Item 7A. Quantitative and Qualitative Disclosures About Market Risk
Item 7A. Quantitative and Qualitative Disclosures About Market Risk.
We are exposed to market risk related to changes in interest rates. As of December 31, 2024, we had cash and cash equivalents of $131.5 million, primarily held in money market mutual funds, and marketable securities of $138.4 million, primarily consisting of U.S. government-backed securities, U.S. Treasuries and corporate debt securities. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates, particularly because our investments, including cash equivalents, are in the form, or may be in the form of, money market funds or marketable securities and are or may be invested in U.S. Treasury and U.S. government agency obligations. Due to the short-term maturities and low risk profiles of our investments, an immediate 100 basis point change in interest rates would not have a material effect on the fair market value of our investments.
While we contract with certain vendors and institutions internationally, substantially all of our total liabilities as of December 31, 2024 were denominated in the United States dollar and we believe that we do not have any material exposure to foreign currency exchange rate risk.
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Item 8. Financial Statement and Other Supplementary Information.
EDITAS MEDICINE, INC.
INDEX TO FINANCIAL STATEMENTS
Report of Independent Registered Public Accounting Firm (PCAOB ID 42 )
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Consolidated Balance Sheets
120
Consolidated Statements of Operations
121
Consolidated Statements of Comprehensive Loss
122
Consolidated Statements of Stockholders’ Equity
123
Consolidated Statements of Cash Flows
124
Notes to Consolidated Financial Statements
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Report of Independent Registered Public Accounting Firm
To the Stockholders and the Board of Directors of Editas Medicine, Inc.
Opinion on the Financial Statements
We have audited the accompanying consolidated balance sheets of Editas Medicine, Inc. (the Company) as of December 31, 2024 and 2023, the related consolidated statements of operations, comprehensive loss, stockholders’ equity and cash flows for each of the three years in the period ended December 31, 2024, and the related notes (collectively referred to as the “consolidated financial statements”). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company at December 31, 2024 and 2023, and the results of its operations and its cash flows for each of the three years in the period ended December 31, 2024, in conformity with U.S. generally accepted accounting principles.
Basis for Opinion
These financial statements are the responsibility of the Company‘s management. Our responsibility is to express an opinion on the Company‘s financial statements based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicable rules and regulations of the Securities and Exchange Commission and the PCAOB.
We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of material misstatement, whether due to error or fraud. The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our audits we are required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness of the Company's internal control over financial reporting. Accordingly, we express no such opinion.
Our audits included performing procedures to assess the risks of material misstatement of the financial statements, whether due to error or fraud, and performing procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the financial statements. Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the financial statements. We believe that our audits provide a reasonable basis for our opinion.
Critical Audit Matter
The critical audit matter communicated below is a matter arising from the current period audit of the financial statements that was communicated or required to be communicated to the audit committee and that: (1) relates to accounts or disclosures that are material to the financial statements and (2) involved our especially challenging, subjective or complex judgments. The communication of the critical audit matter does not alter in any way our opinion on the
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consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.
Accrued Research and Development Expenses
Description of the Matter The Company’s accrued research and development expenses totaled $13.3 million at December 31, 2024, which includes accrued clinical trial expenses. As discussed in Note 2 to the consolidated financial statements, the Company expenses research and development costs as incurred. The Company accrues expenses related to clinical trials based on estimates of the services performed pursuant to their contractual arrangements, including those with clinical research organizations. Payments under these contracts may depend on factors such as the successful enrollment of patients and the completion of clinical milestones and may differ from the level of effort expended in each period.
How We Addressed the Matter in Our Audit Auditing the Company’s accrued research and development expenses was complex in the context of this audit due to the volume of research and development activity and the reliance on third-party vendors. That is, due to the timing and pattern of invoices received from third parties, actual amounts incurred were not always known as of the audit report date.
To test the accrued research and development expenses related to clinical trials, our audit procedures included, among others, obtaining an understanding of the Company’s process, testing the accuracy and completeness of the underlying data used in the estimates and evaluating and testing the significant assumptions that are used by management to estimate the accruals. To test the significant assumptions, we inspected the contracts and any amendments to the contracts with third-party service providers, and corroborated the progress of research and development projects with the Company’s research and development personnel that oversee the clinical trials. We also obtained evidence from the Company’s clinical research organization, including the number of patients, sites, purchase order amounts and total amounts invoiced to date. To further evaluate completeness, we tested invoices and payments to third parties subsequent to year-end.
/ s/ Ernst & Young LLP
We have served as the Company’s auditor since 2015.
Boston, Massachusetts
March 5, 2025
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Editas Medicine, Inc.
Consolidated Balance Sheets
(amounts in thousands, except share and per share data)
December 31,
2024 2023
ASSETS
Current assets:
Cash and cash equivalents $ 131,541 $ 123,652
Marketable securities 138,372 199,459
Accounts receivable 16,266 10,187
Prepaid expenses and other current assets 3,136 7,531
Total current assets 289,315 340,829
Marketable securities — 104,024
Property and equipment, net 14,497 12,032
Right-of-use assets 32,554 33,680
Restricted cash and other non-current assets 5,223 8,588
Total assets $ 341,589 $ 499,153
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 5,493 $ 8,269
Accrued expenses 45,859 34,563
Liability for sale of future revenues, current 5,000 —
Deferred revenue, current 6,221 8,221
Operating lease liabilities 14,652 12,164
Total current liabilities 77,225 63,217
Operating lease liabilities, net of current portion 20,380 24,372
Liability for sale of future revenues - non-current 52,434 —
Deferred revenue, net of current portion 54,204 60,667
Other non-current liabilities 3,072 1,800
Total liabilities 207,315 150,056
Stockholders’ equity
Preferred stock, $ 0.0001 par value per share: 5,000,000 shares authorized; no shares issued or outstanding
— —
Common stock, $ 0.0001 par value per share: 195,000,000 shares authorized; 82,734,696 and 81,767,263 shares issued, and 82,734,696 and 81,767,263 shares outstanding at December 31, 2024 and December 31, 2023, respectively
8 8
Additional paid-in capital 1,602,441 1,580,241
Accumulated other comprehensive income 268 198
Accumulated deficit ( 1,468,443 ) ( 1,231,350 )
Total stockholders’ equity 134,274 349,097
Total liabilities and stockholders’ equity $ 341,589 $ 499,153
The accompanying notes are an integral part of the consolidated financial statements.
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Editas Medicine, Inc.
Consolidated Statements of Operations
(amounts in thousands, except per share and share data)
Year Ended
December 31,
2024 2023 2022
Collaboration and other research and development revenues $ 32,314 $ 78,123 $ 19,712
Operating expenses:
Research and development 199,247 177,651 174,958
General and administrative 71,987 69,653 70,704
Restructuring charges 12,232 — —
Total operating expenses 283,466 247,304 245,662
Operating loss ( 251,152 ) ( 169,181 ) ( 225,950 )
Other income, net:
Other expense, net ( 3 ) ( 1,604 ) —
Interest income, net 14,062 17,566 5,518
Total other income, net 14,059 15,962 5,518
Net loss $ ( 237,093 ) $ ( 153,219 ) $ ( 220,432 )
Net loss per share, basic and diluted $ ( 2.88 ) $ ( 2.02 ) $ ( 3.21 )
Weighted-average common shares outstanding, basic and diluted 82,338,220 75,965,633 68,664,822
The accompanying notes are an integral part of the consolidated financial statements.
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Editas Medicine, Inc.
Consolidated Statements of Comprehensive Loss
(amounts in thousands)
Year Ended
December 31,
2024 2023 2022
Net loss $ ( 237,093 ) $ ( 153,219 ) $ ( 220,432 )
Other comprehensive income (loss):
Unrealized gain (loss) on marketable debt securities 70 3,799 ( 3,108 )
Comprehensive loss $ ( 237,023 ) $ ( 149,420 ) $ ( 223,540 )
The accompanying notes are an integral part of the consolidated financial statements.
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Editas Medicine, Inc.
Consolidated Statements of Stockholders’ Equity
(amounts in thousands except share data)
Common Stock Additional
Paid-In
Capital Accumulated
Deficit Accumulated
Other
Comprehensive
Income (Loss) Total
Stockholders’
Equity
Shares Amount
Balance at December 31, 2021 68,435,257 $ 7 $ 1,411,827 $ ( 857,699 ) $ ( 493 ) $ 553,642
Exercise of stock options 19,769 — 305 — — 305
Stock-based compensation expense — — 29,294 — — 29,294
Vesting of restricted common stock awards 286,642 — — — — —
Purchase of common stock under benefits plans 105,714 — 979 — — 979
Unrealized loss on marketable debt securities — — — — ( 3,108 ) ( 3,108 )
Net loss — — — ( 220,432 ) — ( 220,432 )
Balance at December 31, 2022 68,847,382 $ 7 $ 1,442,405 $ ( 1,078,131 ) $ ( 3,601 ) $ 360,680
Issuance of common stock from public offering, net commissions, underwriting discounts and offering costs 12,500,000 1 117,078 — — 117,079
Exercise of stock options 6,587 — 41 — — 41
Stock-based compensation expense — — 19,798 — — 19,798
Vesting of restricted common stock awards 297,934 — — — — —
Purchase of common stock under benefit plan 115,360 — 919 — — 919
Unrealized gain on marketable debt securities — — — — 3,799 3,799
Net loss — — — ( 153,219 ) — ( 153,219 )
Balance at December 31, 2023 81,767,263 $ 8 $ 1,580,241 $ ( 1,231,350 ) $ 198 $ 349,097
Exercise of stock options 23,809 — 193 — — 193
Stock-based compensation expense — — 21,417 — — 21,417
Vesting of restricted common stock awards 730,157 — — — — —
Purchase of common stock under benefit plans 213,467 — 590 — — 590
Unrealized gain on marketable debt securities — — — — 70 70
Net loss — — — ( 237,093 ) — ( 237,093 )
Balance at December 31, 2024 82,734,696 $ 8 $ 1,602,441 $ ( 1,468,443 ) $ 268 $ 134,274
The accompanying notes are an integral part of the consolidated financial statements.
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Editas Medicine, Inc.
Consolidated Statements of Cash Flows
(amounts in thousands)
Year Ended
December 31,
2024 2023 2022
Cash flow from operating activities
Net loss $ ( 237,093 ) $ ( 153,219 ) $ ( 220,432 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense 21,417 19,798 29,294
Depreciation 5,809 6,064 6,337
Net amortization of premiums and discounts on marketable securities ( 5,791 ) ( 4,823 ) ( 724 )
Loss on disposal of fixed assets 149 — —
Non-cash interest expense on liability for sale of future revenues 2,190 — —
Other non-cash items, net ( 415 ) 1,596 —
Changes in operating assets and liabilities:
Accounts receivable ( 6,079 ) ( 5,042 ) ( 4,878 )
Prepaid expenses and other current assets 4,396 ( 197 ) ( 137 )
Right-of-use assets 1,126 9,968 ( 17,475 )
Other non-current assets 3,366 ( 3,335 ) 1,558
Accounts payable ( 2,286 ) ( 1,492 ) 4,368
Accrued expenses 11,623 4,114 10,505
Deferred revenue ( 8,463 ) — ( 3,333 )
Operating lease liabilities ( 1,504 ) ( 7,410 ) 17,568
Other current and non-current liabilities 1,271 1,800 —
Net cash used in operating activities ( 210,284 ) ( 132,178 ) ( 177,349 )
Cash flow from investing activities
Purchases of property and equipment ( 8,826 ) ( 4,719 ) ( 4,118 )
Proceeds from the sale of equipment — — 18
Purchases of marketable securities ( 86,217 ) ( 258,517 ) ( 315,186 )
Proceeds from maturities of marketable securities 257,189 259,505 433,354
Net cash provided by (used in) investing activities 162,146 ( 3,731 ) 114,068
Cash flow from financing activities
Proceeds from offering of common stock, net of issuance costs — 117,079 —
Proceeds from sale of future revenues, net 55,244
Proceeds from exercise of stock options 193 41 305
Issuance of common stock under benefit plans 590 919 979
Net cash provided by financing activities 56,027 118,039 1,284
Net increase (decrease) in cash, cash equivalents, and restricted cash 7,889 ( 17,870 ) ( 61,997 )
Cash, cash equivalents, and restricted cash, beginning of period 127,529 145,399 207,396
Cash, cash equivalents, and restricted cash, end of period $ 135,418 $ 127,529 $ 145,399
Cash and cash equivalents, end of period 131,541 123,652 141,522
Restricted cash 1
3,877 3,877 3,877
Cash, cash equivalents, and restricted cash, end of period $ 135,418 $ 127,529 $ 145,399
1 As of December 31, 2024, December 31, 2023, and December 31, 2022 restricted cash of $ 3,877 , $ 3,877 , and $ 3,877 , respectively, was included in Restricted cash and other non-current assets on the Consolidated Balance Sheet
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Supplemental disclosure of cash and non-cash activities:
Fixed asset additions included in accounts payable and accrued expenses $ 65 $ 845 $ 1,440
Cash paid in connection with operating lease liabilities 18,447 12,428 14,851
Remeasurement of operating lease liabilities and right-of-use assets due to lease modification 794 2,752 24,545
Commencement of right-of-use asset 7,844 — 5,316
Modification of right-of-use asset 2,683 — —
Issuance costs associated with the sale of future revenues included in accounts payable and accrued expenses 1,479 — —
The accompanying notes are an integral part of the consolidated financial statements.
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Editas Medicine, Inc.
Notes to Consolidated Financial Statements
1. Nature of Business
Editas Medicine, Inc. (the “Company”) is an early stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The Company was incorporated in the state of Delaware in September 2013. Its principal offices are in Cambridge, Massachusetts.
Since its inception, the Company has devoted substantially all of its efforts to business planning, research and development, recruiting management and technical staff, and raising capital. The Company has primarily financed its operations through various equity financings, payments received under a research collaboration with Juno Therapeutics, Inc. (“Juno Therapeutics”), a wholly owned subsidiary of the Bristol-Myers Squibb Company (“BMS”), payments received under its former strategic alliance with Allergan Pharmaceuticals International Limited (together with its affiliates, “Allergan”), which was terminated in August 2020, payments received under our purchase and sale agreement with DRI Healthcare Acquisitions LP, and payments received in conjunction with the Company’s license agreement with Vertex Pharmaceuticals, Inc (“Vertex”, and such agreement, the “Vertex License Agreement”).
The Company is subject to risks common to companies in the biotechnology industry, including but not limited to, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for any drug product candidate that it may identify and develop, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary technology, compliance with government regulations, development by competitors of technological innovations and ability to transition from pilot-scale manufacturing to large-scale production of products.
Liquidity
As of December 31, 2024, the Company has raised an aggregate of $ 1.0 billion in net proceeds through the sale of shares of its common stock in public offerings and at-the-market offerings. The Company also has funded its business from payments received under our purchase and sale agreement with DRI Healthcare Acquisitions LP, payments received under its license agreement with Vertex, its research collaboration with BMS through its wholly owned subsidiary June Therapeutics and its former strategic alliance with Allergan (which was terminated in August 2020). As of December 31, 2024, the Company had cash, cash equivalents and marketable securities of $ 269.9 million.
In May 2021, the Company entered into a common stock sales agreement with TD Securities (USA) LLC (as successor to Cowen and Company, LLC) (“TD Cowen”), under which the Company from time to time can issue and sell shares of its common stock through TD Cowen in at-the-market offerings for aggregate gross sale proceeds of up to $ 300.0 million (the “Prior ATM Facility”). The Company amended the common stock sales agreement with TD Cowen in February 2024 in connection with filing a new registration statement. As of December 31, 2024 the Company has not sold any shares of its common stock under the Prior ATM Facility. In March 2025, the Company further amended the common stock sales agreement with TD Cowen in connection with amending its shelf registration statement following the loss of its status as a “well-known seasoned issuer” (as defined under Rule 405 of the Securities Act of 1933, as amended), lowering the amount of shares of common stock it may issue and sell through TD Cowen to aggregate gross sale proceeds of up to $ 150.0 million (the “ATM Facility”). As of December 31, 2024, the Company had not sold any shares of its common stock under the ATM Facility.
The Company has incurred annual net operating losses in every year since its inception. The Company has an accumulated deficit of $ 1.5 billion at December 31, 2024. As of March 5, 2025, the issuance date of the consolidated financial statements, the Company expects that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements for at least twelve months from the issuance date of the consolidate financial statements. The Company has never generated any product revenue. There can be no assurance that the Company will be able to obtain additional debt or equity financing or generate product revenue or revenues from collaborative partners, on terms acceptable to the Company, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition.
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2. Summary of Significant Accounting Policies
Principles of Consolidation
The accompanying consolidated financial statements include the accounts of Editas Medicine, Inc. and its wholly owned subsidiaries, Editas Securities Corporation, which is a Delaware subsidiary created to buy, sell and hold securities, and Editas Medicine, LLC, a Delaware limited liability company. All intercompany transactions and balances have been eliminated.
Basis of Presentation
The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United States generally accepted accounting principles as found in the ASC and Accounting Standards Updates (“ASU”) of the FASB.
Use of Estimates
The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, stock-based compensation expense, the accrual for research and development expenses, valuations of in-process research and development assets, effective interest rates and deferred tax valuation allowances. The Company bases its estimates on historical experience and other market-specific or relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions.
Fair Value of Financial Instruments
ASC Topic 820, Fair Value Measurement (“ASC 820”), establishes a fair value hierarchy for instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and the Company’s own assumptions (unobservable inputs). Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability, and are developed based on the best information available in the circumstances.
ASC 820 identifies fair value as the exchange price representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants. As a basis for considering market participant assumptions in fair value measurements, ASC 820 establishes a three-tier fair value hierarchy that distinguishes between the following:
• Level 1 – Quoted market prices in active markets for identical assets or liabilities.
• Level 2 – Inputs other than Level 1 inputs that are either directly or indirectly observable, such as quoted market prices, interest rates, and yield curves.
• Level 3 – Unobservable inputs developed using estimates of assumptions developed by the Company, which reflect those that a market participant would use.
To the extent that the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.
The carrying amounts reflected in the consolidated balance sheets for cash and cash equivalents, restricted cash, marketable securities, accounts receivable, prepaid expenses and other current assets, accounts payable, accrued expenses, and other current liabilities approximate their fair values, due to their short-term nature.
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Cash, Cash Equivalents, and Restricted Cash
The Company considers all highly liquid investments purchased with original maturities of 90 days or less at acquisition to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in money market funds.
The Company has restricted cash of $ 3.9 million held as collateral for the Company’s office and lab facilities and credit card program. The restricted funds are maintained in a traditional bank account.
The following table presents cash, cash equivalents, and restricted cash as reported on the consolidated balance sheets that equal the total amounts on the consolidated statements of cash flows (in thousands):
Year Ended
December 31,
2024 2023
Cash and cash equivalents $ 131,541 $ 123,652
Restricted cash included in "Restricted cash and other non-current assets"
3,877 3,877
Total cash, cash equivalents, and restricted cash $ 135,418 $ 127,529
Marketable Securities
The Company classifies marketable securities with a remaining maturity when purchased of greater than three months and less than one year from the balance sheet date as current. Marketable securities are classified as long-term assets on the consolidated balance sheets if the contractual maturity exceeds one year and the Company does not intend to utilize the marketable securities to fund current operations. For the years ended December 31, 2024 and 2023, the Company’s marketable securities consisted of investments in available-for-sale debt securities.
Available-for-sale debt securities are carried at fair value with the unrealized gains and losses included in accumulated other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense over the life of the of the underlying security. Realized gains and losses are included in other income (expense).
At each reporting date, the Company records an allowance for credit losses and reports it as credit loss expense which is included in “Other income (expense), net” in the Company’s consolidated statement of operations. The estimate for credit losses includes a measure of the expected risk of credit loss even if the risk is remote. When assessing financial assets for credit losses, the Company pools financial assets with similar risk characteristics and performs a collective evaluation. A credit loss on an available-for-sale debt security is limited to the difference in fair value and the amortized cost. A previously recognized credit loss may be increased or decreased in subsequent periods if the Company’s estimate of fair value changes. To determine whether to record a credit loss, the Company considers issuer or vendor specific credit ratings and historical losses as well as current economic conditions and its expectations for future economic conditions. To date, the Company has not had any credit losses, and the Company did not have an allowance for credit losses as of December 31, 2024 and 2023.
Accounts Receivable
The Company makes judgments as to its ability to collect outstanding receivables and provides an allowance for receivables when collection becomes doubtful. The Company's receivables primarily relate to amounts reimbursed under its collaboration agreements. The Company believes that credit risk associated with its collaborations partners is not significant. To date, the Company has not had any write-offs of bad debt, and the Company did not have an allowance for doubtful accounts as of December 31, 2024 and 2023.
Concentrations of Credit Risk and Off-Balance Sheet Risk
The Company has no financial instruments with off-balance sheet risk such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. Financial instruments that potentially subject the Company to a concentration of credit risk are cash, cash equivalents, marketable securities and receivables owed to the Company from collaboration partners. The Company’s cash, cash equivalents and marketable securities are held in accounts at a financial
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institution that may exceed federally insured limits. The Company has not experienced any credit losses in such accounts and does not believe it is exposed to any significant credit risk on these funds.
Property and Equipment
Property and equipment consists of computers, laboratory equipment, furniture and office equipment, and leasehold improvements and is stated at cost, less accumulated depreciation. Maintenance and repairs that do not improve or extend the lives of the respective assets are expensed to operations as incurred, while costs of major additions and betterments are capitalized. Depreciation is calculated over the estimated useful lives of the assets using the straight-line method. The Company capitalizes laboratory equipment used for research and development if it has alternative future use in research and development or otherwise.
Asset: Estimated Useful life
Laboratory and manufacturing equipment 5 years
Computer equipment and software 3 years
Furniture and equipment 5 years
Leasehold improvements Shorter of useful life or remaining lease term
Impairment of Long-lived Assets
The Company evaluates long-lived assets for potential impairment when events or changes in circumstances indicate the carrying value of the assets may not be recoverable. Recoverability is measured by comparing the book values of the assets to the expected future net undiscounted cash flows that the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the book values of the assets exceed their fair value. The Company has not recognized any impairment losses from inception through December 31, 2024.
Revenue Recognition
The Company recognizes revenue in accordance with ASC Topic 606, Revenue from Contracts with Customers (“ASC 606”). The Company enters into collaboration agreements and certain other agreements that are within the scope of ASC 606, under which the Company licenses, may license or grants an option to license rights to certain of the Company’s product candidates and performs research and development services in connection with such arrangements. The terms of these arrangements typically include payment of one or more of the following: non-refundable, up-front fees; reimbursement of research and development costs; development, clinical, regulatory and commercial sales milestone payments; and royalties on net sales of licensed products.
Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for arrangements determined to be within the scope of ASC 606, the Company performs the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect consideration it is entitled to in exchange for the goods or services it transfers to the customer.
The promised goods or services in the Company’s arrangements typically consist of a license, or option to license, rights to the Company’s intellectual property or research and development services. The Company provides options to additional items in such arrangements, which are accounted for as separate contracts when the customer elects to exercise such options, unless the option provides a material right to the customer. Performance obligations are promised goods or services in a contract to transfer a distinct good or service to the customer and are considered distinct when (i) the customer can benefit from the good or service on its own or together with other readily available resources and (ii) the promised good or service is separately identifiable from other promises in the contract. In assessing whether promised good or services are distinct, the Company considers factors such as the stage of development of the underlying intellectual
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property, the capabilities of the customer to develop the intellectual property on its own or whether the required expertise is readily available and whether the goods or services are integral or dependent to other goods or services in the contract.
The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each arrangement that includes variable consideration, the Company evaluates the amount of potential payment and the likelihood that the payments will be received. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period.
The Company’s contracts often include development and regulatory milestone payments that are as assessed under the most likely amount method and constrained if it is probable that a significant revenue reversal would occur. Milestone payments that are not within the Company’s control or the licensee’s control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. At the end of each reporting period, the Company re-evaluates the probability of achievement of such development and clinical milestones and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration and other research and development revenues in the period of adjustment.
For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from any of the Company’s collaboration or strategic alliance arrangements.
The Company allocates the transaction price based on the estimated standalone selling price. The Company must develop assumptions that require judgment to determine the stand-alone selling price for each performance obligation identified in the contract. The Company utilizes key assumptions to determine the stand-alone selling price, which may include other comparable transactions, pricing considered in negotiating the transaction and the estimated costs. Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts the Company would expect to receive for the satisfaction of each performance obligation.
The consideration allocated to each performance obligation is recognized as revenue when control is transferred for the related goods or services. For performance obligations which consist of licenses and other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition.
The Company receives payments from its customers based on billing schedules established in each contract. Up-front payments and fees are recorded as deferred revenue upon receipt or when due until the Company performs its obligations under these arrangements. Amounts are recorded as accounts receivable when the Company’s right to consideration is unconditional.
Research and Development Expenses
Research and development expenses are charged to expense as incurred in performing research and development activities. The costs include employee-related expenses including salaries, benefits, and stock-based compensation expense, costs of funding research performed by third parties that conduct research and development and preclinical and clinical activities on the Company’s behalf, the cost of purchasing lab supplies and non-capital equipment used in preclinical and clinical activities and in manufacturing preclinical and clinical study materials, consultant fees, facility costs including rent, depreciation, and maintenance expenses, and fees for acquiring and maintaining licenses under third party licensing agreements which are typically expensed when incurred if the technology licensed has no alternate future uses, including any sublicensing or success payments made to the Company’s licensors. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the Company’s estimate, the accrual or prepaid is
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adjusted accordingly. The Company defers and capitalizes non-refundable advance payments made by the Company for research and development activities until the related goods are received or the related services are performed. In circumstances where amounts have been paid in excess of costs incurred, the Company records a prepaid expense.
In-process Research and Development Assets
In-process research and development assets that are acquired in a transaction that does not qualify as a business combination under GAAP and that do not have an alternative future use are expensed in the period in which the assets are acquired.
Patent Costs
The Company expenses patent and patent application costs and related legal costs for the prosecution and maintenance of such patents and patent applications, including patents and patent applications the Company in-licenses, as incurred, and classifies such costs as general and administrative expenses in the accompanying consolidated statements of operations.
Liability for Sale of Future Revenues
On October 3, 2024, the Company entered into a purchase and sale agreement (the “Purchase and Sale Agreement”) with DRI Healthcare Acquisitions LP (“DRI”) under which it sold, transferred, assigned, and conveyed to DRI certain future license fees and other payments (the “Purchased Receivables”) owed to the Company by Vertex under the terms of the Vertex License Agreement in exchange for an upfront cash payment by DRI to the Company of $ 57.0 million. Under the Purchase and Sale Agreement, DRI is purchasing up to 100% of certain future fixed and sales-based annual license fees owed to the Company under the Vertex License Agreement, which fees range from $ 5.0 million to $ 40.0 million per year (inclusive of certain sales-based annual license fee increases) and a mid-double-digit percentage of a $ 50.0 million contingent upfront payment that the Company may receive under the Vertex License Agreement, in each case after subtracting amounts owing by the Company to Broad and Harvard (each term as defined in Note 8, respectively). The Company has retained rights to certain portions of certain sales-based annual license fees and the contingent upfront payment that may become due under the Vertex License Agreement, and the amounts that correspond to its licensor obligations.
In accordance with ASC Topic 470, Borrower’s Accounting for Debt Modification , the Company has accounted for the transaction as debt. The gross proceeds of $ 57.0 million were recorded as a liability for the sale of future revenues, net of transaction costs of $ 1.8 million, which will be amortized over the estimated life of the arrangement using the effective interest method.
The Company estimates the effective interest rate used to record non-cash interest expense under the Purchase and Sale Agreement based on the estimate of future revenue payments to be made to DRI. As of December 31, 2024, the estimated effective interest rate under the agreement was 15.7 %. Over the life of the arrangement, the actual effective interest rate will be affected by the amount and timing of the payments made to DRI and changes in the Company's revenue forecasts. At each reporting date, the Company will reassess its estimate of total future payments to be made to DRI, and prospectively adjust the effective interest rate and amortization of the liability as necessary. Refer to Note 16 for further details.
Leases
The Company accounts for leases in accordance with ASC Topic 842, Leases . At the inception of an arrangement the Company determines whether the arrangement contains a lease. If a lease is identified in an arrangement, the Company recognizes a right-of-use asset and liability on its balance sheet and determines whether the lease should be classified as a finance or operating lease. The Company does not recognize assets or liabilities for leases with lease terms of less than 12 months. Lease payments for short-term leases are recorded to operating expense on a straight-line basis over the lease term and variable lease payments are recorded in the period in which the obligation for those payments is incurred.
A lease qualifies as a finance lease if any of the following criteria are met at the inception of the lease: (i) there is a transfer of ownership of the leased asset to the Company by the end of the lease term, (ii) the Company holds an option to purchase the leased asset that it is reasonably certain to exercise, (iii) the lease term is for a major part of the remaining economic life of the leased asset, (iv) the present value of the sum of lease payments equals or exceeds substantially all of
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the fair value of the leased asset, and (v) the nature of the leased asset is specialized to the point that it is expected to provide the lessor no alternative use at the end of the lease term. All other leases are recorded as operating leases.
Finance and operating lease assets and liabilities are recognized at the lease commencement date based on the present value of the lease payments over the lease term using the discount rate implicit in the lease. If the rate is not readily determinable, the Company utilizes its incremental borrowing rate at the lease commencement date. Operating lease assets are further adjusted for prepaid or accrued lease payments. Operating lease payments are expensed using the straight-line method as an operating expense over the lease term. Finance lease assets are amortized to depreciation expense using the straight-line method over the shorter of the useful life of the related asset or the lease term. Finance lease payments are bifurcated into (i) a portion that is recorded as imputed interest expense and (ii) a portion that reduces the finance liability associated with the lease.
The Company does not separate lease and non-lease components when determining which lease payments to include in the calculation of its lease assets and liabilities. Variable lease payments are expensed as incurred. If a lease includes an option to extend or terminate the lease, the Company reflects the option in the lease term if it is reasonably certain it will exercise the option.
Stock-based Compensation Expense
The Company accounts for all stock-based awards granted to employees and non-employees as stock-based compensation expense at fair value in accordance with FASB ASC Topic 718 Compensation—Stock Compensation (“ASC 718”). The Company estimates the grant date fair value of restricted stock based on the market value of the Company’s common stock on the date of the grant. The Company estimates the grant date fair value of each option award using the Black-Scholes option-pricing model.
The Black-Scholes option pricing model requires the input of certain assumptions, including (1) the expected stock price volatility, (2) the calculation of expected term of the award, (3) the risk-free interest rate, and (4) the expected dividend yield. The Company used its own historical volatility data for its estimates of expected volatility. The Company calculates historical volatility based on a period of time commensurate with the expected term. The Company uses the simplified method as prescribed by the Securities and Exchange Commission’s Staff Accounting Bulletin No. 107, Share-Based Payment, to calculate the expected term for options granted to employees as the Company does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected term. For options granted to non-employees, the Company utilizes the contractual term of the arrangement as the basis for the expected term. The Company determines the risk-free interest rate based on a treasury instrument whose term is consistent with the expected term of the stock options. The Company uses an assumed dividend yield of zero as the Company has never paid dividends and does not have current plans to pay any dividends on its common stock.
Restricted stock awards (“RSAs”) are subject to repurchase rights. Accordingly, the Company has recorded the proceeds from the issuance of RSAs as a liability in the consolidated balance sheets. The restricted stock liability is reclassified into stockholders’ equity as the restricted stock vests.
Service-Based Awards
For stock-based awards issued to employees, non-employee service providers and members of the Company’s board of directors (the “Board”), the Company recognizes the grant date fair value of the service-based options, RSAs or restricted stock unit awards (“RSUs”) on a straight-line basis over the requisite service period, which is generally the vesting period of the respective award. If an employee or non-employee service requirement is concluded to be non-substantive, the stock-based compensation expense would be expensed immediately.
Market-Based Awards
For market-based awards, the Company recognizes the grant date fair value of the market-based options over the earlier of the derived service period, pursuant to a Monte-Carlo simulation model, or when the market-based vesting conditions are met. The Company estimates an award's derived service period based on the best estimate of the period over which an award's vesting condition(s) will be achieved. If the market-based vesting conditions are met ahead of the derived service period, the expense will be accelerated. If the market-based vesting conditions are not met and the market-based award is cancelled, the expense will not be reversed unless the market-based award is forfeited.
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Performance-Based Awards
For performance-based awards, the Company recognizes the grant date fair value of the performance-based options or RSUs over the requisite service period using the accelerated attribution method to the extent achievement of the performance condition is probable. Certain awards are subject to both performance and continued service conditions.
The Company classifies stock-based compensation expense in its consolidated statement of operations and comprehensive loss in the same manner in which the award recipient’s salary or service payments are classified. Forfeitures are recorded as they occur. If factors change or different assumptions are used, the Company’s stock-based compensation expense could be materially different in the future.
Success Payments, Research Funding Payments and Notes Payables
Certain arrangements require the Company to make payments, if and when, the Company’s market capitalization reaches specified thresholds for a specific period of time or upon a sale of the Company for consideration in excess of those thresholds or above a specific amount. The payments are accounted for under the provisions of ASC 718, whereby the Company recognizes the expense and liability when it becomes probable that the amounts will become due. The Company records this expense as a research and development expense in its consolidated statements of operations. The arrangements and payments are described more fully in Note 8.
The payments are payable in either cash, common stock or promissory notes payable, depending upon the licensor and the Company’s election. If the Company elects to issue a promissory note relating to contractual obligations, the promissory note bears interest at 4.8 % per annum. Outstanding principal and accrued interest on the promissory notes are typically payable on the earlier of five months or a specified period of time following a Company sale or change of control event, subject to certain exceptions.
Income taxes
Income taxes are recorded in accordance with ASC Topic 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and the tax reporting basis of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. The Company provides a valuation allowance against net deferred tax assets unless, based upon the weight of available evidence, it is more likely than not that the deferred tax assets will be realized.
The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances.
The Company assesses the impact of various tax reform proposals and modifications to existing tax treaties in all jurisdictions where they have operations to determine the potential effect on the Company’s business and any assumptions they have made about their future taxable income. The Company cannot predict whether any specific proposals will be enacted, the terms of any such proposals or what effect, if any, such proposals would have on the Company if they were to be enacted.
Comprehensive Loss
Comprehensive loss currently consists of net loss and changes in unrealized gains and losses on marketable securities.
Segment Information
Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or CODM, in deciding how to allocate resources and assess performance. The Company and the Company’s CODM, the Company’s Chief Executive Officer, or CEO, view the Company’s operations and manage the Company’s business as a single operating segment, which is the business of developing and commercializing genome editing technology.
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Recently Adopted Accounting Pronouncements
In November 2023, the FASB issues ASU 2023-07 Segment Reporting - Improvements to Reportable Segment Disclosures which amends Topic 280 by enhancing segment reporting by requiring more detailed expense information for each reportable segment. Under the guidance, public entities are required to disclose (1) significant segment expense categories and amounts as those regularly provided to the CODM for each reportable segment and how the CODM uses the reported measures of a segment’s profit or loss to assess segment performance and decide how to allocate resources; (2) the amount and composition of other segment items included in reported segment profit or loss, and (3) the CODM’s position and title. Additionally, multiple measures of a segment’s profit or loss may be reported, under certain conditions, and single reportable segment entities must apply Topic 280 in its entirety. Refer to Note 18 for additional information on the Company’s segment reporting.
The total of the reportable segment’s amount for the measure of profit or loss is to be reconciled to the public entity’s consolidated income before income taxes and discontinued operations. The ASU is effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024, with early adoption permitted. The Company adopted the ASU as of and for the period ended December 31, 2024.
Recently Issued Accounting Pronouncements
In November 2024, the FASB issued ASU 2024-03 Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses , which is intended to improve the disclosure of expenses by providing more detailed information about the types of expenses in commonly presented expense captions. The amendments in this ASU are effective for annual reporting periods beginning after December 15, 2026, and interim periods beginning after December 15, 2027. Early adoption is permitted. The amendments can be applied either prospectively or retrospectively. The Company has not early adopted this ASU and is currently evaluating the impact of this new standard on its consolidated financial statements and related disclosures.
In December 2023, the FASB issued ASU 2023-09 Income Taxes (Topic 740): Improvements to Income Tax Disclosures . This guidance is intended to enhance the transparency and decision-usefulness of income tax disclosures. The amendments in ASU 2023-09 address investor requests for enhanced income tax information primarily through changes to disclosure regarding rate reconciliation and income taxes paid both in the United States and in foreign jurisdictions. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024 on a prospective basis, with the option to apply the standard retrospectively. Early adoption is permitted. The Company is currently evaluating this guidance to determine the impact it may have on its consolidated financial statement disclosures.
In October 2023, the FASB issued ASU 2023-06 Disclosure Improvements: Codification Amendments in Response to the SEC's Disclosure Update and Simplification Initiative, which incorporates certain SEC disclosure requirements into the ASC. The amendments in the ASU are expected to clarify or improve disclosure and presentation requirements of a variety ASC topics, allow investors to more easily compare entities subject to the SEC's existing disclosures with those entities that were not previously subject to the requirements, and align the requirements in the ASC with the SEC's regulations. The effective date for each amendment will be the date on which the SEC's removal of that related disclosure from Regulation S-X or Regulation S-K becomes effective, with early adoption prohibited. The amendments in this ASU should be applied prospectively. The Company does not expect ASU 2023-06 will have a material impact to its consolidated financial statements.
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3. Cash Equivalents and Marketable Securities
Cash equivalents and marketable securities consisted of the following at December 31, 2024 (in thousands):
December 31, 2024 Amortized
Cost Allowance
for Credit
Losses Gross
Unrealized
Gains Gross
Unrealized
Losses Fair
Value
Cash equivalents and marketable securities:
Money market funds $ 131,541 $ — $ — $ — $ 131,541
U.S. Treasuries 131,582 — 289 131,871
Government agency securities — — — — —
Corporate notes/bonds 6,522 — — ( 21 ) 6,501
Total $ 269,645 $ — $ 289 $ ( 21 ) $ 269,913
Cash equivalents and marketable securities consisted of the following at December 31, 2023 (in thousands):
December 31, 2023 Amortized
Cost Allowance
for Credit
Losses Gross
Unrealized
Gains Gross
Unrealized
Losses Fair
Value
Cash equivalents and marketable securities:
Money market funds $ 123,652 $ — $ — $ — $ 123,652
U.S. Treasuries 168,858 — 611 169,469
Government agency securities 103,507 — — ( 327 ) 103,180
Corporate notes/bonds 30,920 — — ( 86 ) 30,834
Total $ 426,937 $ — $ 611 $ ( 413 ) $ 427,135
The Company's available-for-sale securities are subject to a periodic impairment review. A debt security is considered to be impaired when its fair value is less than its carrying cost, in which case the Company would further review the investment to determine whether it is other-than-temporarily impaired. When evaluating an investment for other-than-temporary impairment, the Company reviews factors such as the length of time and extent to which fair value has been below cost basis, the financial condition of the issuer and any changes thereto, intent to sell, and whether it is more likely than not we will be required to sell the investment before the recovery of its cost basis. If an investment is other-than-temporarily impaired or subject to credit losses, the Company writes it down through the statement of operations and comprehensive loss to its fair value and establishes that value as a new cost basis for the investment.
We determined that none of our available-for-sale securities were other-than-temporarily impaired as of December 31, 2024 and 2023, and we did not hold any marketable securities that had been in an unrealized loss position for more than twelve months. As such, the Company believes that it is more likely than not that investments will be held until maturity or a forecasted recovery of fair value.
Based on our procedures under the expected credit loss model, including an assessment of unrealized losses in our portfolio, the Company concluded that any unrealized losses on marketable securities were not attributable to credit and, therefore, did not record an allowance for credit losses for these securities as of December 31, 2024 and 2023. As of December 31, 2024, the Company holds no securities with remaining maturities greater than one year .
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4. Fair Value Measurements
Assets measured at fair value on a recurring basis as of December 31, 2024 were as follows (in thousands):
Financial Assets December 31,
2024 Quoted Prices
in Active
Markets for
Identical Assets
(Level 1) Significant
Other
Observable
Inputs
(Level 2) Significant
Unobservable
Inputs
(Level 3)
Cash equivalents:
Money market funds $ 131,541 $ 131,541 $ — $ —
Marketable securities:
Corporate bonds 6,501 — 6,501 —
U.S. Treasuries 131,871 131,871 — —
Restricted cash and other non-current assets:
Money market funds 3,877 3,877 — —
Total financial assets $ 273,790 $ 267,289 $ 6,501 $ —
Assets measured at fair value on a recurring basis as of December 31, 2023 were as follows (in thousands):
Financial Assets December 31,
2023 Quoted Prices
in Active
Markets for
Identical Assets
(Level 1) Significant
Other
Observable
Inputs
(Level 2) Significant
Unobservable
Inputs
(Level 3)
Cash equivalents:
Money market funds $ 123,652 $ 123,652 $ — $ —
Marketable securities:
Government agency securities 103,180 — 103,180 —
Corporate bonds 30,834 — 30,834 —
U.S. Treasuries 169,469 169,469 — —
Restricted cash and other non-current assets:
Money market funds 3,877 3,877 — —
Total financial assets $ 431,012 $ 296,998 $ 134,014 $ —
5. Property and Equipment, Net
Property and equipment, net consisted of the following (in thousands):
As of
December 31,
2024 December 31,
2023
Laboratory and manufacturing equipment $ 29,128 $ 25,043
Leasehold improvements 11,749 9,648
Construction-in-progress 827 2,060
Computer equipment 1,490 1,062
Furniture and office equipment 264 264
Software 2,687 215
Total property and equipment 46,145 38,292
Less: accumulated depreciation ( 31,648 ) ( 26,260 )
Property and equipment, net $ 14,497 $ 12,032
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The Company recorded $ 5.8 million, $ 6.1 million, and $ 6.3 million in depreciation expense during the years ended December 31, 2024, 2023 and 2022, respectively.
6. Accrued Expenses
Accrued expenses consisted of the following (in thousands):
As of
December 31,
2024 2023
External research and development expenses $ 11,630 $ 16,204
Employee related expenses 11,516 11,280
Sublicense and license fees 6,609 5,063
Intellectual property and patent related fees 1,471 983
Professional service expenses 1,593 750
Employee termination benefits 10,475 —
Contract termination costs 1,757 —
Other expenses 808 283
Total accrued expenses $ 45,859 $ 34,563
7. Leases
The Company has multiple lease agreements for office, laboratory and manufacturing space with varying contractual terms set to expire between 2025 and 2028. Typically, base rent payments commence at the beginning of each lease term and continue through the term of the respective lease. The Company’s lease agreements have escalating rent clauses, which require higher rent payments in future years. The Company has two significant leases for office and laboratory space located in Cambridge, Massachusetts, and one significant lease for manufacturing space in Devens, Massachusetts.
The Company’s leases are included on its consolidated balance sheet as follows (in thousands):
As of
December 31,
2024 December 31,
2023
Right-of-use assets $ 32,554 $ 33,680
Operating lease liabilities, current $ ( 14,652 ) $ ( 12,164 )
Operating lease liabilities, noncurrent $ ( 20,380 ) $ ( 24,372 )
During the years ended December 31, 2024, 2023 and 2022, the Company recorded $ 21.0 million, $ 15.2 million and $ 13.6 million of expense related to operating lease costs and $ 2.8 million, $ 3.3 million and $ 3.0 million related to variable costs associated with the Company’s operating leases.
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Maturities of the Company’s lease liabilities as of December 31, 2024 were as follows (in thousands):
Maturity of lease liabilities: Year Ended
December 31, 2024
2025 $ 17,143
2026 $ 9,728
2027 $ 7,076
2028 $ 6,000
Total minimum lease payments $ 39,947
Less: imputed interest $ ( 4,915 )
Total operating lease liabilities at December 31, 2024 $ 35,032
The weighted-average remaining lease term is 2.9 years and the weighted-average discount rate is 9.1 %.
8. Commitments and Contingencies
The Company is a party to a number of license agreements under which the Company licenses patents, patent applications and other intellectual property from third parties. As such, the Company is obligated to reimburse licensors for various costs including upfront licenses fees, annual license fees, certain licensor expense reimbursements, success payments, research funding payments, and milestones triggerable upon certain development, regulatory, and commercial events as well as royalties on future products. These contracts are generally cancellable, with notice, at the Company’s option and do not have significant cancellation penalties.
Broad Sponsored Research Agreement
In 2018, the Company entered into a sponsored research agreement, which was amended in January 2021 (as amended, the “Sponsored Research Agreement”) with The Broad Institute, Inc. (“Broad”). The Sponsored Research Agreement provides for Broad to conduct research useful or relevant to genome editing in the field of genomic medicines for the prevention or treatment of human disease with funding from the Company. Under the Sponsored Research Agreement, Broad granted to the Company an exclusive right of first negotiation for licenses from Broad with respect to patentable inventions developed by Broad in the course of the sponsored research, subject to certain limitations and retained rights (“Sponsored Invention Licenses”).
Under the Sponsored Research Agreement, the Company is obligated to make payments (“Market Cap Research Funding”) in the event the Company’s market capitalization reaches certain amounts for a specified period of time. Unless the Company has undergone a change in control, Market Cap Research Funding is payable by the Company in cash, in shares of common stock, or in the form of promissory notes, which may be settled in shares of common stock at the election of the Company. In aggregate, the Company has triggered $ 25.0 million in Market Cap Research Funding and has primarily settled these amounts through the issuance of shares of its common stock. The remaining $ 100.0 million in Market Cap Research Funding may be triggered when the Company’s market capitalization reaches various low- ten to eleven figure dollar amounts or in the event of a Company sale. The Company is not required to make additional Market Cap Research Funding payments if the Company, whether directly or through its affiliates or sublicensees, is not researching, developing, or commercializing products based on or incorporating inventions exclusively licensed to the Company from Broad subject to certain exclusions.
The Sponsored Research Agreement is terminable by each party upon the occurrence of specified bankruptcy events of the other party and otherwise will continue in effect until the remaining Market Cap Research Funding payments are received by Broad and such time as the Company has no further rights of first negotiation for Sponsored Invention Licenses, unless otherwise mutually agreed between the parties.
Broad & Harvard License Agreements
The Company has entered into agreements with Broad and the President and Fellows of Harvard College (“Harvard”) to license certain patent rights owned or co-owned by the institutions. The foundational patent rights that were in-licensed by the Company include Cas9-I (as amended and restated to date, the “Cas9-I License Agreement”), Cas12a
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(formerly known as Cpf1) (as amended, the “Cpf1 License Agreement”), and Cas9-II (as amended, the “Cas9-II License Agreement”) (collectively referred to herein as the “License Agreements”). The Company received exclusive, worldwide, royalty-bearing, sublicensable licenses to certain patent rights to develop and commercialize licensed product and a non-exclusive, worldwide, royalty-bearing sublicensable license under the same patent rights for all other purposes, subject to certain limitations and retained rights. The Company is obligated to use commercially reasonable efforts to research, develop, and commercialize licensed products. The Company is also required to achieve certain development milestones within specified time periods for products covered by the License Agreements, with Broad or Harvard, as applicable, having the right to terminate the License Agreements, on a license agreement-by-license agreement basis, if the Company fails to achieve these milestones within the required time periods. Broad or Harvard may grant licenses under specified circumstances to third parties that wish to develop and commercialize products that target a particular gene that otherwise would fall within the scope of the exclusive licenses granted to the Company, provided that the Company is not, directly or through any of its affiliates, sublicensees, or collaborators, researching, developing, or commercializing a product directed toward the same gene target, or can demonstrate to Broad’s and/or Harvard’s, as applicable, reasonable satisfaction that the Company is interested in researching, developing, and commercializing a product directed toward the same gene target, that the Company has a commercially reasonable research, development, and commercialization plan to do so, and the Company commences and continues reasonable commercial efforts under such plan . The Company has the right to terminate each of the License Agreements at will with four months written notice to Broad. Unless terminated earlier, the term of each of the License Agreements will expire on a country-by-country basis, upon the expiration of the last to expire valid claim of the licensed patent rights in such country.
Milestones
In aggregate, the Company may pay up to $ 14.8 million, $ 20.0 million, and $ 3.7 million in clinical and regulatory milestones under the Cas9-I License Agreement, Cpf1 License Agreement, and Cas9-II License Agreement, respectively. In addition, the Company owes aggregate sales milestones totaling $ 54.0 million, $ 54.0 million, and $ 13.5 million under the Cas9-I License Agreement, Cpf1 License Agreement, and Cas9-II License Agreement, respectively. If the licensed product or service prevents or treats a human disease that afflicts fewer than a specified number of patients in the aggregate in the U.S. or a specified number of patients per year in the U.S., the clinical and regulatory milestones reduce to $ 4.1 million, $ 5.5 million, and $ 1.1 million under the Cas9-I License Agreement, Cpf1 License Agreement, and Cas9-II License Agreement, respectively. In addition, the aggregated sales milestones reduce to $ 36.0 million, $ 36.0 million, and $ 9.0 million under the Cas9-I License Agreement, Cpf1 License Agreement, and Cas9-II License Agreement, respectively. Certain clinical and regulatory milestones are subject to a multiplier payout equivalent to a double-digit percentage in the event of a change of control.
Royalties
The Company is required to pay on a product-by-product and country-by-country basis, a mid single-digit percentage royalty on net sales of licensed products made by the Company, its affiliates, or its sublicensees. The royalty percentage depends on the product and service, and whether such licensed product or licensed service is covered by a valid claim. If the Company is legally required to pay royalties to a third party on net sales of the Company’s products because such third party holds patent rights that cover such licensed product, then the Company can credit up to a specified percentage of the amount paid to such third party against the royalties due to the institutions. Such credit may not exceed 50 % of the applicable royalties paid by the Company to the applicable third party. The Company’s obligation to pay royalties will expire on a product-by-product and country-by-country basis upon the later of the expiration of the last to expire valid claim of the patent rights that covers each licensed product or service in each country or the tenth anniversary of the date of the first commercial sale of the licensed product or licensed service.
Licensor Expense Reimbursements
The Company is obligated to reimburse to Broad and Harvard for expenses incurred by each of them associated with the prosecution and maintenance of the patent rights that the Company licenses from them pursuant to the license agreement by and among the Company, Broad and Harvard, including the interference and opposition proceedings involving patents licensed to the Company under the license agreement, and other license agreements between the Company and Broad. As such, the Company anticipates that it has a substantial commitment in connection with these proceedings until such time as these proceedings have been resolved, but the amount of such commitment is not determinable. The Company incurred an aggregate of $ 8.2 million, $ 7.5 million, and $ 8.8 million in expense during the years ended December 31, 2024, 2023 and 2022, respectively, for such reimbursement.
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Success Payments
Under the Cpf1 License Agreement and Cas9-II License Agreement, the Company is obligated to make payments (“Success Payments”) in the event the Company’s market capitalization reaches certain thresholds for a specified period of time, or in the event of a change in control of the Company, if the consideration is in excess of those thresholds. Unless the Company has undergone a change in control, Success Payments are payable by the Company in cash, shares of the Company’s common stock, or in the form of promissory notes, which may be settled in shares of common stock at the election of the Company. In the event of a change in control of the Company, the Success Payments are required to be paid in cash. The Success Payments under the Cpf1 License Agreement are triggered when the Company’s market capitalization reaches certain amounts ranging from $ 750.0 million to $ 10.0 billion for a specified period of time. The Success Payments under the Cas9-II License Agreement are triggered when the Company’s market capitalization reaches certain amounts ranging from $ 1.0 billion to $ 9.0 billion for a specified period of time. In aggregate, the Company has triggered $ 25.0 million and $ 7.5 million of Success Payments under the Cpf1 License Agreement and Cas9-II License Agreement, respectively. The Company has primarily settled these amounts through the issuance of shares of its common stock.
The remaining $ 100.0 million and $ 22.5 million in Success Payments under the Cpf1 License Agreement and Cas9-II License Agreement, respectively are only payable if the market capitalization threshold are met and the Company or any affiliate or sublicensee has at least one product candidate covered by a claim of a patent right licensed to the Company that is or was subject of a clinical trial.
Other Payments
The Company pays nominal annual license fees to the institutions. If the Company sublicenses any of the patent rights to a third party, the institutions have the right to receive sublicense income, which may be offset by the licensor expense reimbursement payments that the Company has made to the institution subject to certain limitations.
Litigation
The Company is not a party to any litigation and did not have contingency reserves established for any litigation liabilities as of December 31, 2024 or 2023.
9. Collaboration Agreements
The Company has entered into multiple collaborations, out-licenses and strategic alliances with third parties that typically involve payments to or from the Company, including up-front payments, payments for research and development services, option payments, milestone payments and royalty payments to or from the Company.
Collaboration Revenue
As of December 31, 2024, the Company’s contract liabilities were primarily related to the Company’s collaboration with BMS and the Company's license agreement with Vertex. The following table presents changes in the Company’s accounts receivable and contract liabilities for the year ended December 31, 2024 (in thousands):
For the year ended December 31, 2024 Balance at December 31, 2023 Additions Deductions Balance at December 31, 2024
Accounts receivable $ 10,187 $ 22,644 $ ( 16,565 ) $ 16,266
Contract liabilities:
Deferred revenue $ 68,888 $ — $ ( 8,463 ) $ 60,425
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During the years ended December 31, 2024 and 2023, the Company recognized the following collaboration revenue (in thousands):
Revenue recognized in the period from: December 31, 2024 December 31, 2023
Amounts included in deferred revenue at the beginning of the period $ 8,463 $ —
Performance obligations satisfied in previous periods $ — $ —
Amendment to BMS Collaboration Agreement
In March 2024, the Company entered into an amendment (“2024 Amendment”) to the Second Amended and Restated Collaboration and License Agreement, dated as of November 11 2019, by and between the Company and Juno Therapeutics (the “2019 Collaboration Agreement”) extend the collaboration to November 2026, with options to extend the collaboration for up to an additional two years , and provided BMS the ability to select up to three new gene targets for research.
Accounting Assessment
The Company evaluated the 2024 Amendment and concluded that the agreement qualifies as a contract with a customer under ASC 606. The contract modification was accounted for on a prospective basis as if it were a termination of the existing contract and the creation of a new contract since the promised goods and services were distinct from the goods and services that were transferred on or before the effective date of the amendment.
The Company has identified the following performance obligations under the 2024 Amendment: eighteen material rights for additional development and commercialization licenses for other gene editing tools specific to a gene target and enzyme combination (or a “Program”).
As of December 31, 2024 the total transaction price was approximately $ 56.7 million, comprised of the remaining deferred revenue balance that was not recognized pursuant to the 2019 Amended Collaboration Agreement. The Company utilized the most likely amount method to estimate any development and regulatory milestone payments to be received, as well as extension term fees. As of December 31, 2024, there were no milestone or extension term fees included in the transaction price. The Company considers the stage of development and the risks associated with the remaining development required to achieve the milestone, as well as whether the achievement of the milestone is outside the control of the Company or BMS. The outstanding milestone payments and extension term fees were fully constrained as of December 31, 2024, as a result of the uncertainty of whether any of the milestones will be achieved or the term would be extended. The Company has determined that any commercial milestones and sales-based royalties will be recognized when the related sales occur. The Company reevaluates the transaction price at each reporting period and as uncertain events are resolved or other changes in circumstances occur.
The Company concluded that rights and attributes of each of the development and commercialization licenses are identical for both the license granted at inception and the licenses that may be issued in the future upon exercise of the associated option. Each development and commercialization license is differentiated only by the Program to which it relates. The Company has considered the early stage of the science and the uncertainty of success and concluded that the probability of scientific success and opt-in is equal amongst all Programs. In addition, each Program is multi-functional, and a combination of Programs can be utilized in the development of a product candidate. As such, the Company concluded that the standalone selling price of each material right is the same. The Company will recognize the transaction price allocated to each material right when the material right is exercised, lapsed or expired.
During the years ended December 31, 2024, 2023 and 2022, the Company recognized $ 18.1 million, $ 4.5 million and $ 18.8 million of revenue related to BMS. As of December 31, 2024, the Company recorded $ 50.2 million of deferred revenue, of which $ 50.2 million is classified as long-term on the Company's consolidated balance sheets.
Beam Therapeutics License Agreement
In 2018, the Company entered into a license agreement with Beam Therapeutics Inc. (“Beam,” and such agreement, the “Beam License Agreement”). Pursuant to the Beam License Agreement, the Company granted to Beam a worldwide, exclusive (subject to certain exceptions), sublicensable (subject to certain conditions), development and
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commercialization license under certain intellectual property controlled by the Company for the use of base editing therapies for the treatment of any field of human diseases and conditions, such to certain exceptions. Additionally, the Company granted Beam a non-exclusive research license. Lastly, the Company provided to Beam with an exclusive option to obtain three development and commercialization licenses to additional groups of intellectual property owned or controlled by the Company, on a group-by-group basis, during the specified option period, subject to certain exceptions.
The Company received preferred stock valued at $ 3.6 million and received a nominal upfront cash payment. The Company subsequently sold its equity investment in Beam following Beam’s initial public offering in 2023. The Company is also eligible to receive additional consideration if Beam exercises its option to obtain additional licenses for a fee ranging from a mid-teen million-dollar amount to a low to mid-eight-digit dollar amount per license, depending on the timing of the option exercise. To the extent that any products are commercialized, the Company would be entitled to receive tiered low single-digit royalty payments, plus any royalties that would be due from the Company to any applicable licensors related to the sale of such licensed products.
Unless earlier terminated by either party pursuant to the terms of the agreement, the Beam License Agreement will continue in full force and effect and will expire on a licensed product-by-licensed product and country-by-country basis upon the expiration of the royalty term with respect to such licensed product in such country. Beam has the right, at its sole discretion, at any time to terminate the Beam License Agreement in its entirety or on a group-by-group of intellectual property basis, upon ninety days written notice to the Company. Upon termination, all rights and licenses granted by the Company will immediately terminate.
Accounting Assessment
The Company identified the following performance obligations (i) the research license and (ii) the initial development and commercialization license. In addition, the Company concluded that the three options for the additional development and commercialization licenses are not discounted and therefore they do not represent material rights.
The total transaction price at the inception of the arrangement was determined to be approximately $ 3.8 million, consisting of the upfront cash payment and the non-cash value of the preferred shares received by the Company. The consideration associated with the exercise of the option(s) will be accounted for if and when Beam elects to exercise their options. The other forms of consideration, including nominal cost reimbursement for past patent and license fees and sublicense income reimbursement are based on the most-likely amount and were excluded from the initial transaction price as the most likely amount was estimated to be zero or the amount was otherwise fully constrained due to the significant uncertainties surrounding each payment. The commercial-based milestone reimbursement and the sales-based royalty payments will be recognized when the related sales occur as they were determined to relate predominantly to the licenses granted and therefore have also been excluded from the transaction price. Since both of the performance obligations were delivered at the inception of the arrangement and the licenses were made available for Beam’s use and benefit, the Company recognized the total transaction price at the inception of the agreement.
During the years ended December 31, 2024, 2023 and 2022, the Company recognized revenue under the Beam License Agreement of approximately $ 0.8 million, $ 0.2 million and $ 0.3 million, respectively.
Vertex Pharmaceuticals License Agreement
On December 12, 2023, the Company and Vertex entered into a license agreement. Under terms of the agreement, Vertex obtained a non-exclusive license for Editas Medicine’s Cas9 gene editing technology for ex vivo gene editing medicines targeting the BC11A gene in the fields of sickle cell disease and beta thalassemia, including exagamglogene autotemcel (exa-cel). In connection with the license agreement License Agreement, the Company received an upfront payment of $ 50.0 million. The Company is also eligible to receive annual license fees ranging from $ 10.0 million to $ 40.0 million annually, inclusive of certain fixed and sales-based annual license fee increases, through 2034 and other contingent fees of $ 50.0 million.
Accounting Assessment
The Company assessed this arrangement in accordance with Topic 606 and concluded that the contract counterparty, Vertex, is a customer.
The Company identified a single performance obligation, which is the non-exclusive license to certain Cas9-I patents.
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The total transaction price at the inception of the arrangement was determined to be $ 60.0 million, consisting of an upfront cash payment and the first annual license fee installment. The remaining annual license fees and contingent fees represent variable consideration that was evaluated under the most likely amount method, and excluded from the initial transaction price because the amounts were fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including that receipt of the annual license fees and contingent fees are outside the control of the Company. They will be recognized when each payment is determined to be probable. The Company will re-evaluate the transaction price in each reporting period, as uncertain events are resolved, or as other changes in circumstances occur. Since the single performance obligation was delivered at the inception of the arrangement and the license was made available for Vertex’s use and benefit, the Company recognized the total transaction price at the inception of the agreement.
During the years ended December 31, 2024 and 2023, the Company recognized revenue under the Vertex License Agreement of $ 10.0 million and $ 60.0 million, respectively.
10. Preferred Stock
The Company’s amended and restated certificate of incorporation authorized 5,000,000 shares of undesignated preferred stock that may be issued from time to time by the Board in one or more series. As of December 31, 2024, the Company had no shares of preferred stock issued or outstanding.
11. Common Stock
The voting, dividend, and liquidation rights of the holders of the common stock are subject to and qualified by the rights, powers, and preferences of holders of the preferred stock that may be issued from time to time. The common stock had the following characteristics as of December 31, 2024:
Voting
The holders of shares of common stock are entitled to one vote for each share of common stock held at any meeting of stockholders and at the time of any written action in lieu of a meeting.
Dividends
The holders of shares of common stock are entitled to receive dividends, if and when declared by the Board. Cash dividends may not be declared or paid to holders of shares of common stock until all unpaid dividends on any outstanding redeemable convertible preferred stock have been paid in accordance with their terms. No dividends have been declared or paid by the Company since its inception.
2013 Stock Incentive Plan
In September 2013, the board of directors adopted the 2013 Stock Incentive Plan, which was subsequently amended (as amended, the “2013 Plan”), which provides for the grant of incentive stock options and nonqualified stock options or other awards including restricted stock awards, unrestricted stock awards, and restricted stock units to the Company’s employees, officers, directors, advisors, and consultants for the purchase of up to 1,057,692 shares of the Company’s common stock, which has been amended several times, and as of July 2015, a total of 6,317,769 shares were reserved.
The terms of stock awards agreements, including vesting requirements, are determined by the board of directors and are subject to the provisions of the 2013 Plan. The stock options granted to employees generally vest over a four-year period and expire ten years from the date of grant. Certain awards contain performance based vesting criteria. There has only been one such award to date. Certain options provide for accelerated vesting in the event of a change in control, as defined in the applicable options. Awards granted to non-employee consultants generally vest monthly over a period of one to four years . In connection with the Company’s initial public offering (“IPO”), the Board determined to grant no further awards under the 2013 Plan.
2015 Stock Incentive Plan
The Board adopted and the Company’s stockholders approved the 2015 stock incentive plan (the “2015 Plan”). The 2015 Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards,
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restricted stock units, stock appreciation rights and other stock-based awards. The Company’s employees, officers, directors and consultants and advisors are eligible to receive awards under the 2015 Plan.
The number of shares reserved for issuance under the 2015 Plan is subject to further increases for (a) any additional shares of the Company’s common stock subject to outstanding awards under the 2013 Plan that expire, terminate, or are otherwise surrendered, cancelled, forfeited, or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right and (b) annual increases, to be added as of the first day of each fiscal year, from January 1, 2017 until, and including, January 1, 2026, equal to the lowest of 2,923,076 shares of common stock, 4 % of the number of shares of common stock outstanding on such first day of the fiscal year in question and an amount determined by the Company’s board of directors. In January 2025, the remaining shares reserved but unissued under the 2015 Plan increased to 12,248,887 shares pursuant to the annual increase described in the prior sentence.
2015 Employee Stock Purchase Plan
The Company’s board of directors adopted and the Company’s stockholders approved the 2015 employee stock purchase plan (the “2015 ESPP”). The number of shares reserved for issuance under the 2015 ESPP is subject to annual increases, to be added as of the first day of each fiscal year, from January 1, 2017 until, and including, January 1, 2026, in an amount equal to the least of (a) 769,230 shares of common stock, (b) 1 % of the total number of shares of common stock outstanding on the first day of the applicable year, and (c) an amount determined by the board of directors. The first offering under the 2015 ESPP opened on December 1, 2017. In January 2025, the board of directors determined that there should be no increase in shares available under the 2015 ESPP for 2025.
Inducement Awards
From time to time the Board approves inducement awards to certain employees outside of the existing equity compensation plans in connection with such employees commencing employment with the Company. Inducement awards are typically a service-based option and a restricted stock unit and are subject to the Company’s typical vesting terms and the employee’s continued service relationship with the Company through the applicable vesting dates. In May 2023, July 2023 and September 2023, the Board approved three inducement grants to the Company’s recently hired Chief Financial Officer, Chief Scientific and Chief Commercial and Strategy Officer, respectively.
Shares Reserved for Future Issuance
As of December 31,
2024 2023
Shares reserved for outstanding stock option awards under the 2013 Plan, as amended 114,108 115,942
Shares reserved for outstanding stock option awards and restricted stock units under the 2015 Plan 8,395,995 6,450,615
Shares reserved for outstanding inducement stock option award and restricted stock units 1,536,461 1,690,235
Remaining shares reserved, but unissued, for future awards under the 2015 Plan 9,673,426 9,100,247
Remaining shares reserved, but unissued, for future awards under the 2015 ESPP 2,972,026 3,185,493
22,692,016 20,542,532
12. Stock-Based Compensation
Total compensation cost recognized for all stock-based compensation awards in the consolidated statements of operations was as follows (in thousands):
Year Ended
December 31,
2024 2023
Research and development $ 8,642 $ 9,842
General and administrative 12,775 9,956
Total stock-based compensation expense $ 21,417 $ 19,798
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Restricted Stock and Restricted Stock Unit Awards
The following table summarizes restricted stock and restricted stock unit awards activity for the instruments discussed above as of December 31, 2024 and 2023 is as follows:
Shares Weighted
Average
Grant Date
Fair Value
Per Share
Unvested restricted stock and restricted stock unit awards as of December 31, 2023 2,107,147 $ 11.96
Issued 1,504,321 $ 9.17
Vested ( 730,157 ) $ 12.58
Forfeited ( 554,788 ) $ 12.71
Unvested restricted stock and restricted stock unit awards as of December 31, 2024 2,326,523 $ 9.78
The expense related to restricted stock and restricted stock unit awards granted for the years ended December 31, 2024, 2023 and 2022 was $ 5.3 million, $ 5.1 million, and $ 7.9 million, respectively.
The restricted stock and restricted stock units granted in the year ended December 31, 2024 include 422,000 units granted to certain employees that contain performance-based vesting provisions. The expense related to the performance-based vesting of restricted stock units was $ 3.5 million, $ 0.5 million and $ 4.4 million for the years ended December 31, 2024, 2023 and 2022, respectively.
As of December 31, 2024, total unrecognized compensation expense related to unvested restricted stock and restricted stock unit awards was $ 12.2 million, which the Company expects to recognize over a remaining weighted-average period of 2.3 years.
Stock Options
The following is a summary of stock option activity for the year ended December 31, 2024:
Shares Weighted Average
Exercise Price Remaining
Contractual Life (years) Aggregate Intrinsic
Value (in thousands)
Outstanding at December 31, 2023 6,149,645 $ 16.47 6.98 $ 3,195
Granted 2,966,836 $ 8.07
Exercised ( 23,809 ) $ 8.10
Cancelled ( 1,436,662 ) $ 30.43
Outstanding at December 31, 2024 7,656,010 $ 14.70 7.19 $ —
Exercisable at December 31, 2024 3,964,202 $ 19.87 5.76 $ —
The total intrinsic value of options exercised for the years ended December 31, 2024, 2023 and 2022 was $ 0.04 million, $ 0.03 million, and $ 0.09 million, respectively.
Using the Black-Scholes option pricing model, the weighted average fair value of options containing service-based vesting granted during the years ended December 31, 2024, 2023, and 2022 was $ 5.62 , $ 6.04 , and $ 15.87 , respectively. The expense related to options containing service-based vesting was $ 12.2 million, $ 13.9 million, and $ 14.1 million for the years ended December 31, 2024, 2023, and 2022, respectively.
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The fair value of each service-based vesting option issued was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions:
Year Ended
December 31,
2024 2023 2022
Expected volatility 75.6 % 75.2 % 64.2 %
Expected option term (in years) 6.20 6.19 6.25
Risk free interest rate 4.2 % 4.2 % 1.7 %
Expected dividend yield — — —
As of December 31, 2024, total unrecognized compensation expense related to stock options was $ 22.1 million, which the Company expects to recognize over a remaining weighted-average period of 2.60 years.
13. 401(k) Savings Plan
The Company has a defined-contribution savings plan under Section 401(k) of the Internal Revenue Code of 1986, as amended (the “401(k) Plan”). The 401(k) Plan covers all employees who meet defined minimum age and service requirements, and allows participants to defer a portion of their annual compensation on a pretax basis. Effective in 2017, the Company will provide a 200 % match of employee contributions up to a limit on the Company’s contributions of the lesser of $ 7,000 and 3 % of the employee’s salary. The Company made $ 1.7 million, $ 1.2 million, and $ 1.4 million in contributions to the 401(k) Plan for the years ended December 31, 2024, 2023 and 2022, respectively.
14. Income Taxes
The Company had no income tax expense due to operating losses incurred for the years ended December 31, 2024 and 2023.
A reconciliation of the income tax expense computed using the federal statutory income tax rate to the Company’s effective income tax rate is as follows:
Year Ended
December 31,
2024 2023
Income tax computed at federal statutory tax rate 21.0 % 21.0 %
State taxes, net of federal benefit 4.9 % 6.0 %
General business credit carryovers 2.6 % 2.5 %
162m limitation ( 0.2 ) % — %
Stock options ( 0.2 ) % ( 4.3 ) %
Non-deductible expenses ( 0.1 ) % ( 0.2 ) %
Tax rate changes ( 2.6 ) % ( 4.5 ) %
Change in valuation allowance ( 25.5 ) % ( 21.8 ) %
Other — % 1.3 %
( 0.1 ) % — %
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The principal components of the Company’s deferred tax assets and liabilities consist of the following at December 31, 2024 and 2023 (in thousands):
Year Ended
December 31,
2024 2023
Deferred tax assets:
Net operating loss carryforwards $ 170,770 $ 153,365
Tax credit carryforwards 31,766 25,646
Accrued expenses 6,763 3,975
Capitalized patent costs 52,178 56,955
Capitalized research 84,703 60,891
Lease liabilities 9,134 9,966
Deferred revenue 15,755 18,791
Depreciation and amortization 978 905
Stock compensation 10,431 7,323
Other 14,976 —
Total deferred tax assets 397,454 337,817
Less valuation allowance ( 388,967 ) ( 328,630 )
Net deferred tax assets 8,487 9,187
Deferred tax liabilities ( 8,487 ) ( 9,187 )
Depreciation and amortization — —
Right-of-use assets ( 8,487 ) ( 9,187 )
Net deferred taxes $ — $ —
For taxable years beginning after December 31, 2021, the Tax Cuts and Jobs Act (the "Tax Act”) eliminated the option to deduct research and development expenditures in the current year and requires taxpayers to capitalize such expenses pursuant to Internal Revenue Code ( "IRC" ) Section 174. As a result of this provision of the Tax Act, deferred tax assets related to capitalized research expenses pursuant to IRC Section 174 was $ 84.7 million for the year ended December 31, 2024.
The Company has incurred net operating losses (“NOL”) since inception. At December 31, 2024 and 2023, the Company had federal net operating loss carryforwards of $ 612.8 million and $ 546.7 million, respectively. Of the amount as of December 31, 2024, $ 538.1 million will carryforward indefinitely while $ 74.8 million will expire beginning in 2035 and will continue to expire through 2037. As of December 31, 2024, and 2023, the Company also had state net operating loss carryforwards of approximately $ 710.5 million and $ 648.0 million, respectively, which may be available to offset future income tax liabilities and will expire beginning in 2035 and will continue to expire through 2044.
Under the provisions of the Internal Revenue Code of 1986, as amended (the “Code”), the NOL and tax credit carryforward are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. NOL and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Code, respectively, as well as other similar state provisions. The Company conducted an analysis under Section 382 to determine if historical changes in ownership through June 30, 2024 would limit or otherwise restrict its ability to utilize its NOL and research and development credit carryforwards. As a result of this analysis, the Company does not believe there are any significant limitations on its ability to utilize these carryforwards. However, future changes in ownership occurring after June 30, 2024 could affect the limitation in future years, and any limitation may result in expiration of a portion of the NOL or research and development credit carryforwards before utilization.
Management has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets, which principally comprise of NOL carryforwards, research and development credit carryforwards and capitalized license and patent costs. The Company’s management has determined that it is more likely than not that the Company will not recognize the benefits of its federal and state deferred tax assets, and as a result, a valuation allowance of $ 389.0
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million and $ 328.6 million has been established at December 31, 2024 and 2023, respectively. The increase in the valuation allowance of $ 60.3 million for the year ended December 31, 2024 was primarily due to current period pre-tax losses incurred and research tax credits generated.
The Company applies ASC 740 related to accounting for uncertainty in income taxes. The Company’s reserves related to income taxes are based on a determination of whether, and how much of, a tax benefit taken by the Company in its tax filings or positions is more likely than not to be realized following resolution of any potential contingencies present related to the tax benefit.
The following table summarizes the activity related to the Company’s gross unrecognized tax benefits at the beginning and end of the years ended December 31, 2024 and 2023 (in thousands):
Year Ended
December 31, 2024
Balance as of December 31, 2023 $ 13,659
Gross increases for tax positions related to current year 2,020
Gross increases for tax positions related to prior year —
Balance as of December 31, 2024 $ 15,679
At December 31, 2024 and 2023, the Company had unrecognized tax benefits of $ 15.7 million and $ 13.7 million, respectively. The Company will recognize interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2024 and 2023, the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’s statement of operations. The Company does not anticipate a material change to unrecognized tax benefits in the next twelve months.
The Company has not as of yet conducted a study of its research and development credit carry forwards. This study may result in an adjustment to the Company’s research and development credit carryforwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. A full valuation allowance has been provided against the Company’s research and development credits, and if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance. Thus, there would be no impact to the consolidated balance sheets or statements of operations if an adjustment were required.
The Company files income tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In the normal course of business, the Company is subject to examination by federal and state jurisdictions, where applicable. Since the Company is in a loss carryforward position, the Company is generally subject to examination by the U.S. federal, state and local income tax authorities for all tax years in which a loss carryforward is available. The Company did not have any international operations as of December 31, 2024. An examination by the Internal Revenue Service (“IRS”) for the period ended December 31, 2018 related to its R&D tax credits concluded in December 31, 2022 and resulted in a reduction to the Company’s deferred tax assets.
15. Net Loss per Share
Basic net loss per common share is calculated by dividing the net loss attributable to common stockholders by the weighted average number of shares of common stock outstanding during the period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss attributable to common stockholders by the weighted average number of shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury stock and if converted methods. Contingently issuable shares are included in the calculation of basic loss per share as of the beginning of the period in which all the necessary conditions have been satisfied. Contingently issuable shares are included in diluted loss per share based on the number of shares, if any, that would be issuable under the terms of the arrangement if the end of the reporting period was the end of the contingency period, if the results are dilutive.
For purposes of the diluted net loss per share calculation, stock options are considered to be common stock equivalents, but they were excluded from the Company’s calculation of diluted net loss per share allocable to common stockholders because their inclusion would have been anti-dilutive. Therefore, basic and diluted net loss per share applicable to common stockholders was the same for all periods presented.
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The following common stock equivalents were excluded from the calculation of diluted net loss per share allocable to common stockholders because their inclusion would have been anti-dilutive:
As of December 31,
2024 2023
Unvested restricted stock and restricted stock unit awards 2,326,523 2,107,147
Outstanding stock options 7,656,010 6,149,645
Total 9,982,533 8,256,792
The table above reflects restricted stock issued upon exercise of unvested stock options as exercised on the dates that the shares are no longer subject to repurchase.
16. Debt
Liability for the Sale of Future Revenues
The following table presents the changes in the liability related to the sale of future revenues under the Purchase and Sale Agreement with DRI as of December 31, 2024 (in thousands):
December 31, 2024
Proceeds from sale of future revenues $ 57,000
Issuance costs ( 1,756 )
Non-cash interest expense associated with sale of future revenues 2,128
Amortization of issuance costs 62
Deferred royalty obligation related to the sale of future revenues net $ 57,434
17. Restructuring
On December 11, 2024, the Board approved the discontinuation of the clinical development of the Company's reni-cel program (the “Discontinuation”) to treat sickle cell disease and transfusion-dependent beta thalassemia. As a result of the Discontinuation, the Company has ceased activities towards the filing of a biologic license application and potential commercialization of reni-cel. In connection with the Discontinuation, the Board also approved a reduction in the Company’s employee workforce by approximately 180 positions, or approximately 65 % (the “Reduction”).
As a result of these events, the Company incurred the following restructuring charges for the year ended December 31, 2024 in accordance with ASC 420 - Exit or Disposal Cost Obligations and ASC 712 Compensation - Non-retirement Postemployment Benefits , which are recorded in the consolidated statement of operations (in thousands):
Employee termination benefits $ 10,475
Contract termination costs 1,757
Restructuring charges $ 12,232
The actions associated with the Discontinuation and Reduction commenced in December 2024 and are expected to be substantially completed by the end of June 2025. In addition to the items discussed within this note, the Company anticipates that as part of the Discontinuation, it will incur impairment charges in the range of $ 11.0 million to $ 14.0 million for the related right of use asset and property and equipment.
Employee Termination Benefits
Employees affected by the Reduction obtained involuntary termination benefits that are provided pursuant to either a one-time benefit or arrangement or salary continuation for a set period of time in accordance with the Company’s
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Amended and Restated Severance Benefit Plan (the “Benefit Plan”). For employees who were notified of their termination in December 2024 and have no requirements to provide future services or were subject to the Benefit Plan, the Company recognized the liability for the termination benefits in full at fair value in the fourth quarter of 2024. For employees who are required to render services beyond a minimum retention period to receive their one-time termination benefits or salary continuation, the Company is recognizing the termination benefits ratably over their future service periods. The service periods began in December 2024 and all will end at various dates throughout 2025.
The following table shows the liability related to employee termination benefits as of December 31, 2024 (in thousands):
Employee Termination Benefits
Accrued employee termination benefits beginning balance $ —
Employee termination benefits charges incurred during period 10,475
Amounts paid or otherwise settled during the period —
Accrued employee termination benefits as of December 31, 2024
$ 10,475
In total, the Company expects that it will incur approximately $ 14.9 million of employee termination benefit expense to implement the Reduction.
Contract Termination Costs
The Discontinuation resulted in contract termination costs from vendor contracts before the end of their term, as well as costs that continue to be incurred under certain contracts with no future economic benefit to the Company. In accordance with ASC 420, the Company recognized these unavoidable contract termination costs when incurred or when it has been determined that there is no future economic benefit to the Company, as it relates to costs that continue to be incurred.
The following table shows the liability related to contract termination costs as of December 31, 2024 (in thousands):
Contract Termination Costs
Accrued contract termination costs $ —
Contract termination costs incurred during the period 1,757
Amounts paid or otherwise settled during the period —
Accrued contract termination costs as of December 31, 2024
$ 1,757
In total, the Company anticipates incurring $ 30.0 million to $ 40.0 million in costs related to the completion of the Discontinuation.
18. Segments
Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the CODM or decision-making group in making decisions on how to allocate resources and assess performance. The Company’s CODM is its CEO. The CEO views the Company’s operations and manages the Company’s business as one operating segment, which is the business of developing and commercializing genome editing technology.
The Company’s CEO manages and allocates resources to the operations of the Company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and research and development projects that are in line with our long-term company-wide strategic goals. In making these decisions, the Company’s CEO uses consolidated financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets. The CODM performs this assessment based on the Company’s consolidated net loss. Through this analysis, the CODM assesses performance by comparing actual
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consolidated net loss versus the budget, and then decides how to allocate resources to invest in the Company’s research and development programs. The measure of segment assets is reported on the consolidated balance sheet as total assets.
The following table contains additional information on our consolidated revenue and net loss, including significant segment expenses (in thousands):
Year Ended
December 31,
2024 2023 2022
Collaboration and other research and development revenues $ 32,314 $ 78,123 $ 19,712
Operating expenses:
Research and development 1
Employee related expenses 61,136 47,363 47,320
External research and development expenses 79,900 53,321 79,822
Facility expenses 26,430 21,447 21,032
Stock-based compensation expenses 8,642 9,842 12,425
Sublicense and license fees 18,953 35,011 5,334
Other expenses 3
12,768 10,667 9,025
General and administrative 2
Employee related expenses 24,335 16,969 17,321
Professional service expenses 14,358 19,575 11,496
Intellectual property and patent related fees 14,016 13,268 14,784
Stock-based compensation expenses 12,775 9,956 16,869
Facility and other expenses 4
10,153 9,885 10,234
Total operating expenses 283,466 247,304 245,662
Operating loss ( 251,152 ) ( 169,181 ) ( 225,950 )
Other income, net:
Other expense, net ( 3 ) ( 1,604 ) —
Interest income, net 14,062 17,566 5,518
Total other income, net 14,059 15,962 5,518
Net loss $ ( 237,093 ) $ ( 153,219 ) $ ( 220,432 )
1 For the years ended December 31, 2024, 2023, and 2022 includes $ 8,582 , $ 0 , and $ 0 of restructuring charges, respectively
2 For the years ended December 31, 2024, 2023, and 2022 includes $ 3,650 , $ 0 , and $ 0 of restructuring charges, respectively
3 Other expenses primarily consists of consultant fees and office expenses
4 Facility and other expenses primarily consists of rent expense, insurance premiums, software licenses and office expenses
For the year ended December 31, 2024, there were two customers for which revenues amount to 10% or more of the Company’s consolidated collaboration and other research and development revenues, representing $ 18.1 million and $ 10.0 million of the Company’s consolidated collaboration and other research and development revenues. For the year ended December 31, 2023, there was one customer for which revenues amounts to 10% or more of the Company’s consolidated collaboration and other research and development revenues, representing $ 60.0 million of the Company’s consolidated collaboration and other research and development revenues. For the years ended December 31, 2024 and 2023, 100 % and 100 %, respectively, of the Company’s consolidated collaboration and other research and development revenues was attributed to the U.S.
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19. Subsequent Events
The Company evaluated subsequent events after the audited balance sheet date of December 31, 2024 but prior to the issuance of the financial statements to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The Company did not identify any subsequent events requiring further disclosure.
Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure.
None.