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Item 7. Management’s Discussion and Analysis

2026-03-09 compared with 2025-03-05 · 153 added, 129 removed, 183 unchanged (61% of the section changed)

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Our product development strategy is to target diseases where gene editing can be used to enable or enhance therapeutic outcomes for patients, while maximizing probability of technical, regulatory and commercial success.

We are focused on the development of in vivo gene editing medicines utilizing functional upregulation, which aims to increase the expression and function of a normal gene copy and its normal protein function to treat diseases caused by genetic mutations that eliminate or disrupt normal function.

We are focused on the development of in vivo gene editing medicines utilizing functional upregulation, which aims to increase the expression of a normal gene copy and its normal protein function to treat diseases caused by genetic mutations that eliminate or disrupt normal function.

We believe the ability to provide in vivo gene editing, in which the medicine is injected or infused into the patient to edit the cells inside their body, and functionally upregulates normal gene expression and normal protein function in the target tissues holds the potential to significantly expand the addressable therapeutic possibilities of CRISPR-based gene editing.

To that end, our preclinical efforts are also focused on the creation of a “plug ‘n play” LNP platform that enables delivery of the gene editing cargo to multiple cells and tissues, including hematopoietic stem cells (“HSCs”), the liver and other cells and tissues.

We previously demonstrated proof of concept of our functional upregulation strategy in our clinical trials of renizgamglogene autogedtemcel (“reni-cel”), an experimental ex vivo gene-edited medicine to treat sickle cell disease (“SCD”) and transfusion-dependent beta thalassemia (“TDT”).

Despite the robust and clinically meaningful improvements observed in these trials, we determined in December 2024 not to pursue commercialization for reni-cel in order to optimize our cost structure and accelerate our intent to achieve in vivo human proof of concept in approximately two years.

However, l everaging our differentiated approach and the insights gained from these trials, we are initially focused on pursuing next generation in vivo gene editing medicines targeting HSCs.

In October 2024, we shared our achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell editing and fetal hemoglobin induction in

humanized mice engrafted with human HSCs and lacking their own hematopoietic cells using a novel and proprietary targeted lipid nanoparticle (“tLNP”) formulation for non-liver, or extrahepatic, tissue delivery.

Building on this achievement, in January 2025, we announced in vivo preclinical proof of concept of editing HSCs in non-human primates, a key step in developing a treatment for SCD and TDT.

We are on track to declare a development candidate in HSCs for the in vivo treatment of SCD and TDT in mid-2025.

Beyond hemoglobinopathies, our discovery and development efforts are focused on in vivo gene editing medicines in liver cells and other cells and tissues.

In preclinical studies for an undisclosed liver target, we have achieved an editing level of approximately 65% in non-human primates, near the theoretical maximum liver editing level of 70%, with minimal non-target tissue editing using AsCas12a and a liver targeted lipid nanoparticle (“LNP”).

This editing resulted in robust serum biomarker reduction from the baseline of approximately 80%.

We further announced in vivo delivery to two additional cell types in humanized mice using our proprietary LNP targeting platform.

We are on track to declare a development candidate for an in vivo gene editing medicine in liver cells for an undisclosed indication in mid-2025 and to establish and disclose an extrahepatic, non-HSC target cell type or tissue by the end of 2025.

We expect to present further in vivo HSC data and present further in vivo data in one liver indication by the end of 2025.

To that end, our preclinical efforts are also focused on the creation of a “plug ‘n play” lipid nanoparticle (“LNP”) platform to enable targeted delivery of in vivo gene editing medicines to multiple cells and tissues, including the liver, hematopoietic stem cells (“HSCs”), and other cells and tissues.

In September 2025, we announced the nomination of our lead in vivo development candidate, EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (“LDL-C”) through upregulation of the LDL receptor (“LDLR”).

EDIT-401 is designed to treat elevated levels of LDL-C, or hyperlipidemia, by directly editing the noncoding region of the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.

This targeted approach has demonstrated an approximately 90% mean reduction of LDL-C in non-human primates (“NHPs”) in our preclinical studies with favorable tolerability data, and supports the potential of EDIT-401 to deliver

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meaningful clinical outcomes for patients underserved by current lipid-lowering therapies.

We are on track to submit an investigational new drug application (“IND”) or foreign equivalent to conduct a clinical trial of EDIT-401 in patients with heterozygous familial hypercholesteremia by mid-2026 with the expectation of achieving early human proof-of-concept data for EDIT-401 by the end of 2026.

We plan to complete enrolling the dose-finding portion of the first-in-human clinical trial with topline data results available in 2027.

We expect to present additional preclinical data for EDIT-401 by mid-2026.

Our discovery and development efforts further include HSCs and other cells and tissues.

Building on our experience in our clinical trials of renizgamglogene autogedtemcel (“reni-cel”) , we have achieved in vivo preclinical proof-of-concept data of HSC editing in NHPs.

In addition, w e previously announced in vivo delivery to two additional cell types in humanized mice using our proprietary LNP targeting platform, demonstrating the “plug ‘n play” potential of our proprietary extrahepatic LNP platform.

We intend to continue optimizing candidates for our HSC program and exploring other cell types and tissues for development, but plan to focus our resources on the advancement of our lead EDIT-401 program to human proof-of-concept.

We are pursuing the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies to build our preclinical pipeline and accelerate the achievement of our goal of delivering lifesaving medicines to patients with previously untreatable diseases.

Through in-licensing of complementary technologies, we can expand our existing gene editing platform and further drive the development of our in vivo pipeline.

This was recently demonstrated with our entry in 2024 into a collaboration and license agreement to access LNPs targeting the liver, which we used in our achievement of in vivo editing of liver cells in non-human primates.

This was demonstrated with our entry in 2024 into a collaboration and license agreement to access LNPs targeting the liver, including the LNP we are using in our EDIT-401 program.

We also actively seek opportunities to out-license and partner our robust intellectual property portfolio to drive the development of CRISPR-based medicines in therapeutic areas outside of our core focus and to provide non-dilutive capital.

For example, in cellular therapy medicines, we are leveraging partnerships to progress engineered cell medicines to treat various cancers, including in our collaboration with Bristol Myers Squibb Company (“BMS”) through its wholly owned subsidiary, Juno Therapeutics, Inc.

For example, we are leveraging partnerships to progress engineered cell medicines to treat various cancers, including in our collaboration with Bristol Myers Squibb Company (“BMS”) through its wholly owned subsidiary, Juno Therapeutics, Inc.

(“Juno Therapeutics”).

This collaboration, which leverages our Cas9 and AsCas12a platform technologies, seeks to advance alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors, and has resulted in 14 total programs to date.

In addition, in December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement, under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel).

This collaboration, which leverages our Cas9 and AsCas12a platform technologies, seeks to advance alpha-beta T-cell experimental medicines for the treatment of solid tumors, liquid tumors, and autoimmune disease, and has resulted in 14 total programs to date, including BMS’ CD19 HD Allo CAR T program for the treatment of autoimmune disease currently in Phase I clinical development.

In addition, in December 2023, we and Vertex Pharmaceuticals Incorporated (“Vertex”) entered into a license agreement (the “Vertex License Agreement”), under which Vertex obtained a non-exclusive license for our Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex’s CASGEVY TM (exagamglogene autotemcel).

We received a $50.0 million upfront cash payment in the fourth quarter of 2023 and the 2024 annual license fee of $10.0 million in the first quarter of 2024.

The license agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further future fixed and sales-based annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.

The Vertex License Agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further future fixed and sales-based annual license fees, ranging from $5.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.

We are required to pay The Broad Institute, Inc.

(“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts payable to us from Vertex under the license agreement as it relates to Cas9 technology licensed by us from Broad and Harvard.

In October 2024, we entered into an agreement (the “DRI Agreement”) with a wholly owned subsidiary of DRI Healthcare Trust (“DRI”) providing for an upfront cash payment by DRI to us of $57.0 million in exchange for the acquisition by DRI of up to 100% of certain of the annual license fees owed to us 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 fees that may become due), and a mid-double-digit percentage of the $50.0 million contingent upfront payment, in each case after subtracting amounts owed by us to Broad and Harvard.

We have retained rights to our portions of certain other sales-based annual license fees and the contingent upfront payment that may become due under the license agreement with Vertex, and the amounts that correspond to our licensor obligations.

In August 2023, we entered into a license agreement with Vor Biopharma, Inc.

(“Vor Bio”), providing Vor Bio a non-exclusive license for the development of ex vivo Cas 9 gene edited HSC therapies for the treatment and/or prevention of hematological malignancies.

Under this agreement, we received an upfront payment and will be eligible for future development, regulatory and commercial milestone payments, as well as royalties on medicines utilizing the related intellectual property.

Since our inception in September 2013, our operations have focused on organizing and staffing our company, business planning, raising capital, establishing our intellectual property portfolio, assembling our core capabilities in gene editing, seeking to identify potential product candidates, and undertaking preclinical studies and clinical trials.

All of our ongoing research programs are still in the preclinical or research stage of development and the risk of failure of all of our

research programs is high.

(“Broad”) and the President and Fellows of Harvard College (“Harvard”) a mid-double-digit percentage of amounts payable to us from Vertex under the Vertex License Agreement as it relates to Cas9 technology licensed by us from Broad and Harvard.

Under the DRI Agreement, DRI is purchasing up to 100% of certain future fixed and sales-based annual license fees that the Company is entitled to receive under the Vertex License Agreement, which fees range from $5.0 million to $40.0 million per year, including increases based on sales.

In addition, DRI is purchasing a mid-double-digit percentage of a $50.0 million contingent upfront payment that the Company may receive under the Vertex License Agreement.

All amounts above will be adjusted to exclude payments that the Company owes to Broad and Harvard.

The Company has retained rights to certain portions of certain other 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 our licensor obligations.

Our operations to date have focused on organizing and staffing our company, business planning, raising capital, establishing our intellectual property portfolio, assembling our core capabilities in gene editing, seeking to identify potential product candidates, and undertaking preclinical studies and clinical trials.

All of our ongoing research programs are still in the preclinical or research stage of development and the risk of failure of all of our research programs is high.

We have not generated any revenue from product sales.

We have primarily financed our operations through various equity financings, payments received under our research collaboration with BMS through its wholly owned subsidiary Juno Therapeutics, our former strategic alliance with Allergan Pharmaceuticals International Limited (together with its affiliates, “Allergan”), which was terminated in August 2020, payments received under the DRI Agreement in connection with our license agreement with Vertex, and payments under the Vertex license agreement.

Since inception, we have incurred significant operating losses.

Our net losses were $237.1 million, $153.2 million, and $220.4 million for the years ended December 31, 2024, 2023 and 2022 respectively.

We have primarily financed our operations through various equity financings, payments received under our research collaboration with BMS, our former strategic alliance with Allergan Pharmaceuticals International Limited (together with its affiliates, “Allergan”), which was terminated in August 2020, payments received under the DRI Agreement in connection with the Vertex License Agreement, and payments under the Vertex License Agreement.

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We have incurred significant operating losses since inception.

Our net losses were $160.1 million and $237.1 million for the years ended December 31, 2025 and 2024, respectively.

As of December 31, 2025, we had an accumulated deficit of $1.6 billion.

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Our net losses may fluctuate significantly from quarter to quarter and from year to year.

We anticipate that our expenses will increase substantially as we continue our current research programs and our preclinical development activities;

We anticipate that our expenses will inc rease as we continue to support preclinical studies and prepare for the clinical development of EDIT-401;

commence and conduct clinical trials of EDIT-401;

continue our current research programs and our preclinical development activities;

seek to identify additional research programs and additional product candidates;

initiate preclinical testing and clinical trials for other product candidates we identify and develop;

maintain, expand, and protect our intellectual property portfolio, including reimbursing our licensors for such expenses related to the intellectual property that we in-license from such licensors;

further develop our genome editing platform;

hire additional clinical, quality control, and scientific personnel;

and incur additional costs associated with operating as a public company.

We do not expect to be profitable for the year ending December 31, 2025 or the foreseeable future.

initiate preclinical testing for other product candidates we identify and develop;

maintain, expand, and protect our intellectual property portfolio, including reimbursing our licensors for such expenses related to the intellectual property that we in-license from such lice nsors;

further develop our gene editing platform;

and hire personnel.

We do not expect to be profitable for the year ending December 31, 2026 or for the foreseeable future.

Financial Operations Overview

To date, we have not generated any revenue from product sales and we do not expect to generate any revenue from product sales for the foreseeable future.

In connection with our collaboration with BMS, we have received an aggregate of $146.5 million in payments, which have primarily consisted of the initial upfront and amendment payments, development milestone payments and research funding support.

In connection with our collaboration with BMS, we have received an aggregate of $159.0 million in payments, which have primarily consisted of the initial upfront and amendment payments, development milestone payments, research funding support, and certain opt-in fees.

We no longer receive research funding support.

During the year ended December 31, 2025, we recognized $23.2 million of revenue related to our collaboration with BMS of which $9.7 million was previously deferred revenue.

As of December 31, 2024, we had $50.2 million of deferred revenue related to BMS, of which $50.2 million is classified as long-term on our consolidated balance sheet.

Under this collaboration, we will recognize revenue upon delivery of option packages to BMS or when milestones are achieved.

As such, we expect that our revenue will fluctuate from quarter-to-quarter and year-to-year as a result of the timing of when these events occur.

Pursuant to the license agreement with Vertex, we received a $50.0 million upfront cash payment in the fourth quarter of 2023 upon execution of the agreement and the 2024 annual license fee of $10.0 million in the first quarter of 2024.

For the year ended, December 31, 2024, we have recorded $10.0 million of revenue related to the agreement.

As of December 31, 2025, we had $40.5 million of deferred revenue related to BMS, all of which is classified as long-term deferred revenue on our consolidated balance sheet.

Under this collaboration, we recognize revenue upon delivery of option packages to BMS or upon receipt of development milestone payments.

We expect that our revenue will fluctuate from quarter-to-quarter and year-to-year as a result of the timing of when we deliver such option packages or receive such milestone payments.

Pursuant to the Vertex License Agreement, we received a $50.0 million upfront cash payment in the fourth quarter of 2023 upon execution of the agreement and the 2024 and 2025 annual license fees of $10.0 million in each of the first quarters of 2024 and 2025.

The license agreement further provides for the payment by Vertex of a potential additional $50.0 million contingent upfront payment and further annual license fees, ranging from $5.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.

For the year ended, December 31, 2025, we recorded $10.0 million of revenue related to the annual license fee under the agreement.

For additional information about our revenue recognition policy related to the Vertex License Agreement and BMS collaboration, see Part II, Item 7 “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Estimates—Revenue Recognition” included in this Annual Report on Form 10-K.

For the foreseeable future we expect substantially all of our revenue will be generated from our license agreements with Vertex and Vor Bio, collaboration with BMS, and any other collaborations or license agreements we may enter into.

For the foreseeable future we expect substantially all of our revenue will be generated from the Vertex License Agreement, our collaboration with BMS, and any other collaborations or license agreements we may enter into.

Operating Expenses

Research and Development Expenses

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These costs are expensed as incurred and include:

• costs associated with our continued development of EDIT-401 as we progress EDIT-401 to IND and/or foreign equivalent submission and commence clinical trials;

• employee-related expenses including salaries, benefits, and stock-based compensation expense;

• costs incurred under clinical trial agreements with investigative sites for our former reni-cel program;

• costs associated with conducting our preclinical, process and scale-up development, manufacturing, quality, clinical and regulatory activities, including fees paid to third-party professional consultants, service providers and suppliers;

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• costs associated with conducting our other preclinical, process and scale-up development, manufacturing, quality, clinical and regulatory activities, including fees paid to third-party professional consultants, service providers and suppliers;

• costs of purchasing lab supplies and non-capital equipment used in our preclinical activities and in manufacturing preclinical and clinical study materials;

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• successful completion of preclinical studies, IND-enabling studies and natural history studies;

• successful enrollment in, and completion of, clinical trials;

• successful initiation of, enrollment in, and completion of, clinical trials;

• receipt of marketing approvals from applicable regulatory authorities;

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Research and development activities are central to our business model.

We expect research and development costs to increase significantly for the foreseeable future as our development programs progress, including as we continue to support preclinical studies and prepare for the clinical development of our research programs.

We expect research and development expenses to decrease in future periods compared to prior periods, due to the discontinuation of clinical development of our ex vivo reni-cel program that contributed significantly to expense in prior periods.

General and Administrative Expenses

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Other significant costs include corporate facility costs not otherwise included in research and development expenses, legal fees related to intellectual property and corporate matters, and fees for accounting and consulting services.

We anticipate that our general and administrative expenses will decrease or remain flat in the near future to support continued research and development activities.

We anticipate that expenses associated with operating as a public company, including costs for audit, legal, regulatory, and tax-related services, director and officer insurance premiums, and investor relation costs will remain flat or decrease in the near future.

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We anticipate that our general and administrative expenses that support continued research and development activities will decrease in the near future.

With respect to reimbursement of third-party intellectual property-related expenses specifically, given the ongoing nature of the opposition and interference proceedings involving the patents licensed to us under our license agreement with Broad and Harvard, we anticipate general and administrative expenses associated with reimbursement of third-party intellectual property-related expense will continue to fluctuate as the interference proceedings continue.

Restructuring Charges

In December 2024, our board of directors approved the discontinuation of the clinical development of our ex vivo reni-cel program.

As part of the discontinuation, our board approved a reduction in our employee workforce by approximately 180 positions, or by approximately 65%.

Restructuring charges consist primarily of expenses in connection with the wind-down of various activities related to clinical development of reni-cel, including contract termination costs, impairment charges and non-cash charges, and expenses related to the employee workforce reduction, primarily consisting of severance payments and employee benefit costs.

We may also incur additional costs not currently contemplated due to events that may occur as a result of or that are associated with the discontinuation and headcount reduction.

We estimate that we will incur approximately $45.0 million to $55.0 million in cash costs to implement the discontinuation and workforce reduction.

We expect restructuring charges to be substantially incurred through the end of June 2025, when the related activities are expected to be substantially complete.

Restructuring and Impairment Charges

In December 2024, our board of directors approved the discontinuation of the clinical development of our ex vivo reni-cel program (the “Discontinuation”).

As part of the Discontinuation, our Board of Directors approved a reduction in our employee workforce by approximately 180 positions, or by approximately 65% (the “Reduction”).

Restructuring charges associated with the Discontinuation consist primarily of expenses in connection with the wind-down of various activities related to clinical development of reni-cel, including contract termination costs, impairment charges and non-cash charges, and expenses related to the Reduction, primarily consisting of severance payments and employee benefit costs.

The actions associated with the Discontinuation and Reduction commenced in December 2024 and were substantially completed by December 31, 2025.

Other Income (Expense), Net

For the years ended December 31, 2024, and 2023, other income (expense), net consisted primarily of changes in interest income and accretion of discounts associated with other marketable securities.

For the year ended December 31, 2025, other income (expense), net consisted primarily of interest income on cash and cash equivalents and marketable securities as well as interest expense accretion related to the liability for the sale of future revenues.

For the year ended December 31, 2024, other income (expense), net was primarily attributable to interest income and accretion of discounts associated with marketable securities.

Critical Accounting Policies and Estimates

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The effects of material revisions in estimates, if any, will be reflected in the consolidated financial statements prospectively from the date of change in estimates.

While our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K, we believe the following accounting policies used in the preparation of our consolidated financial statements requires the most significant judgments and estimates.

Revenue Recognition

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We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

As part of the accounting for these arrangements, we must develop assumptions that require judgment to determine the standalone selling price for each performance obligation identified in the contract and use judgement in the determination of the transaction price and the application of the constraint.

As part of the accounting for these arrangements, we must develop assumptions that require judgment to determine the standalone selling price for each performance obligation identified in the contract and use judgment in the determination of the transaction price and the application of the

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The determination of standalone selling price has not had a significant impact on the accounting for our revenue arrangements given the nature of the performance obligations.

We have also not been required to apply significant judgement in determining the transaction price given the nature of the variable consideration and the application of the constraint.

We have also not been required to apply significant judgment in determining the transaction price given the nature of the variable consideration and the application of the constraint.

Accrued Research and Development Expenses

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To date, there have been no material differences from our estimates to the amounts actually incurred.

Restructuring

We record liabilities for costs associated with restructuring activities in the period in which the liability is incurred.

Typical costs associated with restructuring activities include employee termination benefits, contract termination costs and on-going contract costs for which there is no economic benefit.

For costs associated with employee terminations in which the employee is subject to an existing benefit arrangement, the post-employment benefits are recognized when probable and estimable.

Other employee termination costs are measured and recognized on the communication date, unless there is a required future service period, in which case, the expense is recognized over the service period.

Contract termination costs are recognized upon termination of the contract and costs for on-going contracts for which there is no future benefit are recognized at fair value on the cease-use date.

We have made estimates and judgments regarding the amount and timing of our restructuring expense and liability, including current and future period termination benefits and other exit costs to be incurred when related actions take place.

Restructuring charges are reflected in our consolidated statements of operations.

Actual results may differ from these estimates.

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Results of Operations

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General and administrative 49,903 71,987 (22,084) (31) %

Restructuring charges 12,232 — 12,232 100 %

Restructuring and impairment charges 60,674 12,232 48,442 n/m

Total operating expenses 200,530 283,466 (82,936) (29) %

Operating loss (160,010) (251,152) 91,142 (36) %

Other income, net:

Other expense, net (3) (1,604) 1,601 n/m

Other income (expense), net:

Interest expense related to sale of future revenues (6,171) (2,190) (3,981) n/m

Interest income, net 8,310 16,252 (7,942) (49) %

Total other income, net 14,059 15,962 (1,903) (12) %

Other expense, net (2,189) (3) (2,186) n/m

Total other income (expense), net (50) 14,059 (14,109) n/m

Net loss $ (160,060) $ (237,093) $ 77,033 32 %

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Collaboration and Other Research and Development Revenues

Collaboration and other research and development revenues decreased by $45.8 million, to $32.3 million for the year ended December 31, 2024, from $78.1 million for the year ended December 31, 2023.

The decrease was primarily attributable to revenue recognized in 2023 under our license agreement with Vertex, which was executed in December 2023.

Collaboration and other research and development revenues increased by $8.2 million, to $40.5 million for the year ended December 31, 2025, from $32.3 million for the year ended December 31, 2024.

The increase was attributable to recognition of the remaining deferred revenue upon the conclusion of a collaboration agreement with a strategic partner, as well as recognition of revenue related to a milestone achieved in 2025 under our collaboration with BMS.

Research and Development Expenses

Research and development expenses increased by $21.5 million, to $199.2 million for the year ended December 31, 2024 from $177.7 million for the year ended December 31, 2023.

Research and development expenses decreased by $109.2 million, to $90.0 million for the year ended December 31, 2025 from $199.2 million for the year ended December 31, 2024.

The following table summarizes our research and development expenses for the years ended December 31, 2025 and December 31, 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

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Total research and development expenses $ 89,953 $ 199,247 $ (109,294) (55) %

The increase in research and development expenses for the year ended December 31, 2024 compared to the year ended December 31, 2023 was primarily attributable to:

• approximately $25.1 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the progression of our former reni-cel program as well as costs attributable to in vivo research and discovery;

• approximately $6.9 million in increased employee related expenses related to increased headcount;

• approximately $5.0 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space in the second quarter of 2024;

• approximately $1.9 million in increased other expenses attributable to consulting and external fees to support clinical and quality initiatives.

These increases were partially offset by the following decreases in research and development expenses:

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The decrease in research and development expenses for the year ended December 31, 2025 compared to the year ended December 31, 2024 was primarily attributable to:

• approximately $51.2 million in decreased external research and development expenses primarily resulting from reduced clinical and manufacturing costs due to the Discontinuation, partially offset by costs attributable to in vivo research and discovery;

• approximately $24.0 million in decreased employee related expenses related to reduced headcount associated with the Reduction;

• approximately $11.5 million in decreased sublicense and license fees related to reduced licensing activity in 2025 compared to 2024;

• approximately $1.2 million in decreased stock-based compensation expense due primarily to a reduction in the market price of our common stock, resulting in a lower valuation of equity awards granted.

• approximately $11.1 million in decreased facility expenses primarily due to the end of leases for manufacturing space due to the Discontinuation;

• approximately $5.8 million in decreased other expenses attributable to professional services to support our reni-cel program due to the Discontinuation;

• approximately $5.7 million in decreased stock-based compensation expense primarily related to expense in connection with the achievement of certain performance-based vesting milestones for restricted stock units recognized in 2024 for which there was no equivalent expense in 2025, a reduction in the market price of our common stock year-over-year resulting in lower fair value, and a reduction in headcount associated with the Reduction.

General and Administrative Expenses

General and administrative expenses increased by approximately $2.3 million, to $72.0 million for the year ended December 31, 2024 from $69.7 million for the year ended December 31, 2023.

General and administrative expenses decreased by approximately $22.1 million, to $49.9 million for the year ended December 31, 2025 from $72.0 million for the year ended December 31, 2024.

The following table summarizes our general and administrative expenses for the years ended December 31, 2025 and December 31, 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

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Total general and administrative expenses $ 49,903 $ 71,987 $ (22,084) (31) %

The increase in general and administrative expenses for the year ended December 31, 2024 compared to the year ended December 31, 2023 was primarily attributable to:

• approximately $3.8 million in increased employee related expenses related to increased headcount;

• approximately $2.8 million in increased stock-based compensation expense due primarily to the vesting of certain restricted stock units upon the achievement of performance-based milestones in 2024, along with an increased headcount;

• approximately $0.7 million in increased intellectual property and patent related fees for legal activity;

• approximately $0.3 million in increased facility and other expenses.

These increases were partially offset by approximately $5.3 million in decreased professional services expenses primarily related to one-time expenses incurred in 2023 to support strategic initiatives and business development activities.

The decrease in general and administrative expenses for the year ended December 31, 2025 compared to the year ended December 31, 2024 was primarily attributable to:

• approximately $8.6 million in decreased employee related expenses related to reduced headcount associated with the Reduction;

• approximately $6.0 million in decreased professional services expenses primarily related to reduced licensing and strategic business activities in 2025 relative to 2024;

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• approximately $4.6 million in decreased facility and other expenses primarily related to the end of a lease.

These decreases were partially offset by approximately $2.8 million in increased intellectual property and patent related fees for legal activity.

Restructuring Charges

Restructuring charges were $12.2 million for the year ended December 31, 2024, with no equivalent charges for the year ended December 31, 2023.

Restructuring charges increased by approximately $48.4 million, to $60.7 million for the year ended December 31, 2025, from $12.2 million for the year ended December 31, 2024.

The following table summarizes our restructuring charges for the years ended December 31, 2025 and December 31, 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

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Employee termination benefits $ 3,723 $ 10,475 $ (6,752) (64) %

Contract termination costs 1,757 — 1,757 100 %

Total restructuring charges $ 12,232 $ — $ 12,232 100 %

During the year ended December 31, 2024, we recorded $10.5 million and $1.8 million related to employee termination benefits and program-related contract termination costs, respectively, due to the discontinuation of the clinical development of our reni-cel program that we initiated in December 2024 and the related workforce reduction.

Total Other Income, Net

For the years ended December 31, 2024, and 2023, total other income, net was $14.1 million and $16.0 million, respectively, which was primarily attributable to interest income and accretion of discounts associated with marketable securities.

The decrease was attributable to reduction of investment balances offset by increased market rates.

Comparison of Years Ended December 31, 2023 and 2022

For a discussion of our results of operations for the year ended December 31, 2023 as compared to the year ended December 31, 2022, see Part II, Item 7, “Management's Discussion and Analysis of Financial Condition and Results of Operations” in our annual report on Form 10-K for the year ended December 31, 2023, which was filed with the U.S.

Securities and Exchange Commission (the “SEC”) on February 28, 2024.

Costs for ongoing contracts and terminated contracts 46,645 1,757 44,888 n/m

Acceleration of expense for change in useful life estimate and lease termination charges 6,548 — 6,548 100 %

Impairment charges 3,758 — 3,758 100 %

Total restructuring and impairment charges $ 60,674 $ 12,232 $ 48,442 100 %

The increase in restructuring and impairment charges for the year ended December 31, 2025 compared to the year ended December 31, 2024 was primarily attributable to reni-cel related contract costs recognized at the contract cease-use-date in 2025, accelerated expense recognized due to changes in useful life estimates for leasehold improvements, software, and a right-of-use asset, and impairment charges related to the sale of certain assets, resulting from the actions associated with the Discontinuation and the Reduction.

The increase was partially offset by decreased employee termination benefits due to the settlement of costs accrued as of December 31, 2024 during the year ended December 31, 2025.

Refer to Note 17, Restructuring and Impairment Charges, for further information.

Other Income (Expense), Net

For the years ended December 31, 2025, and 2024, other income (expense), net was $0.1 million, which was primarily attributable to interest income and interest accretion related to the liability for the sale of future revenues.

For the year ended December 31, 2024, other income (expense), net was $14.1 million, which was primarily attributable to interest income and accretion of discounts associated with marketable securities.

The decrease is attributable to the interest accretion related to the liability for the sale of future revenues and reductions in investment income due to a decrease in our investments.

Liquidity and Capital Resources

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As of December 31, 2025, we have raised an aggregate of $1.1 billion in net proceeds through the sale of shares of our common stock in public offerings and at-the-market offerings.

We also have funded our business from our research collaboration with BMS through its wholly owned subsidiary Juno Therapeutics, our former strategic alliance with Allergan (which was terminated in August 2020), payments received under the DRI Agreement in connection with our license agreement with Vertex, and payments under the Vertex license agreement.

We also have funded our business from our research collaboration with BMS, our former strategic alliance with Allergan, which was terminated in August 2020, payments received under the DRI Agreement in connection with the Vertex License Agreement, and payments under the Vertex License Agreement.

As of December 31, 2025, we had cash, cash equivalents and marketable securities of $146.6 million.

In May 2021, we entered into a common stock sales agreement with TD Securities (USA) LLC (as successor to Cowen and Company, LLC) (“TD Cowen”) under which we from time to time can issue and sell shares of our 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”).

We amended the common stock sales agreement with TD Cowen in February 2024 in connection with filing a new registration statement.

In March 2025, we further amended our common stock sales agreement with TD Cowen in connection with amending our existing shelf registration statement following the loss of our status as a “well-known seasoned issuer” (as defined under Rule 450 of the Securities Act of 1933, as amended), reducing the amount of shares of common stock we 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, we had not sold any shares of our common stock under the Prior ATM Facility or the ATM Facility.

In addition to our existing cash, cash equivalents and marketable securities, we are eligible to earn milestone and other payments under our collaboration agreement with BMS and our other collaboration and license agreements.

ability to earn applicable milestone and other payments and the timing of earning these amounts are dependent upon the timing and outcome of development, regulatory and commercial activities and, as such, are uncertain at this time.

As of December 31, 2024 , our right to contingent payments under our collaboration agreements with BMS and Vor Bio, as well as the retained portions of the contingent upfront payment and other amounts under our license agreement with Vertex, are our only significant committed potential external source of funds.

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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, 2025, we have sold 14,327,365 shares of our common stock under the ATM Facility at a weighted average price of $3.07 per share for aggregate gross proceeds of $43.9 million and have $106.1 million of shares of our common stock remaining available for sale under the ATM Facility.

In addition to our existing cash and cash equivalents, we are eligible to earn milestone and other payments under our collaboration with BMS and our other collaboration and license agreements.

Our ability to earn applicable milestone and other payments and the timing of earning these amounts are dependent upon the timing and outcome of development, regulatory and commercial activities and, as such, are uncertain at this time.

As of December 31, 2025 , our right to contingent payments under our collaboration with BMS, as well as the retained portions of the contingent upfront payment and other amounts under the Vertex License Agreement, are our only significant committed potential external source of funds.

The following table provides information regarding our cash flows for the years ended December 31, 2025 and 2024, respectively (in thousands):

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The use of cash in all periods resulted primarily from our net losses adjusted for non-cash charges and changes in components of working capital.

Net cash used in operating activities was approximately $210.3 million for the year ended December 31, 2024, which primarily consisted of operating expenses that related to increasing our research efforts, the progression of clinical and manufacturing activities in support of our former reni-cel program and supporting business operations.

Net cash used in operating activities was approximately $132.2 million for the year ended December 31, 2023, which primarily consisted of operating expenses related to the focused progression of clinical and manufacturing activities in support of our former reni-cel program, sublicense and license fees, and supporting business operations.

Net Cash Provided by (Used in) Investing Activities

Net cash used in operating activities was approximately $165.2 million for the year ended December 31, 2025, which primarily consisted of operating expenses related to our ongoing research and pre-clinical efforts, the wind-down of clinical and manufacturing activities related to our former reni-cel program and supporting business operations.

Net cash used in operating activities was approximately $210.3 million for the year ended December 31, 2024, which primarily consisted of operating expenses related to increasing our research efforts, the progression of clinical and manufacturing activities in support of our former reni-cel program and supporting business operations.

Net Cash Provided by Investing Activities

Net cash provided by investing activities was approximately $138.7 million for the year ended December 31, 2025, primarily related to maturities of marketable securities of $139.0 million and the proceeds from the sale of property and equipment of $0.3 million.

This was offset by the purchases of property and equipment of $0.6 million.

Net cash provided by investing activities was approximately $162.1 million for the year ended December 31, 2024, primarily related to maturities of marketable securities of $257.2 million.

This was offset by $86.2 million of purchases of marketable securities and purchases of property and equipment of $8.8 million.

Net cash used in investing activities was approximately $3.7 million for the year ended December 31, 2023, primarily related to the purchase of property and equipment of $4.7 million.

The remaining offset related to maturities of marketable securities of $259.5 million and purchases of marketable securities of $258.5 million.

Net Cash Provided by Financing Activities

Net cash provided by financing activities was approximately $40.5 million for the year ended December 31, 2025 primarily related to net proceeds from issuance of common stock from our at-the-market offering program of $42.8 million, proceeds received from issuance of common stock under our employee stock purchase plan of $0.4 million, and proceeds from the exercise of stock options of $0.1 million.

This was offset by the repayment on the sale of future revenues of $2.9 million.

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Net cash provided by financing activities was approximately $56.0 million for the year ended December 31, 2024, primarily related to net proceeds received from the sale of future revenue of $55.2 million, proceeds received from issuance of common stock under our employee stock purchase plan of $0.6 million and proceeds from the exercise of stock options of $0.2 million.

Net cash provided by financing activities was approximately $118.0 million for the year ended December 31, 2023, primarily related to proceeds received from the public offering of common stock, net of issuance costs, of $117.1 million in June 2023 and proceeds received from issuance of common stock under our employee stock purchase plan of $0.9 million.

For a discussion of our cash flows for the year ended December 31, 2022, see Part II, Item 7, “Management's Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the year ended December 31, 2023, which was filed with the SEC on February 28, 2024.

Funding Requirements

We expect our expenses to decrease over the next few years due to the discontinuation of the reni-cel program, but we will continue to incur significant expenses as we continue to advance our research programs and our preclinical development activities;

seek to identify product candidates and additional research programs;

We expect expenses to decrease in future periods compared to prior periods, due to the Discontinuation.

Our expenses for the foreseeable future will support preclinical studies and prepare for the clinical development of EDIT-401;

commence clinical trials for EDIT-401;

continue our current research programs and our preclinical development of product candidates from our current research programs;

seek to identify additional product candidates and research programs;

initiate preclinical testing and clinical trials for other product candidates we identify and develop;

maintain, expand, and project our intellectual property portfolio, including reimbursing our licensors for expenses related to the intellectual property that we in-license from such licensors;

maintain, expand, and protect our intellectual property portfolio, including reimbursing our licensors for expenses related to the intellectual property that we in-license from such licensors;

and incur costs associated with operating as a public company.

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If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, reduce, or eliminate our research and development programs or future commercialization efforts.

We expect that our existing cash, cash equivalents and marketable securities on December 31, 2024, together with the retained portions of the payments payable under our license agreement with Vertex, will fund our operating expenses and capital expenditure requirements into the second quarter of 2027.

Our forecast of the period of time through which our existing cash and cash equivalents and investments will be adequate to support our operations is a forward-looking statement and involves significant risks and uncertainties.

We expect that our existing cash and cash equivalents on December 31, 2025 will enable us to fund our operating expenses and capital expenditure requirements into the third quarter of 2027.

Our forecast of the period of time through which our existing cash and cash equivalents will be adequate to support our operations is a forward-looking statement and involves significant risks and uncertainties.

We have based this forecast on assumptions that may prove to be wrong, and actual results could vary materially from our expectations, which may adversely affect our capital resources and liquidity.

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The amount and timing of future funding requirements, both near- and long-term, will depend on many factors, including, but not limited to :

• the restructuring costs associated with the discontinuation of reni-cel;

• the scope, progress, results, and costs of clinical trials, drug discovery, preclinical development, laboratory testing, and clinical or natural history study trials for other product candidates we develop;

• the costs of progressing the preclinical and clinical development of EDIT-401;

• the scope, progress, results, and costs of drug discovery, preclinical development, laboratory testing, and any clinical or natural history study trials for product candidates we develop;

• the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property and proprietary rights, and defending intellectual property-related claims;

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• our ability to establish and maintain healthcare coverage and adequate reimbursement for any product candidates for which we receive regulatory approval.

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Identifying potential product candidates and conducting preclinical studies and clinical trials is a time-consuming, expensive, and uncertain process that takes many years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales.

In addition, even if we successfully identify and develop product candidates and those are approved, we will require significant additional amounts in order to launch and commercialize our product candidates and may not achieve commercial success.

Our commercial revenues, if any, will be derived from sales of genomic medicines that we do not expect to be commercially available for years, if at all.

In addition, even if we successfully identify and develop product candidates that are approved, we will require significant additional amounts in order to launch and commercialize our product candidates and may not achieve commercial success.

Our commercial revenues, if any, will be derived from sales of genomic medicines that we do not expect to be commercially available for many years, if at all.

Accordingly, we will need to continue to rely on additional financing to achieve our business objectives.

Adequate additional financing may not be available to us on acceptable terms, or at all.

Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, licensing arrangements and future royalty sales.

To the extent that we raise additional capital through the sale of equity or convertible debt securities, our stockholders’ ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders.

Debt financing, if available, would result in increased fixed payment obligations and may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends.

If we raise funds through additional collaborations, strategic alliances, licensing arrangements, or future royalty sales with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or to grant licenses on terms that may not be favorable to us.

If we raise funds through additional collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or to grant licenses on terms that may not be favorable to us.

If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce, or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Contractual Obligations

As of December 31, 2024, we had non-cancelable operating leases with total future minimum lease payments of $39.9 million, of which $17.1 million will be payable in 2025.

As of December 31, 2025, we had operating leases with future minimum lease payments for a total of $20.5 million, of which $7.4 million will be payable in 2026.

These minimum lease payments exclude our share of the facility operating expenses, real-estate taxes and other costs that are reimbursable to the landlord under the leases.

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In September 2024, we modified the lease, and as a result of the modification the lease payments decreased and the notification period for the termination of the license and service agreement increased from 12 months’ prior written notice to 18 months’ prior written notice.

In January 2025, we gave our termination notice on the license and service agreement.

The license and service agreement will terminate in July 2026.

From January 2025 through July 2026, we will incur minimum lease payments of up to $11.1 million.

In October 2024, we entered into a purchase and sale agreement (the “Purchase and Sale Agreement”) with DRI Healthcare Acquisitions LP (the “DRI”) under which we sold, transferred, assigned, and conveyed to DRI certain future license fees and other payments (the “Purchased Receivables”) owed to us by Vertex Pharmaceuticals, Inc.

(“Vertex”) under the terms of a license agreement (the “License Agreement”) in exchange for an upfront cash payment by DRI to us 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 us under the 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 the $50.0 million contingent upfront payment that we may receive under the License Agreement, in each case after subtracting amounts owing by us to our licensors, The Broad Institute, Inc.

In January 2025, we gave our termination notice on the license and service agreement, which resulted in the end of the term of the agreement being July 2026, and $8.9 million of remaining payments owed.

In April 2025, we modified the lease to terminate on April 30, 2025 with a final fixed payment of $3.7 million.

In October 2024, we entered into the DRI Agreement under which we sold, transferred, assigned, and conveyed to DRI certain future license fees and other payments owed to us by Vertex under the Vertex License Agreement in exchange for an upfront cash payment by DRI to us of $57.0 million.

In addition, DRI is purchasing a mid-double-digit percentage of the $50.0 million contingent upfront payment that the Company may receive under the Vertex License Agreement, in each case after subtracting amounts owing by us to our licensors, The Broad Institute, Inc.

and the President and Fellows of Harvard College.

We have retained rights to our portions of certain other sales-based annual license fees and the contingent upfront payment that may become due under the License Agreement, and the amounts that correspond to our licensor obligations.

Our agreements with certain institutions to license intellectual property include potential milestone and success fees, sublicense fees, royalty fees, licensing maintenance fees, and reimbursement of patent maintenance costs that we may be required to pay.

Our agreements to license intellectual property include potential milestone payments that are dependent upon the development of products using the intellectual property licensed under the agreements and contingent upon the achievement of development or regulatory approval milestones, as well as commercial milestones.

These potential obligations are contingent upon the occurrence of future events and the timing and likelihood of such potential obligations are not known with certainty.

These potential obligations are contingent upon future events and the timing and likelihood of such potential obligations are not known with certainty.

For further information regarding these agreements, please see Part I, Item 1 “Business—Our Collaborations and Licensing Strategy” of this Annual Report on Form 10-K.

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We also enter into contracts in the normal course of business with contract research organizations, contract manufacturing organizations and other vendors to assist in the performance of our research and development activities and other services and products for operating purposes.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 7. Management’s Discussion and Analysis