 3,135 2027 2,810 2028 2,894 Thereafter 2,981 Total future minimum lease payments 17,435 Less: Amount representing interest ( 3,264 ) Present value of future minimum lease payments 14,171 Less: Current portion of operating lease liabilities 3,173 Long-term portion of operating lease liabilities $ 10,998 Indemnification Agreements In the ordinary course of business, the Company enters into agreements that may include indemnification provisions, such as with vendors and other parties. Pursuant to such agreements, the Company may indemnify, hold harmless and defend an indemnified party for losses suffered or incurred by the indemnified party. Some of the provisions will limit losses to those arising from third-party actions. In some cases, the indemnification will continue after the termination of the agreement. The maximum potential amount of future payments the Company could be required to make under these provisions is not determinable. The Company has never incurred material costs to defend lawsuits or settle claims related to these indemnification provisions. The Company has also entered into indemnification agreements with its directors and officers that require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers to the fullest extent permitted by Delaware corporate law. The Company currently maintains directors’ and officers’ liability insurance that would generally enable it to recover a portion of any future amounts paid. The Company believes the estimated fair value of its indemnification agreements in excess of applicable insurance coverage is not material. Legal Proceedings From time to time, the Company may become involved in legal proceedings arising from the ordinary course of its business. If applicable, the Company records a legal liability when it believes that it is both probable that a liability may be imputed, and the amount of the liability can be reasonably estimated. Significant judgment by the Company is required to determine both probability and the estimated amount. There are no material legal proceedings outstanding at March 31, 2024. |
Common Stock
Common Stock | 3 Months Ended |
Mar. 31, 2024 | |
Equity [Abstract] | |
Common Stock | 10. Common Stock As of March 31, 2024 and December 31, 2023, the Company’s certificate of incorporation authorized the Company to issue 300,000,000 shares of common stock, at the par value of $ 0.0001 per share. The holder of each share of common stock is entitled to one vote per share . Common stockholders are entitled to dividends, if and when declared by the board of directors. To date, no dividends on common stock have been declared by the board of directors. As of March 31, 2024 and December 31, 2023, the Company has reserved common stock, on an as-converted basis, for future issuance as follows: March 31, December 31, Issuance of common stock under the 2020 Equity Incentive Award Plan 2,426,209 524,078 Issuance of common stock under the 2020 Employee Stock Purchase Plan 363,034 313,034 Exercise of options issued and outstanding and future vesting of restricted stock units 8,438,028 8,423,481 Exercise of common stock warrants 3,637,145 53,669 Total common stock reserved for future issuance 14,864,416 9,314,262 Funding Agreement with CFF — In April 2020, CFF made a $ 10.0 million investment in the Company's Series C redeemable convertible preferred stock financing. In return for the investment, CFF received shares of Series C redeemable convertible preferred stock, and the Company and CFF entered into a Funding Agreement (the “Funding Agreement”). Pursuant to the terms of the Funding Agreement, except in the event of a technical failure, the $ 10.0 million received from CFF will be used to advance the development program for 4D-710, the Company’s lead product in cystic fibrosis, or any other therapeutic approved by the Program Advisory Group (“PAG”) to alleviate pulmonary complications of cystic fibrosis (the “Funding Agreement Product”). CFF committed to provide an additional $ 4.0 million of funding upon acceptance of an Investigational New Drug (“IND”) application or its equivalent to allow for human testing of the Funding Agreement Product (“Acceptance”). In October 2021, the IND was cleared by the U.S. Food and Drug Administration and CFF made the additional investment of $ 4.0 million in cash for the issuance of 125,715 shares of the Company's common stock to CFF. The Company was committed to providing an amount equal to the funding provided by CFF to be used solely to advance the Funding Agreement Product. As of March 31, 2024, the funding commitment has been fulfilled. Under the terms of the Funding Agreement, neither the $ 10.0 million investment in the Series C redeemable convertible preferred stock, which converted to common stock as of December 31, 2020, nor the $ 4.0 million of funding upon Acceptance are restricted as to withdrawal or usage. |
Stock-based Compensation
Stock-based Compensation | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-based Compensation | 11. Stock-based Compensation 2020 Equity Incentive Award Plan In December 2020, the Company adopted the 2020 Incentive Award Plan (“2020 Plan”), which became effective on December 10, 2020. The 2020 Plan initially reserved 2,606,546 shares of common stock for the issuance of stock options, stock appreciation rights, restricted stock awards, restricted stock units, performance bonus awards, performance stock units, dividend equivalents or other stock or cash based award granted to employees, directors and consultants of the Company. The number of shares reserved for future issuance under the 2020 Plan will increase annually on the first day of each fiscal year beginning in 2021 and ending in 2030 by the lesser of (i) 5 % of the shares of common stock outstanding (on an as converted basis) on the last day of the immediately preceding fiscal year and (ii) such number of shares of common stock as determined by the Company's board of directors, provided, however, no more than 18,000,000 shares of the Company's common stock may be issued upon the exercise of incentive stock options. On February 29, 2024, an additional 2,153,533 shares of common stock became available for issuance under the 2020 Plan, as a result of the operation of the automatic annual increase provision. All options are exercisable over a period not to exceed the contractual term of ten years from the date the stock options were issued. As of March 31 , 2024 there were 2,090,604 shares available for grant under the 2020 Plan. Following the effectiveness of the 2020 Plan, the Company will not make any further grants under the 2015 Equity Incentive Plan (the “2015 Plan”). However, the 2015 Plan continues to govern the terms of options that remain outstanding under the 2015 Plan. As of March 31 , 2024, there were 335,605 shares available for grant under the 2015 Plan. 2015 Equity Incentive Plan The 2015 Plan provided for grants of stock options, stock appreciation rights, restricted stock and restricted stock unit awards to employees, directors and consultants of the Company. As of March 31 , 2024, options to purchase 1,563,448 shares of common stock were outstanding under the 2015 Plan. All options are exercisable over a period not to exceed the contractual term of ten years from the date the stock options were issued and are granted at prices not less than the estimated fair market value of the Company’s common stock on the grant date as determined by the board of directors. No additional grants will be made under the 2015 Plan, and all outstanding grants under the 2015 Plan that are repurchased, forfeited, expire or are cancelled are returned to the 2015 Plan and are not available for grant under the 2020 Plan. Employee Stock Purchase Plan In December 2020, the Company adopted the 2020 Employee Stock Purchase Plan (the “2020 ESPP”). Under the 2020 ESPP, 252,337 shares of the Company's common stock were initially reserved for employee purchases of the Company's common stock under terms and provisions established by the Company's board of directors and approved by the Company's stockholders. The number of shares reserved for future issuance under the 2020 ESPP will increase annually on the first day of each fiscal year beginning in 2021 and ending in 2030 by the lesser of (i) 1 % of the shares of common stock outstanding (on an as converted basis) on the last day of the immediately preceding fiscal year and (ii) such number of shares of common stock as determined by the Company's board of directors, provided, however, no more than 15,000,000 shares the Company's common stock may be issued under the 2020 ESPP. On February 29, 2024, an additional 50,000 shares of common stock became available for future issuance under the 2020 ESPP, as a result of the operation of this automatic annual increase provision. Under the 2020 ESPP the Company's employees may purchase common stock through payroll deductions at a price equal to 85 % of the lower of the fair market value of the stock at the beginning of the offering period or at the end of each applicable purchase period. The 2020 ESPP provides for a series of overlapping 24-month offering periods comprising four six-month purchase periods. The initial offering period under the 2020 ESPP is longer than 24 months, commencing February 15, 2021 and ending on May 14, 2023. Contributions under the 2020 ESPP are limited to a maximum of 15 % of an employee's eligible compensation. Restricted Stock Units The Company has granted restricted stock unit (RSU) awards under the 2020 Equity Incentive Award Plan that vest over a period of four years . The following table summarizes the RSU activity for the three months ended March 31, 2024: Number of Unvested balance at December 31, 2023 181,181 Issued 55,400 Vested ( 10,996 ) Canceled/Forfeited ( 15,685 ) Unvested balance at March 31, 2024 209,900 Stock Options The following table summarizes the stock options activity for the three months ended March 31, 2024: Number of Balances at December 31, 2023 8,240,154 Options granted 286,000 Options exercised ( 225,860 ) Options expired ( 7,081 ) Options forfeited ( 65,085 ) Balances at March 31, 2024 8,228,128 Options exercisable at March 31, 2024 3,321,825 Options vested and expected to vest at March 31, 2024 8,228,128 The Company estimates the fair value of stock options using the Black-Scholes option pricing model. The fair value of employee and nonemployee options is recognized on a straight-line basis over the requisite service period of the awards. The fair value of the Company’s stock options was estimated using the following assumptions for the three months ended March 31, 2024 and 2023: Three Months Ended March 31, 2024 2023 Expected term 6.0 - 6.1 years 6.0 - 6.1 years Expected volatility 81.4 % - 85.3 % 80.9 % - 84.5 % Risk-free interest rate 3.9 % - 4.3 % 3.7 % - 4.0 % Expected dividend yield 0 % 0 % Expected Term . The expected term for employee options is calculated using the simplified method as the Company does not have sufficient historical information to provide a basis for this estimate. The simplified method is based on the average of the vesting tranches and the contractual life of each grant. The expected term for nonemployee options is the contractual term of the options. Expected Volatility . The expected volatility is based on a mix of the Company's historical volatility and the historical volatility of comparable companies with similar attributes to the Company, including industry, stage of life cycle, size and financial leverage. For each grant, the Company measured historical volatility over a period equivalent to the expected term. Risk-free Interest Rate. The risk-free interest rate is based on the yield available on U.S. Treasury zero-coupon issues whose term is similar in duration to the expected term of the respective stock option. Expected Dividend Yield . The Company has not paid and does not anticipate paying any dividends on its common stock in the future. Accordingly, the Company has estimated the dividend yield to be zero . Stock-Based Compensation Expense The following table is a summary of stock-based compensation expense incurred for employees and nonemployees by function (in thousands): Three Months Ended 2024 2023 Research and development $ 3,034 $ 2,470 General and administrative 2,940 1,751 Total stock-based compensation expense $ 5,974 $ 4,221 |
Common Stock Warrants
Common Stock Warrants | 3 Months Ended |
Mar. 31, 2024 | |
Equity [Abstract] | |
Common Stock Warrants | 12. Common Stock Warrants In May 2018, the Company issued a warrant for 23,669 shares of the Company’s common stock to a service provider with an exercise price of $ 3.19 per share. The fair value of the warrant was determined at the issuance date using the Black-Scholes option pricing model, expires in 2025 and upon issuance was fully vested. In December 2020, the Company issued a warrant for 30,000 shares of the Company’s common stock to a service provider with an exercise price of $ 18.00 per share. This warrant vests over a period of four years and expires in 2027 . The fair value of the warrant was determined at the issuance date using the Black-Scholes option pricing model. In February 2024, the Company completed an underwritten public follow-on offering which included the sale of pre-funded warrants (the “Pre-funded Warrants”) to purchase 3,583,476 shares of the Company’s common stock at an offering price of $ 29.4999 . The exercise price of each Pre-funded Warrant is $ 0.0001 per share, and each Pre-funded Warrant is exercisable from the date of issuance. The Pre-funded Warrants are classified as a component of stockholders’ equity within additional paid-in-capital. The Company valued the Pre-funded Warrants at issuance and recorded net proceeds of $ 99.4 million after deducting underwriters fees during the three months ended March 31, 2024 related to the sale of the Pre-funded Warrants. There was no material expense related to the above warrant shares during each of the three months ended March 31, 2024 and 2023. |
Net Loss Per Share, Basic and D
Net Loss Per Share, Basic and Diluted | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share, Basic and Diluted | 13. Net Loss Per Share, Basic and Diluted The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Three Months Ended March 31, 2024 2023 Numerator Net loss $ ( 32,401 ) $ ( 28,682 ) Denominator Weighted-average shares outstanding used in computing net loss per share, basic and diluted 49,271,984 32,723,530 Net loss per share, basic and diluted $ ( 0.66 ) $ ( 0.88 ) In February 2024, the Company issued and sold Pre-funded Warrants to purchase 3,583,476 shares of common stock at a nominal exercise price of $ 0.0001 (see Note 12). The shares of common stock into which the Pre-funded Warrants may be exercised are considered outstanding for the purposes of computing earnings per share, because the shares may be issued for little or no consideration, they are fully vested and the Pre-funded Warrants are immediately exercisable upon their issuance date. The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: March 31, 2024 2023 Options issued and outstanding 8,228,128 6,589,104 Restricted stock units subject to future vesting 209,900 — 2020 ESPP 250,727 360,769 Common stock warrants 53,669 53,669 Total 8,742,424 7,003,542 |
Derivative Liability
Derivative Liability | 3 Months Ended |
Mar. 31, 2024 | |
Derivative Instruments and Hedging Activities Disclosure [Abstract] | |
Derivative Liability | 14. Derivative Liability The Company identified an embedded derivative resulting from the change of control provision in the CFF Agreement. Embedded derivatives that are required to be bifurcated from the underlying host instrument are accounted for and valued as separate financial instruments. At the inception of the derivative in 2017, the Company recognized this derivative as a liability and revenue was reduced by the initial fair value of the derivative liability. The Company remeasures the derivative liability to fair value at each reporting period and records the change in fair value of the derivative liability as other income (expense), net. The Company uses a present value analysis with multiple scenarios, which incorporates assumptions and estimates to value the derivative instrument. The Company assesses these assumptions and estimates on a periodic basis as additional information impacting the assumptions is obtained. Estimates and assumptions impacting the fair value measurement include the change of control payment to CFF (range of zero to $ 18.9 million at March 31, 2024 and December 31, 2023), the probability of a change of control event, the probability of the product achieving development or commercial status at time of change of control (range of 4.8 % to 17.2 % at March 31, 2024 and December 31, 2023) and the discount rate ( 15 % at March 31, 2024 and December 31, 2023). The Company determined the estimated fair value of this liability as of the inception date of the CFF Agreement and concluded that the amount was immaterial. The Company determined the fair value of this derivative liability was $ 0.4 million as of March 31, 2024 and December 31, 2023. |
Related Party Transactions
Related Party Transactions | 3 Months Ended |
Mar. 31, 2024 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 15. Related Party Transactions In March 2024, the Company entered into a research and option agreement (the “Reignite Agreement”) with Reignite Therapeutics Inc. (“Reignite”), which was founded by David Kirn, M.D., Chief Executive Officer of the Company. Reignite has the expertise to develop high-capacity, helper-dependent adenovirus capsids which the Company plans to utilize as they expand their therapeutic capsid evolution platform. Reignite and the Company plan to collaborate on a one-to-two year program to develop these high capacity, helper-dependent adenovirus capsids. Under the Reignite Agreement, the Company shall have the final authority to amend and make updates to the plan and budget of the program. Further, the Company is responsible for the funding of the related research which includes the budgeted full-time employees and CRO costs, equipment costs not to exceed $ 60 thousand and all other costs, in total not to exceed $ 1.5 million in any year of the program. The Company will have an option to acquire up to three capsids resulting from the program. The Company shall pay to Reignite an option exercise fee of $ 1.0 million per selected capsid for which the Company has exercised its option. The maximum total amount payable is $ 3.0 million. No research and development related expenses and no option exercise fees were incurred during the three months ended March 31, 2024, and as of March 31, 2024, no amounts were due to Reignite under the Reignite Agreement. |
401(K) Plan
401(K) Plan | 3 Months Ended |
Mar. 31, 2024 | |
Retirement Benefits [Abstract] | |
401(K) Plan | 16. 401(k) Plan In 2014, the Company adopted a 401(k) plan for all employees who have met certain eligibility requirements. The 401(k) plan allows employees to make pre-tax and post-tax contributions up to the maximum allowable amount set by the Internal Revenue Service. The Company made contributions to the 401(k) plan for all eligible participants and recorded contribution expenses of $ 0.4 million for each of the three months ended March 31 , 2024 and 2023. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with United States Generally Accepted Accounting Principles (“U.S. GAAP”) and the rules and regulations of the United States Securities and Exchange Commission (“SEC”) for interim reporting. Certain information and note disclosures normally included in annual financial statements prepared in accordance with U.S. GAAP have been condensed or omitted. Accordingly, the unaudited condensed financial statements should be read in conjunction with the audited financial statements and the related notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 filed with the SEC. The accompanying financial information for the three months ended March 31, 2024 and 2023 is unaudited. The unaudited condensed financial statements have been prepared on the same basis as the annual audited financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s financial position as of March 31, 2024 and December 31, 2023 and its results of operations and cash flows for the three months ended March 31, 2024 and 2023. The results for interim periods are not necessarily indicative of the results expected for the full fiscal year or any other periods. |
Use of Estimates and Judgements | Use of Estimates and Judgments The preparation of condensed financial statements in conformity with U.S. GAAP requires management to make estimates and judgments that affect the reported amounts of assets, liabilities, revenue and expenses; and disclosure of contingent assets and liabilities as of the date of the financial statements. Such estimates include the determination of useful lives for property and equipment, the contract term, transaction price and costs of collaboration agreements, as well as estimates of the fair value of common stock (prior to the IPO), stock options and the derivative instrument and income tax uncertainties. Actual results could differ from those estimates. Due to the war in Ukraine, conflicts in the Middle East, rising interest rates and inflation, and the lingering impacts of the coronavirus (“COVID-19”) pandemic, there has been uncertainty and disruption in the global economy and financial markets. The Company is not aware of any specific event or circumstance that would require an update to its estimates or judgments or a revision of the carrying value of its assets or liabilities as of March 31, 2024. While there was not a material impact to the Company’s unaudited condensed financial statements as of March 31, 2024 , these estimates may change, as new events occur and additional information is obtained, as well as other factors that could result in material impacts to the unaudited condensed financial statements in future reporting periods. |
Segment Information | Segment Information The Company operates and manages its business as one reportable and operating segment. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on a company-wide basis for purposes of allocating resources and assessing financial performance. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, marketable securities and accounts receivable. The Company’s cash is held at two financial institutions in the United States of America. The Company’s cash equivalents are invested in money market funds. The Company also invests in U.S. Treasuries, U.S. government sponsored agencies, commercial paper, corporate bonds and certificates of deposit. The Company has not experienced any losses on its deposits of cash and cash equivalents. Such deposits may, at times, exceed federally insured limits. The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Three Months Ended March 31, 2024 2023 Customer A 0 % 99 % Customer B 100 % * Total 100 % 99 % * Less than 10 % The Company did no t have accounts receivable from its partners in collaboration and license agreements as of March 31, 2024 and December 31, 2023. The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Three Months Ended March 31, 2024 2023 Netherlands $ — $ 295 United States 28 3 Total revenue $ 28 $ 298 |
Other Risks and Uncertainties | Other Risks and Uncertainties The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, suppliers for key raw materials, contract manufacturing organizations (“CMOs”) and contract research organizations (“CROs”), compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies, clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance and reporting. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third parties (including for clinical trials and some aspects of research and preclinical testing). |
Recently Issued Accounting Pronouncements Not Yet Adopted | Recently Issued Accounting Pronouncements Not Yet Adopted On December 14, 2023, the Financial Accounting Standards Board issued Accounting Standards Update (“ASU”) No. 2023-09, Improvements to Income Tax Disclosures. The final guidance adds clarifications related to the presentation of rate reconciliation for public business entities and definitions of specific categories in rate reconciliation. These amendments are effective for public business entities for annual periods beginning after December 15, 2024. For other entities, amendments are effective for annual periods beginning after December 15, 2025. The Company is not early adopting ASU 2023-09. The Company expects to make additional disclosures for income taxes upon adoption of this ASU. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Accounting Policies [Abstract] | |
Schedule of Concentration of Risk | The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Three Months Ended March 31, 2024 2023 Customer A 0 % 99 % Customer B 100 % * Total 100 % 99 % * Less than 10 % The Company did no t have accounts receivable from its partners in collaboration and license agreements as of March 31, 2024 and December 31, 2023. |
Schedule of Revenues by Geographic Region | The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Three Months Ended March 31, 2024 2023 Netherlands $ — $ 295 United States 28 3 Total revenue $ 28 $ 298 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Fair Value Disclosures [Abstract] | |
Fair Value Hierarchy for Financial Assets and Financial Liabilities Measured at Fair Value on Recurring Basis | The following tables represent the Company’s fair value hierarchy for financial assets and financial liabilities measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023 (in thousands): Basis for Fair Value Measurements Fair Value as of Level 1 Level 2 Level 3 March 31, 2024 Assets Money market funds $ 245,111 $ — $ — $ 245,111 Commercial paper — 64,624 — 64,624 Corporate bonds — 169,106 — 169,106 U.S. Treasuries — 26,262 — 26,262 U.S. government sponsored agencies — 1,398 — 1,398 Certificates of deposit — 51,869 — 51,869 Total $ 245,111 $ 313,259 $ — $ 558,370 Liabilities Derivative liability $ — $ — $ 369 $ 369 Total $ — $ — $ 369 $ 369 Basis for Fair Value Measurements Fair Value as of Level 1 Level 2 Level 3 December 31, 2023 Assets Money market funds $ 217,644 $ — $ — $ 217,644 Commercial paper — 18,243 — 18,243 Corporate bonds — 28,939 — 28,939 U.S. government sponsored agencies — 2,896 — 2,896 Total $ 217,644 $ 50,078 $ — $ 267,722 Liabilities Derivative liability $ — $ — $ 369 $ 369 Total $ — $ — $ 369 $ 369 |
Summary of Changes in Fair Value of Level 3 Financial Instruments | The following tables set forth a summary of the changes in the fair value of the Company’s Level 3 financial instrument during the three-month periods ended March 31, 2024 and 2023 (in thousands): Derivative Balance as of December 31, 2023 $ 369 Change in fair value included in other income (expense), net — Balance as of March 31, 2024 $ 369 Derivative Balance as of December 31, 2022 $ 212 Change in fair value included in other income (expense), net 7 Balance as of March 31, 2023 $ 219 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available for Sale Securities | Marketable securities, which are classified as available-for-sale, consisted of the following as of March 31, 2024 and December 31, 2023 (in thousands): Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of March 31, 2024 Short-term marketable securities: Commercial paper $ 61,428 $ 5 $ ( 46 ) $ 61,387 Corporate bonds 109,432 16 ( 47 ) 109,401 U.S. Treasuries 26,262 1 ( 1 ) 26,262 U.S. government sponsored agencies 1,400 — ( 2 ) 1,398 Certificates of deposit 51,875 3 ( 9 ) 51,869 Total short-term marketable securities $ 250,397 $ 25 $ ( 105 ) $ 250,317 Long-term marketable securities: Commercial paper $ 3,234 $ 3 $ — $ 3,237 Corporate bonds 59,737 14 ( 46 ) 59,705 Total long-term marketable securities $ 62,971 $ 17 $ ( 46 ) $ 62,942 Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of December 31, 2023 Short-term marketable securities: Commercial paper $ 17,603 $ 1 $ ( 26 ) $ 17,578 Corporate bonds 18,641 12 ( 3 ) 18,650 U.S. government sponsored agencies 2,900 — ( 4 ) 2,896 Total short-term marketable securities $ 39,144 $ 13 $ ( 33 ) $ 39,124 Long-term marketable securities: Corporate bonds $ 661 $ 4 $ — $ 665 Commercial paper 10,257 32 — 10,289 Total long-term marketable securities $ 10,918 $ 36 $ — $ 10,954 |
Summary of Aggregate Fair Values of Marketable Securities with Unrealized Losses and Gains | Aggregate fair values of marketable securities with unrealized losses and gains were as follows as of March 31, 2024 and December 31, 2023 (in thousands): March 31, December 31, 2024 2023 Aggregate fair value of marketable securities in a continuous loss position for less than twelve months $ 209,564 $ 20,494 Aggregate fair value of marketable securities in a continuous loss position for more than twelve months — 4,986 Aggregate fair value of marketable securities in unrealized gain position 103,695 24,598 Total marketable securities $ 313,259 $ 50,078 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net, consisted of the following (in thousands): March 31, December 31, Machinery and equipment $ 13,605 $ 12,996 Leasehold improvements 17,325 16,902 Furniture and fixtures 566 566 Office equipment 239 239 Computer equipment and software 835 805 Construction in progress — 550 Total property and equipment 32,570 32,058 Less: Accumulated depreciation and amortization ( 13,106 ) ( 11,932 ) Property and equipment, net $ 19,464 $ 20,126 |
Accrued and Other Current Lia_2
Accrued and Other Current Liabilities (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Payables and Accruals [Abstract] | |
Summary of Accrued and Other Current Liabilities | Accrued and other current liabilities consisted of the following (in thousands): March 31, December 31, Payroll and related expenses $ 4,516 $ 8,149 Accrued clinical and preclinical study costs 2,908 2,180 Consulting and professional 1,447 1,581 Other accrued expenses 142 104 Total accrued and other current liabilities $ 9,013 $ 12,014 |
Research and Collaboration Ar_2
Research and Collaboration Arrangements (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Research And Collaboration Arrangements [Abstract] | |
Summary of Collaboration and License Revenue | Collaboration and license revenue for each period was as follows (in thousands): Three Months Ended March 31, 2024 2023 uniQure $ — $ 295 CFF 28 3 Total revenue $ 28 $ 298 |
Summary of Deferred Revenue | Deferred revenue is summarized as follows (in thousands): March 31, December 31, CFF $ 1,322 $ 1,245 Total deferred revenue $ 1,322 $ 1,245 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Summary of Components of Lease Expense | The following table summarizes the components of lease expense for the three months ended March 31, 2024 and 2023, which are included in operating expenses in the Company’s condensed statements of operations (in thousands): Three Months Ended March 31, 2024 2023 Operating lease cost $ 698 $ 697 Variable lease cost 354 486 Total $ 1,052 $ 1,183 |
Summary of Supplemental Information Related to Leases | The following table summarizes supplemental information related to operating leases: March 31, December 31, Weighted-average remaining lease term (in years): 5.5 5.7 Weighted-average discount rate: 7.8 % 7.8 % |
Summary of Maturities of Lease Liabilities | The following table summarizes the maturities of lease liabilities as of March 31, 2024 (in thousands): 2024 (remaining nine months) $ 2,371 2025 3,244 2026 3,135 2027 2,810 2028 2,894 Thereafter 2,981 Total future minimum lease payments 17,435 Less: Amount representing interest ( 3,264 ) Present value of future minimum lease payments 14,171 Less: Current portion of operating lease liabilities 3,173 Long-term portion of operating lease liabilities $ 10,998 |
Common Stock (Tables)
Common Stock (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Equity [Abstract] | |
Schedule of Common Shares Reserved for Future Issuance | As of March 31, 2024 and December 31, 2023, the Company has reserved common stock, on an as-converted basis, for future issuance as follows: March 31, December 31, Issuance of common stock under the 2020 Equity Incentive Award Plan 2,426,209 524,078 Issuance of common stock under the 2020 Employee Stock Purchase Plan 363,034 313,034 Exercise of options issued and outstanding and future vesting of restricted stock units 8,438,028 8,423,481 Exercise of common stock warrants 3,637,145 53,669 Total common stock reserved for future issuance 14,864,416 9,314,262 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Restricted Stock Units (RSU) | The Company has granted restricted stock unit (RSU) awards under the 2020 Equity Incentive Award Plan that vest over a period of four years . The following table summarizes the RSU activity for the three months ended March 31, 2024: Number of Unvested balance at December 31, 2023 181,181 Issued 55,400 Vested ( 10,996 ) Canceled/Forfeited ( 15,685 ) Unvested balance at March 31, 2024 209,900 |
Summary of Stock Options Activity | The following table summarizes the stock options activity for the three months ended March 31, 2024: Number of Balances at December 31, 2023 8,240,154 Options granted 286,000 Options exercised ( 225,860 ) Options expired ( 7,081 ) Options forfeited ( 65,085 ) Balances at March 31, 2024 8,228,128 Options exercisable at March 31, 2024 3,321,825 Options vested and expected to vest at March 31, 2024 8,228,128 |
Summary of Assumptions Used in Black-Scholes Valuation Model to Estimate Fair Value of Stock Options | The Company estimates the fair value of stock options using the Black-Scholes option pricing model. The fair value of employee and nonemployee options is recognized on a straight-line basis over the requisite service period of the awards. The fair value of the Company’s stock options was estimated using the following assumptions for the three months ended March 31, 2024 and 2023: Three Months Ended March 31, 2024 2023 Expected term 6.0 - 6.1 years 6.0 - 6.1 years Expected volatility 81.4 % - 85.3 % 80.9 % - 84.5 % Risk-free interest rate 3.9 % - 4.3 % 3.7 % - 4.0 % Expected dividend yield 0 % 0 % |
Summary of Stock-based Compensation Expense for Employees and Nonemployees by Function | The following table is a summary of stock-based compensation expense incurred for employees and nonemployees by function (in thousands): Three Months Ended 2024 2023 Research and development $ 3,034 $ 2,470 General and administrative 2,940 1,751 Total stock-based compensation expense $ 5,974 $ 4,221 |
Net Loss Per Share, Basic and_2
Net Loss Per Share, Basic and Diluted (Tables) | 3 Months Ended |
Mar. 31, 2024 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Loss Per Share | The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Three Months Ended March 31, 2024 2023 Numerator Net loss $ ( 32,401 ) $ ( 28,682 ) Denominator Weighted-average shares outstanding used in computing net loss per share, basic and diluted 49,271,984 32,723,530 Net loss per share, basic and diluted $ ( 0.66 ) $ ( 0.88 ) |
Outstanding Shares of Potentially Dilutive Securities Excluded from Computation of Diluted Net Loss Per Share | The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: March 31, 2024 2023 Options issued and outstanding 8,228,128 6,589,104 Restricted stock units subject to future vesting 209,900 — 2020 ESPP 250,727 360,769 Common stock warrants 53,669 53,669 Total 8,742,424 7,003,542 |
The Company - Additional Inform
The Company - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | ||||||
Mar. 31, 2024 | Feb. 29, 2024 | May 31, 2023 | Nov. 30, 2021 | Dec. 31, 2020 | Dec. 31, 2023 | Jan. 01, 2021 | |
Organization And Nature Of Business [Line Items] | |||||||
Outstanding shares of redeemable convertible preferred stock | 0 | ||||||
Accumulated deficit | $ 447,728 | $ 415,327 | |||||
Common Stock | Initial Public Offering | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares sold and issued | 9,660,000 | ||||||
Stock sold and issued price per share | $ 23 | ||||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 204,700 | ||||||
Number shares issued upon conversion of redeemable convertible preferred shares | 11,575,984 | ||||||
Common Stock | Exercise of Overallotment Option | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares sold and issued | 1,259,299 | 1,260,000 | |||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 34,900 | ||||||
Common Stock | 2021 Follow On Public Offering | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares priced under public offering | 4,750,000 | ||||||
Stock sold and issued price per share | $ 25 | ||||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 111,100 | ||||||
Common Stock | 2023 Follow On Public Offering | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares priced under public offering | 8,625,000 | ||||||
Stock sold and issued price per share | $ 16 | ||||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 129,200 | ||||||
Common Stock | 2024 Follow On Public Offering | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares priced under public offering | 6,586,015 | ||||||
Stock sold and issued price per share | $ 29.5 | ||||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 281,200 | ||||||
Warrants to purchase shares of common stock | 3,583,476 | ||||||
Common Stock | 2024 Follow On Public Offering | Pre-funded Warrants | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Warrants to purchase shares of common stock | 3,583,476 | ||||||
Common stock offering price | $ 29.4999 | ||||||
Common Stock | Maximum | Exercise of Overallotment Option | |||||||
Organization And Nature Of Business [Line Items] | |||||||
Number of shares sold and issued | 1,525,423 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) - Segment | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Summary Of Significant Accounting Policies [Line Items] | ||
Number of reportable segments | 1 | |
Number of operating segments | 1 | |
Accounts Receivable | Credit Concentration Risk | Customer | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Concentration risk, percentage | 0% | 0% |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule of Concentration of Risk (Details) - Revenue - Customer Concentration Risk | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Customer A | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 0% | 99% |
Customer B | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 100% | |
Customer | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 100% | 99% |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Schedule of Concentration of Risk (Details) (Parenthetical) - Revenue - Customer Concentration Risk | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Customer A | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 0% | 99% |
Customer B | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 100% | |
Maximum | Customer B | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 10% |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Revenues by Geographic Region (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Disaggregation Of Revenue [Line Items] | ||
Total revenue | $ 28 | $ 298 |
Netherlands | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenue | 0 | 295 |
United States | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenue | $ 28 | $ 3 |
Fair Value Measurements - Fair
Fair Value Measurements - Fair Value Hierarchy for Financial Assets and Financial Liabilities Measured at Fair Value on Recurring Basis (Details) - Fair Value on Recurring Basis - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | $ 558,370 | $ 267,722 |
Liabilities | 369 | 369 |
Derivative Liability | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | 369 | 369 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 64,624 | 18,243 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 169,106 | 28,939 |
U.S. Treasuries | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 26,262 | |
U.S. Government Sponsored Agencies | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 1,398 | 2,896 |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 245,111 | 217,644 |
Certificates of Deposit | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 51,869 | |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 245,111 | 217,644 |
Level 1 | Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 245,111 | 217,644 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 313,259 | 50,078 |
Level 2 | Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 64,624 | 18,243 |
Level 2 | Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 169,106 | 28,939 |
Level 2 | U.S. Treasuries | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 26,262 | |
Level 2 | U.S. Government Sponsored Agencies | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 1,398 | 2,896 |
Level 2 | Certificates of Deposit | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 51,869 | |
Level 3 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | 369 | 369 |
Level 3 | Derivative Liability | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | $ 369 | $ 369 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) - USD ($) | Mar. 31, 2024 | Dec. 31, 2023 |
Fair Value Disclosures [Abstract] | ||
Assets, Level 1 and Level 2 transfers | $ 0 | $ 0 |
Liabilities, Level 1 and Level 2 transfers | $ 0 | $ 0 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Changes in Fair Value of Level 3 Financial Instruments (Details) - Derivative Liability - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Beginning Balance | $ 369 | $ 212 |
Change in fair value included in other income (expense), net | $ 0 | $ 7 |
Fair Value, Recurring Basis, Unobservable Input Reconciliation, Liability, Gain (Loss), Statement of Income [Extensible List] | Other expense, net | Other expense, net |
Ending Balance | $ 369 | $ 219 |
Marketable Securities - Summary
Marketable Securities - Summary of Available for Sale Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | $ 250,397 | $ 39,144 |
Gross Unrealized Gains | 25 | 13 |
Gross Unrealized Losses | (105) | (33) |
Fair Value | 250,317 | 39,124 |
Long-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 62,971 | 10,918 |
Gross Unrealized Gains | 17 | 36 |
Gross Unrealized Losses | (46) | 0 |
Fair Value | 62,942 | 10,954 |
Commercial Paper | Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 61,428 | 17,603 |
Gross Unrealized Gains | 5 | 1 |
Gross Unrealized Losses | (46) | (26) |
Fair Value | 61,387 | 17,578 |
Commercial Paper | Long-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 3,234 | 10,257 |
Gross Unrealized Gains | 3 | 32 |
Gross Unrealized Losses | 0 | 0 |
Fair Value | 3,237 | 10,289 |
Corporate Bonds | Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 109,432 | 18,641 |
Gross Unrealized Gains | 16 | 12 |
Gross Unrealized Losses | (47) | (3) |
Fair Value | 109,401 | 18,650 |
Corporate Bonds | Long-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 59,737 | 661 |
Gross Unrealized Gains | 14 | 4 |
Gross Unrealized Losses | (46) | 0 |
Fair Value | 59,705 | 665 |
U.S. Treasuries | Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 26,262 | |
Gross Unrealized Gains | 1 | |
Gross Unrealized Losses | (1) | |
Fair Value | 26,262 | |
U.S. Government Sponsored Agencies | Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 1,400 | 2,900 |
Gross Unrealized Gains | 0 | 0 |
Gross Unrealized Losses | (2) | (4) |
Fair Value | 1,398 | $ 2,896 |
Certificates of Deposit | Short-term Marketable Securities | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Amortized Cost Basis | 51,875 | |
Gross Unrealized Gains | 3 | |
Gross Unrealized Losses | (9) | |
Fair Value | $ 51,869 |
Marketable Securities - Summa_2
Marketable Securities - Summary of Aggregate fair values of marketable securities with unrealized losses and gains (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Debt Securities, Available-for-Sale [Line Items] | ||
Aggregate fair value of securities held in a continuous unrealized loss position for less than twelve months | $ 209,564 | $ 20,494 |
Aggregate fair value of marketable securities in a continuous loss position for more than twelve months | 0 | 4,986 |
Aggregate fair value of marketable securities in unrealized gain position | 103,695 | 24,598 |
Total marketable securities | $ 313,259 | $ 50,078 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Debt Securities, Available-for-Sale [Line Items] | ||
Realized gains or losses on marketable securities | $ 0 | $ 0 |
Maximum unrealized loss at individual instrument level | 9,000 | |
Aggregate unrealized loss | 151,000 | |
Allowance for credit loss | 0 | 0 |
Other impairment charges related to marketable securities | $ 0 | $ 0 |
Maximum | ||
Debt Securities, Available-for-Sale [Line Items] | ||
Maturity period of marketable securities | 2 years |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 32,570 | $ 32,058 |
Less: Accumulated depreciation and amortization | (13,106) | (11,932) |
Property and equipment, net | 19,464 | 20,126 |
Machinery and Equipment | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 13,605 | 12,996 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 17,325 | 16,902 |
Furniture and Fixtures | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 566 | 566 |
Office Equipment | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 239 | 239 |
Computer Equipment and Software | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 835 | 805 |
Construction in Progress | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 0 | $ 550 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation expense | $ 1.2 | $ 1 |
Accrued and Other Current Lia_3
Accrued and Other Current Liabilities - Summary of Accrued and Other Current Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Payables and Accruals [Abstract] | ||
Payroll and related expenses | $ 4,516 | $ 8,149 |
Accrued clinical and preclinical study costs | 2,908 | 2,180 |
Consulting and professional | 1,447 | 1,581 |
Other accrued expenses | 142 | 104 |
Total accrued and other current liabilities | $ 9,013 | $ 12,014 |
Research and Collaboration Ar_3
Research and Collaboration Arrangements - Summary of Collaboration and License Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total revenue | $ 28 | $ 298 |
Collaboration and License Revenue | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total revenue | 28 | 298 |
Collaboration and License Revenue | uniQure | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total revenue | 0 | 295 |
Collaboration and License Revenue | CFF | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total revenue | $ 28 | $ 3 |
Research and Collaboration Ar_4
Research and Collaboration Arrangements - Summary of Deferred Revenue (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | $ 1,322 | $ 1,245 |
CFF | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | $ 1,322 | $ 1,245 |
Research and Collaboration Ar_5
Research and Collaboration Arrangements - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | ||||
Jul. 05, 2023 | Jan. 31, 2014 | Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2023 | Aug. 31, 2019 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Revenue recognized | $ 0 | $ 300,000 | ||||
Deferred revenue | 1,322,000 | $ 1,245,000 | ||||
Astellas GeneTherapies Inc | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Upfront payments received | $ 20,000,000 | |||||
uniQure | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Accounts receivable | 0 | 0 | ||||
uniQure | uniQure Agreement | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Collaborative arrangement, initial research term | 3 years | |||||
Upfront payments received | $ 200,000 | |||||
Options to purchase ordinary shares | 609,744 | |||||
Ordinary shares vesting period | 3 years | |||||
Fair value of options | $ 10,600,000 | |||||
Fair value of non monetary consideration of intellectual property right | $ 5,100,000 | |||||
uniQure | uniQure Agreement | Maximum | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Contingent payments entitled to receive for achievement of research and development milestones | $ 5,000,000 | |||||
uniQure | Amended uniQure Agreement | Maximum | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Contingent payments eliminated | $ 5,000,000 | |||||
uniQure | Amended uniQure Agreement and Second uniQure Agreement | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Revenue recognized | 0 | $ 300,000 | ||||
Deferred revenue | 0 | 0 | ||||
Remaining performance obligations | 0 | $ 0 | ||||
Arbor Biotechnologies Inc | Arbor Agreement | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Shared product designated by JSC | 0 | |||||
Research and development expenses | $ 0 |
Research and Collaboration Ar_6
Research and Collaboration Arrangements - Additional Information (Details 1) | Jan. 31, 2014 |
uniQure | uniQure Agreement | Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date: 2024-04-01 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 4 years |
Research and Collaboration Ar_7
Research and Collaboration Arrangements - CFF - Additional Information (Details) - CFF Agreement - CFF | 1 Months Ended | 3 Months Ended | 12 Months Ended | |
Aug. 31, 2023 USD ($) | Sep. 30, 2016 USD ($) Installment | Mar. 31, 2024 USD ($) | Dec. 31, 2023 USD ($) | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Collaborative arrangement total amount of award | $ 6,300,000 | $ 3,500,000 | ||
Collaborative arrangement achieved milestones | $ 1,800,000 | $ 1,700,000 | ||
Expected payments multiples to equal actual award received | 6 | |||
Number of installments to pay actual award | Installment | 3 | |||
Actual award payment period upon first commercial sale of product | 4 years | |||
Continuous period where the Company ceases to develop or commercialize any product | 180 days | |||
Collaborative arrangements special term | If at any time prior to the first commercial sale of a product developed as a result of the CFF Agreement, the Company ceases to use commercially reasonable efforts to develop or commercialize any product under the CFF Agreement for a continuous period of 180 consecutive days and fails to present a reasonable plan to resume commercially reasonable efforts, the Company will grant to CFF an irrevocable, exclusive worldwide interruption license under all of the Company’s interest in the research plan technology to exploit such product. Any third-party license granted by the Company shall be subject to such interruption license. | |||
Deferred revenue | 1,300,000 | 1,200,000 | ||
Accounts receivable | 0 | 0 | ||
Aggregate amount of transaction price allocated to remaining performance obligation | $ 1,300,000 | $ 1,200,000 | ||
Maximum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Future sales-based milestone payments multiples | 3 | |||
Royalties multiples on actual award received | 6 |
Research and Collaboration Ar_8
Research and Collaboration Arrangements - CFF - Additional Information (Details 1) - CFF | Mar. 31, 2024 |
Minimum | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 1 year |
Maximum | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 3 years |
License Arrangements - Addition
License Arrangements - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | ||||||
Jul. 05, 2023 | Apr. 21, 2023 | Jul. 31, 2021 | Jan. 31, 2019 | Jan. 31, 2016 | Mar. 31, 2024 | Mar. 31, 2023 | Dec. 31, 2018 | |
Astellas GeneTherapies Inc | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Upfront payments received | $ 20,000,000 | |||||||
Contingent payments development milestones initial target | 15,000,000 | |||||||
Deferred revenue | $ 0 | |||||||
Accounts receivable | 0 | |||||||
Astellas GeneTherapies Inc | Maximum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Future option fees and milestones | $ 942,500,000 | |||||||
Aevitas Therapeutics, Inc. Agreement | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Upfront consideration payment | $ 0 | |||||||
Liability related to contingent consideration | 0 | |||||||
Aevitas Therapeutics, Inc. Agreement | Maximum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Potential cash payment upon achievement of certain late-stage development, regulatory and sales milestones | 144,000,000 | |||||||
License Agreement for sCFH with University of Pennsylvania | Maximum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Potential cash payment upon achievement of certain late-stage development, regulatory and sales milestones | $ 42,000,000 | |||||||
UC Regents and the Trustees of the University of Pennsylvania | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Amount paid non-refundable license fee | $ 100,000 | |||||||
Non-refundable license fee payable annually | 10,000 | |||||||
Incurred expenses | $ 0 | $ 0 | ||||||
UC Regents and the Trustees of the University of Pennsylvania | Maximum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Contingent payments development milestones | 3,900,000 | |||||||
UC Regents and the Trustees of the University of Pennsylvania | Minimum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Annual royalty | $ 100,000 | |||||||
UC Agreements | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Non-refundable license fee payable upon execution | $ 5,000 | |||||||
Non-refundable license fee payable upon execution every year thereafter until sales of a licensed product and payment of royalties | 5,000 | |||||||
Amount obligated to pay upon closing first qualified financing at option of UC Regents | $ 50,000 | |||||||
Percentage of equity interest upon closing of first qualified financing at option of UC Regents | 3% | |||||||
Percentage of equity interests issued as common stock | 6% | |||||||
Amount paid non-refundable license fee | $ 50,000 | |||||||
Non-refundable license fee payable annually | 5,000 | |||||||
UC Agreements | Maximum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Contingent payments development milestones | 3,100,000 | |||||||
UC Agreements | Minimum | ||||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||||
Annual royalty | $ 100,000 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||
Jan. 01, 2024 USD ($) ft² | Feb. 28, 2021 USD ($) | Dec. 31, 2019 USD ($) | Nov. 30, 2018 USD ($) | Oct. 31, 2018 | May 31, 2015 | Mar. 31, 2024 USD ($) | Dec. 31, 2023 USD ($) | Jan. 31, 2016 | |
Commitment And Contingencies [Line Items] | |||||||||
Operating lease right-of-use ("ROU") assets | $ 11,118 | $ 11,539 | |||||||
Operating lease liability | 14,171 | ||||||||
Future minimum lease payments | 17,435 | ||||||||
5980 Horton Street Building Lease | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Lease extended term date | Mar. 31, 2023 | ||||||||
Operating lease right-of-use ("ROU") assets | 1,100 | 1,200 | |||||||
Operating lease liability | $ 1,300 | 1,400 | |||||||
Operating lease, discount rate | 7.50% | ||||||||
Lease extended term period | 2026-09 | ||||||||
Tenant improvement allowance | $ 200 | ||||||||
Lease renewal term | 3 years | 5 years | |||||||
5858 Horton Street Building Lease and Expansion | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Operating lease right-of-use ("ROU") assets | $ 10,000 | 10,300 | |||||||
Operating lease liability | $ 12,900 | 13,200 | |||||||
Operating lease, discount rate | 7.80% | ||||||||
Tenant improvement allowance | $ 2,300 | $ 400 | $ 2,300 | ||||||
Lease initial term | 87 months | ||||||||
Lease option to renew term description | The new lease has an initial term of 87 months beginning on the rent commencement date with the option to renew the lease for one additional term of five years at market rent. | ||||||||
Lease existence of option to renew term | true | ||||||||
Lease renewal term | 5 years | ||||||||
Warehouse Lease Agreement | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Operating lease right-of-use ("ROU") assets | $ 0 | ||||||||
Operating lease liability | $ 0 | ||||||||
Lease initial term | 5 years 4 months | ||||||||
Lease commencement date | Aug. 31, 2024 | ||||||||
Area of warehouse space | ft² | 7,800 | ||||||||
Future minimum lease payments | $ 1,500 |
Commitments and Contingencies-
Commitments and Contingencies- Summary of Components of Lease Expense - (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | ||
Operating lease cost | $ 698 | $ 697 |
Variable lease cost | 354 | 486 |
Total | $ 1,052 | $ 1,183 |
Commitments and Contingencies_2
Commitments and Contingencies - Summary of Supplemental Information Related to Leases - (Details) | Mar. 31, 2024 | Dec. 31, 2023 |
Commitments and Contingencies Disclosure [Abstract] | ||
Weighted-average remaining lease term (in years) | 5 years 6 months | 5 years 8 months 12 days |
Weighted-average discount rate | 7.80% | 7.80% |
Commitments and Contingencies_3
Commitments and Contingencies - Summary of Maturities of Lease Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2024 | Dec. 31, 2023 |
Commitments and Contingencies Disclosure [Abstract] | ||
2024 (remaining nine months) | $ 2,371 | |
2025 | 3,244 | |
2026 | 3,135 | |
2027 | 2,810 | |
2028 | 2,894 | |
Thereafter | 2,981 | |
Total future minimum lease payments | 17,435 | |
Less: Amount representing interest | (3,264) | |
Present value of future minimum lease payments | 14,171 | |
Less: Current portion of operating lease liabilities | 3,173 | $ 3,149 |
Long-term portion of operating lease liabilities | $ 10,998 | $ 11,522 |
Common Stock - Additional Infor
Common Stock - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | 12 Months Ended | |
Oct. 31, 2021 | Apr. 30, 2020 | Mar. 31, 2024 | Dec. 31, 2023 | |
Class of Stock [Line Items] | ||||
Common stock, shares authorized | 300,000,000 | 300,000,000 | ||
Common stock, par value | $ 0.0001 | $ 0.0001 | ||
Common stock voting rights | one vote per share | |||
Common stock dividend | $ 0 | $ 0 | ||
Funding Agreement | CFF | ||||
Class of Stock [Line Items] | ||||
Additional funding upon acceptance of investigational New Drug application | $ 4,000,000 | $ 4,000,000 | ||
Additional investment in cash | $ 4,000,000 | |||
Funding Agreement | CFF | Common Stock | ||||
Class of Stock [Line Items] | ||||
Number of shares sold and issued | 125,715 | |||
Funding Agreement | CFF | Series C Redeemable Convertible Preferred Stock | ||||
Class of Stock [Line Items] | ||||
Investment in redeemable convertible preferred stock financing | $ 10,000,000 |
Common Stock - Schedule of Comm
Common Stock - Schedule of Common Share Reserved for Future Issuance (Details) - shares | Mar. 31, 2024 | Dec. 31, 2023 | Dec. 31, 2020 |
Class Of Stock [Line Items] | |||
Common stock reserved | 14,864,416 | 9,314,262 | |
2020 Equity Incentive Award Plan | |||
Class Of Stock [Line Items] | |||
Common stock reserved | 2,426,209 | 524,078 | 2,606,546 |
2020 Employee Stock Purchase Plan | |||
Class Of Stock [Line Items] | |||
Common stock reserved | 363,034 | 313,034 | 252,337 |
Exercise of Options Issued and Outstanding and Future Vesting of Restricted Stock Units | |||
Class Of Stock [Line Items] | |||
Common stock reserved | 8,438,028 | 8,423,481 | |
Common Stock Warrants | |||
Class Of Stock [Line Items] | |||
Common stock reserved | 3,637,145 | 53,669 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Details) | 3 Months Ended | ||||
Mar. 31, 2024 Period shares | Mar. 31, 2023 | Feb. 29, 2024 shares | Dec. 31, 2023 shares | Dec. 31, 2020 shares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares reserved for issuance | 14,864,416 | 9,314,262 | |||
Number of options outstanding | 8,228,128 | 8,240,154 | |||
Expected dividend yield | 0% | 0% | |||
2020 Equity Incentive Award Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares reserved for issuance | 2,426,209 | 524,078 | 2,606,546 | ||
Percentage of common stock outstanding increased annually for future issuance | 5% | ||||
Number of shares available for grant | 2,090,604 | 2,153,533 | |||
2020 Equity Incentive Award Plan | Restricted Stock Units (RSU) | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Vesting period | 4 years | ||||
2020 Equity Incentive Award Plan | Maximum | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares authorized | 18,000,000 | ||||
Options exercisable period | 10 years | ||||
2015 Equity Incentive Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares available for grant | 335,605 | ||||
Options exercisable period | 10 years | ||||
Number of options outstanding | 1,563,448 | ||||
Number of additional shares available for grant | 0 | ||||
2020 ESPP | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares reserved for issuance | 363,034 | 313,034 | 252,337 | ||
Number of shares available for grant | 50,000 | ||||
Increase in shares available for issuance percentage | 1% | ||||
Employee purchase price of common stock as percentage of fair market value | 85% | ||||
Common stock overlapping offering period | 24 months | ||||
Number of duration purchase period | Period | 4 | ||||
Percentage of maximum contributions of employee's eligible compensation | 15% | ||||
2020 ESPP | Maximum | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares authorized | 15,000,000 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Restricted Stock Units (RSU) (Details) - Restricted Stock Units (RSU) | 3 Months Ended |
Mar. 31, 2024 shares | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Number of Shares, Unvested Beginning Balance | 181,181 |
Issued | 55,400 |
Vested | (10,996) |
Canceled/Forfeited | (15,685) |
Number of Shares, Unvested Ending Balance | 209,900 |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of Stock Options Activity (Details) | 3 Months Ended |
Mar. 31, 2024 shares | |
Share-Based Payment Arrangement [Abstract] | |
Number of Shares Underlying Outstanding Options, Balances at December 31, 2023 | 8,240,154 |
Number of Shares Underlying Outstanding Options, granted | 286,000 |
Number of Shares Underlying Outstanding Options, exercised | (225,860) |
Number of Shares Underlying Outstanding Options, expired | (7,081) |
Number of Shares Underlying Outstanding Options, forfeited | (65,085) |
Number of Shares Underlying Outstanding Options, Balances at March 31, 2024 | 8,228,128 |
Number of Shares Underlying Outstanding Options, Options exercisable at March 31, 2024 | 3,321,825 |
Number of Shares Underlying Outstanding Options, Options vested and expected to vest at March 31, 2024 | 8,228,128 |
Stock-based Compensation - Sche
Stock-based Compensation - Schedule of Assumptions Used in Black-Scholes Valuation Model to Estimate Fair Value of Stock Options (Details) | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected volatility, minimum | 81.40% | 80.90% |
Expected volatility, maximum | 85.30% | 84.50% |
Risk-free interest rate, minimum | 3.90% | 3.70% |
Risk-free interest rate, maximum | 4.30% | 4% |
Expected dividend yield | 0% | 0% |
Minimum | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected term | 6 years | 6 years |
Maximum | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected term | 6 years 1 month 6 days | 6 years 1 month 6 days |
Stock-based Compensation - Su_3
Stock-based Compensation - Summary of Stock-based Compensation Expense for Employees and Nonemployees by Function (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | $ 5,974 | $ 4,221 |
Research and Development | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | 3,034 | 2,470 |
General and Administrative | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | $ 2,940 | $ 1,751 |
Common Stock Warrants - Additio
Common Stock Warrants - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | |||
Dec. 31, 2020 | May 31, 2018 | Mar. 31, 2024 | Mar. 31, 2023 | Feb. 29, 2024 | |
Class Of Warrant Or Right [Line Items] | |||||
Number of warrant shares issued | 30,000 | 23,669 | |||
Exercise price | $ 18 | $ 3.19 | |||
Warrant expiration year | 2027 | 2025 | |||
Warrant vesting period | 4 years | ||||
Warrant expense | $ 0 | $ 0 | |||
2024 Follow On Public Offering | Common Stock | |||||
Class Of Warrant Or Right [Line Items] | |||||
Warrant shares become exercisable | 3,583,476 | ||||
2024 Follow On Public Offering | Common Stock | Pre-funded Warrants | |||||
Class Of Warrant Or Right [Line Items] | |||||
Exercise price | $ 0.0001 | ||||
Warrant shares become exercisable | 3,583,476 | ||||
Common stock offering price | $ 29.4999 | ||||
Issuance of pre-funded warrants | $ 99,400,000 |
Net Loss Per Share, Basic and_3
Net Loss Per Share, Basic and Diluted - Computation of Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Numerator | ||
Net loss | $ (32,401) | $ (28,682) |
Denominator | ||
Weighted-average shares outstanding used in computing net loss per share, basic | 49,271,984 | 32,723,530 |
Weighted-average shares outstanding used in computing net loss per share, diluted | 49,271,984 | 32,723,530 |
Net loss per share, basic | $ (0.66) | $ (0.88) |
Net loss per share, diluted | $ (0.66) | $ (0.88) |
Net Loss Per Share, Basic and_4
Net Loss Per Share, Basic and Diluted - Additional Information (Details) - $ / shares | Feb. 29, 2024 | Dec. 31, 2020 | May 31, 2018 |
Class of Warrant or Right [Line Items] | |||
Exercise price | $ 18 | $ 3.19 | |
Common Stock | 2024 Follow On Public Offering | |||
Class of Warrant or Right [Line Items] | |||
Warrants to purchase shares of common stock | 3,583,476 | ||
Common Stock | Pre-funded Warrants | 2024 Follow On Public Offering | |||
Class of Warrant or Right [Line Items] | |||
Warrants to purchase shares of common stock | 3,583,476 | ||
Exercise price | $ 0.0001 |
Net Loss Per Share, Basic and_5
Net Loss Per Share, Basic and Diluted - Outstanding Potentially Dilutive Securities Excluded From Computation of Diluted Net Loss Per Share (Details) - shares | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 8,742,424 | 7,003,542 |
Options Issued and Outstanding | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 8,228,128 | 6,589,104 |
Restricted Stock Units Subject to Future Vesting | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 209,900 | 0 |
2020 ESPP | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 250,727 | 360,769 |
Common Stock Warrants | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 53,669 | 53,669 |
Derivative Liability - Addition
Derivative Liability - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | 12 Months Ended |
Mar. 31, 2024 | Dec. 31, 2023 | |
Derivative [Line Items] | ||
Change in control discount rate | 15% | 15% |
Estimated fair value of derivative liability | $ 0.4 | $ 0.4 |
Minimum | ||
Derivative [Line Items] | ||
Change of control payment | $ 0 | $ 0 |
Change in control event percentage | 4.80% | 4.80% |
Maximum | ||
Derivative [Line Items] | ||
Change of control payment | $ 18.9 | $ 18.9 |
Change in control event percentage | 17.20% | 17.20% |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Details) - Reignite Agreement $ in Thousands | 1 Months Ended | 3 Months Ended |
Mar. 31, 2024 USD ($) Product | Mar. 31, 2024 USD ($) Product | |
Related Party Transaction [Line Items] | ||
Research and development expenses | $ 0 | |
Option exercise fee | 0 | |
Option exercise fees per selected capsid | $ 1,000 | |
Amount due to related party | 0 | $ 0 |
Maximum | ||
Related Party Transaction [Line Items] | ||
Related party expenses | 1,500 | |
Equipment costs | $ 60 | |
Number of capsids | Product | 3 | 3 |
Option exercise fee | $ 3,000 |
401(K) Plan - Additional Inform
401(K) Plan - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2024 | Mar. 31, 2023 | |
2014 401(k) Plan | ||
Defined Contribution Plan Disclosure [Line Items] | ||
Employer contribution | $ 0.4 | $ 0.4 |