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Item 2. Management’s Discussion and Analysis

2025-11-10 compared with 2025-08-12 · 121 added, 117 removed, 239 unchanged (50% of the section changed)

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The forward-looking statements contained in this Quarterly Report on Form 10-Q are made as of the date of this Quarterly Report on Form 10-Q, and we do not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law.

We are a pioneering gene editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases.

We are a pioneering gene editing company dedicated to developing transformative genomic medicines to treat a broad range of serious diseases.

We have developed a proprietary gene editing platform based on CRISPR technology and we continue to expand its capabilities.

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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 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” lipid nanoparticle (“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 will select the lead development candidate to advance towards human proof-of-concept in September 2025 and are on track to submit an investigational new drug application by mid-2026 and achieve human proof-of-concept by year-end 2026.

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 human proof of concept of our in vivo gene editing medicines in approximately two years.

However, leveraging our differentiated approach and the insights gained from these trials, we are pursuing next generation in vivo gene editing medicines targeting HSCs for the treatment of SCD and beta thalassemia.

In January 2025, we announced in vivo preclinical proof of concept of editing HSCs in non-human primates (“NHPs”), and in June 2025 we shared new in vivo preclinical data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in HSCs with a single dose of a novel targeted LNP (“tLNP”) in NHPs.

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

In May 2025, we shared preclinical proof-of-concept data for an undisclosed liver target using in vivo CRISPR editing to upregulate target protein expression and reduce a disease-associated biomarker in a relevant mouse model.

We previously announced in vivo delivery to two additional cell types in humanized mice using our proprietary LNP targeting platform and remain on track to establish and disclose a further target cell type or tissue by the end of 2025.

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 upregulate 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” lipid nanoparticle (“LNP”) platform that enables delivery of the gene editing cargo 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 hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.

This targeted approach has demonstrated a favorable preclinical profile in both efficacy data and tolerability and supports the potential of EDIT-401 to deliver meaningful clinical outcomes for patients underserved by current lipid-lowering therapies.

In October 2025, we reported in vivo preclinical proof-of-concept data for EDIT-401.

In preclinical studies in non-human primates (“NHPs”), EDIT-401 achieved LDL-C reductions equal to or exceeding 90% within 48 hours of a single dose of EDIT-401.

EDIT-401 also achieved LDL-C reductions equal to or exceeding 90% in mice with high baseline LDL-C and reduced LDLR function.

The LDL-C reductions were achieved with an at least six-fold mean increase in LDLR protein in the NHP liver, requiring only approximately 10-40% functional editing of LDLR alleles in the liver.

LDL-C reductions was maintained in mouse models in a three-month study, demonstrating the durability of effect.

We plan to submit an investigational new drug

application (“IND”) or clinical trial application (“CTA”) for EDIT-401 by mid-2026 with the goal of achieving initial in vivo human proof-of-concept data for EDIT-401 by the end of 2026.

We also 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”).

In January 2025, we announced in vivo preclinical proof of concept of editing HSCs in NHPs, and in June 2025 we shared new in vivo preclinical data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in HSCs with a single dose of a novel targeted LNP in NHPs.

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

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

We additionally intend to continue work on optimizing candidates for our HSC program and to continue 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.

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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.

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.

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 $129.3 million and $129.6 million for the six months ended June 30, 2025 and 2024, respectively.

As of June 30, 2025, we had an accumulated deficit of $1.6 billion.

“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 have incurred significant operating losses since inception.

Our net losses were $154.4 million and $191.7 million for the nine months ended September 30, 2025 and 2024, respectively.

As of September 30, 2025, we had an accumulated deficit of $1.6 billion.

We expect to continue to incur significant expenses and operating losses for the foreseeable future.

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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 licensors;

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 gene editing platform;

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We no longer receive research funding support.

During the three and six months ended June 30, 2025 and 2024 we did not recognize revenue related to our collaboration with BMS.

As of June 30, 2025, we had $50.2 million of deferred revenue in relation to our collaboration with BMS, all of which was classified as long-term deferred revenue on our consolidated balance sheet.

During the three months ended September 30, 2025, we recognized $ 7.5 million of revenue related to our collaboration with BMS, none of which was previously deferred.

We did not recognize any revenue related to our collaboration with BMS during the three months ended September 30, 2024.

As of September 30, 2025, we had $50.2 million of deferred revenue in relation to our collaboration with BMS, all of which was classified as long-term deferred revenue on our consolidated balance sheet.

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

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• 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;

• costs associated with our continued development of EDIT-401 as we progress EDIT-401 to IND or CTA submission;

• costs incurred as part of the discontinuation of our former reni-cel program;

• 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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This is due to the numerous risks and uncertainties associated with developing such product candidates, including the uncertainty of:

• successful completion of preclinical studies, investigational new drug application-enabling studies and natural history studies;

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

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

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

Restructuring charges consist

primarily of expenses in connection with the wind-down of various activities related to the 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.

Restructuring charges consist primarily of expenses in connection with the wind-down of various activities related to the 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.

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 Reduction.

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

Other Income, Net

For the six months ended June 30, 2025 and June 30, 2024, other income, net consisted primarily of interest income and the amortization of premiums or discounts on marketable securities and interest accretion related to the liability for the sale of future revenues.

Other (Expense) Income, Net

For the nine months ended September 30, 2025 and September 30, 2024, other (expense) income, net consisted primarily of interest income on cash and cash equivalents and interest accretion related to the liability for the sale of future revenues.

Critical Accounting Policies and Estimates

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

During the three months ended March 31, 2025, we had the following material change to our critical accounting estimates as described in Part II, “Item 7.

During the nine months ended September 30, 2025, we had the following material change to our critical accounting estimates as described in Part II, “Item 7.

Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Estimates” in the Annual Report:

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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 income.

Restructuring charges are reflected in our condensed consolidated statements of operations.

Actual results may differ from these estimates.

Results of Operations

Comparison of the Three Months ended June 30, 2025 and 2024

The following table summarizes our results of operations for the three months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Comparison of the Three Months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the three months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended

June 30, Dollar Change Percentage Change

September 30, Dollar Change Percentage Change

Collaboration and other research and development revenues $ 7,543 $ 61 $ 7,482 n/m

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Other (expense) income, net

Other expense, net (1,758) (1) (1,757) n/m

Interest related to sale of future revenues (2,020) — (2,020) 100 %

Other expense, net (1) (5) 4 (80) %

Interest expense related to sale of future revenues (2,399) — (2,399) 100 %

Interest income, net 1,831 3,530 (1,699) (48) %

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Collaboration and Other Research and Development Revenues

Collaboration and other research and development revenues were $3.6 million for the three months ended June 30, 2025 compared to $0.5 million for the same period in 2024.

The increase from the three months ended June 30, 2024 is primarily attributable to the recognition of revenue related to specified deliverables that were achieved in the second quarter of 2025.

Collaboration and other research and development revenues were $7.5 million for the three months ended September 30, 2025 compared to $0.1 million for the same period in 2024.

The increase from the three months ended September 30, 2024 is primarily attributable to the recognition of revenue related to a milestone achieved in the third quarter of 2025 under our collaboration with BMS.

Research and Development Expenses

Research and development expenses decreased by $38.0 million to $16.2 million for the three months ended June 30, 2025 compared to $54.2 million for the same period in 2024.

The following table summarizes our research and development expenses for the three months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Research and development expenses decreased by $27.9 million to $19.8 million for the three months ended September 30, 2025 compared to $47.6 million for the same period in 2024.

The following table summarizes our research and development expenses for the three months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended

June 30, Dollar Change Percentage Change

September 30, Dollar Change Percentage Change

Employee related expenses $ 6,468 $ 14,486 $ (8,018) (55) %

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Stock-based compensation expenses 634 2,048 (1,414) (69) %

Sublicense and license fees 95 857 (762) (89) %

Sublicense and license fees 1,292 307 985 n/m

Other expenses 1,662 3,511 (1,849) (53) %

Total research and development expenses $ 19,775 $ 47,639 $ (27,864) (58) %

The decrease in research and development expenses for the three months ended June 30, 2025 compared to the three months ended June 30, 2024 was primarily attributable to:

• approximately $24.4 million in decreased external research and development expenses primarily resulting from reduced clinical and manufacturing costs under our reni-cel program due to the Discontinuation, partially offset by costs attributable to in vivo research and discovery;

The decrease in research and development expenses for the three months ended September 30, 2025 compared to the three months ended September 30, 2024 was primarily attributable to:

• approximately $13.6 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 $8.0 million in decreased employee-related expenses related to reduced headcount associated with the Reduction;

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• approximately $1.8 million in decreased other expenses related to professional services to support our reni-cel program due to the Discontinuation;

• approximately $1.3 million in decreased stock-based compensation expense primarily related to reduction in the market price of common stock year-over-year, resulting in a lower valuation of equity awards, along with reduced headcount as a result of the Reduction;

• approximately $0.8 million in decreased sublicense and license fees.

• approximately $1.4 million in decreased stock-based compensation expense primarily related to reduction in the market price of our common stock year-over-year, resulting in a lower valuation of equity awards, along with reduced headcount as a result of the Reduction.

These decreases were partially offset by approximately $1.0 million in increased sublicense and license fees related to the achievement of certain milestones under our collaboration with BMS in the three months ended September 30, 2025 for which there was no similar activity in the three months ended September 30, 2024.

General and Administrative Expenses

General and administrative expenses decreased by $5.3 million to $12.9 million for the three months ended June 30, 2025 compared to $18.2 million for the three months ended June 30, 2024.

The following table summarizes our general and administrative expenses for the three months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

General and administrative expenses decreased by $5.8 million to $12.3 million for the three months ended September 30, 2025 compared to $18.1 million for the three months ended September 30, 2024.

The following table summarizes our general and administrative expenses for the three months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended

June 30, Dollar Change Percentage Change

September 30, Dollar Change Percentage Change

Employee related expenses $ 2,491 $ 5,902 $ (3,411) (58) %

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Total general and administrative expenses $ 12,316 $ 18,088 $ (5,772) (32) %

The decrease in general and administrative expenses for the three months ended June 30, 2025 compared to the three months ended June 30, 2024 was primarily attributable to:

The decrease in general and administrative expenses for the three months ended September 30, 2025 compared to the three months ended September 30, 2024 was primarily attributable to:

• approximately $3.4 million in decreased employee related expenses related to reduced headcount associated with the Reduction;

• approximately $2.2 million in decreased professional service expenses related to reduced licensing and strategic business activities;

• approximately $1.2 million in decreased stock-based compensation expenses primarily related to reduction in the market price of our common stock year-over-year, resulting in a lower valuation of equity awards, along with reduced headcount as a result of the Reduction;

• approximately $1.0 million in decreased facility and other expenses related to the end of a lease.

• approximately $1.1 million in decreased stock-based compensation expenses primarily related to reduction in the market price of common stock year-over-year, resulting in a lower valuation of equity awards, along with reduced headcount as a result of the Reduction;

• approximately $0.6 million in decreased intellectual property and patent related legal fees;

• approximately $0.3 million in decreased professional service expenses.

These decreases were partially offset by approximately $2.0 million in increased intellectual property and patent related legal fees.

Restructuring and Impairment Charges

Restructuring charges were $26.1 million for the three months ended June 30, 2025, with no equivalent charges for the three months ended June 30, 2024.

The following table summarizes our restructuring charges for the three months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended June 30, Dollar Change Percentage Change

Employee termination benefits $ 813 $ — $ 813 100 %

Costs for ongoing contracts and terminated contracts 23,466 — 23,466 100 %

Acceleration of expense for change in useful life estimate and lease termination 1,803 — 1,803 100 %

Total restructuring and impairment charges $ 26,082 $ — $ 26,082 100 %

During the three months ended June 30, 2025, we recorded $0.8 million, $23.5 million, and $1.8 million of expense related to employee termination benefits, reni-cel-related contract costs, and acceleration in expense due to changes in useful life estimates for leasehold improvements, software and a right of use asset, respectively, due to the Discontinuation and the Reduction.

Other Income, Net

For the three months ended June 30, 2025, and June 30, 2024 other (expense) income, net was $(1.7) million and $4.3 million, respectively, which primarily relate to interest income and accretion of discounts and premiums associated with marketable securities and interest accretion related to the liability for the sale of future revenues.

The decrease is attributable to interest accretion related to the DRI Purchase and Sale Agreement and reductions in investment income due to a decrease in our investments for the three months ended June 30, 2025 compared to the three months ended June 30, 2024.

Comparison of the Six Months ended June 30, 2025 and 2024

The following table summarizes our results of operations for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Six Months Ended

June 30, Dollar Change Percentage Change

During the three months ended September 30, 2025 and September 30, 2024, we recorded no expense related to restructuring and impairment, respectively, due to the Discontinuation and the Reduction.

Other (Expense) Income, Net

For the three months ended September 30, 2025, and September 30, 2024 other (expense) income, net was $(0.6) million and $3.5 million, respectively, which primarily relate to interest income and accretion of discounts and premiums associated with marketable securities and interest accretion related to the liability for the sale of future revenues.

The decrease is attributable to interest accretion related to the DRI Purchase and Sale Agreement and reductions in investment income due to a decrease in our investments for the three months ended September 30, 2025 compared to the three months ended September 30, 2024.

Comparison of the Nine Months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Nine Months Ended

September 30, Dollar Change Percentage Change

Collaboration and other research and development revenues $ 15,779 $ 1,710 $ 14,069 n/m

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Other (expense) income, net

Other (expense) income, net (2,183) 5 (2,188) n/m

Interest related to sale of future revenues (4,236) — (4,236) 100 %

Other expense, net (2,184) — (2,184) 100 %

Interest expense related to sale of future revenues (6,635) — (6,635) 100 %

Interest income, net 6,634 12,861 (6,227) (48) %

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Collaboration and Other Research and Development Revenues

Collaboration and other research and development revenues increased by $6.6 million to $8.2 million for the six months ended June 30, 2025 compared to $1.6 million for the six months ended June 30, 2024.

Collaboration and other research and development revenues increased by $14.1 million to $15.8 million for the nine months ended September 30, 2025 compared to $1.7 million for the nine months ended September 30, 2024.

The increase was primarily attributable to the recognition of the remaining deferred revenue upon the closing out of a collaboration agreement with a strategic partner, and the recognition of revenue related to specified deliverables under that collaboration agreement, as well as recognition of revenue related to a milestone achieved in the third quarter of 2025 under our collaboration with BMS.

Research and Development Expenses

Research and development expenses decreased by $60.2 million to $42.8 million for the six months ended June 30, 2025 compared to $103.0 million for the six months ended June 30, 2024.

The following table summarizes our research development expenses for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Six Months Ended

June 30, Dollar Change Percentage Change

Research and development expenses decreased by $88.1 million to $62.5 million for the nine months ended September 30, 2025 compared to $150.6 million for the nine months ended September 30, 2024.

The following table summarizes our research development expenses for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Nine Months Ended

September 30, Dollar Change Percentage Change

Employee related expenses $ 25,856 $ 41,830 $ (15,974) (38) %

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Total research and development expenses $ 62,549 $ 150,636 $ (88,087) (58) %

The decrease in research and development expenses for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was primarily attributable to:

• approximately $34.5 million in decreased external research and development expenses primarily resulting from reduced clinical and manufacturing costs under our reni-cel program due to the Discontinuation, partially offset by costs attributable to in vivo research and discovery;

The decrease in research and development expenses for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 was primarily attributable to:

• approximately $48.1 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 $16.0 million in decreased employee-related expenses related to reduced headcount associated with the Reduction;

• approximately $6.6 million in decreased sublicense and license fees related to the achievement of certain milestones in 2024 for which there was no equivalent achievement in 2025;

• approximately $4.0 million in decreased other expenses related to professional services to support our reni-cel program due to the Discontinuation;

• approximately $7.8 million in decreased facility expenses primarily due to the end of leases for manufacturing space;

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

• approximately $5.7 million in decreased sublicense and license fees related to the achievement of certain milestones in the first quarter of 2024 for which there was no equivalent expense in 2025;

• approximately $4.7 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 decreased by $11.3 million to $26.2 million for the six months ended June 30, 2025 compared to $37.5 million for the six months ended June 30, 2024.

The following table summarizes our general and administrative expenses for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Six Months Ended

June 30, Dollar Change Percentage Change

General and administrative expenses decreased by $17.1 million to $38.6 million for the nine months ended September 30, 2025 compared to $55.6 million for the nine months ended September 30, 2024.

The following table

summarizes our general and administrative expenses for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Nine Months Ended

September 30, Dollar Change Percentage Change

Employee related expenses $ 10,065 $ 15,594 $ (5,529) (35) %

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Total general and administrative expenses $ 38,550 $ 55,633 $ (17,083) (31) %

The decrease in general and administrative expenses for the six months ended June 30, 2025 compared to the six months ended June 30, 2024 was primarily attributable to:

• approximately $3.7 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;

• approximately $2.6 million in decreased facility and other expense primarily related to the end of a lease;

The decrease in general and administrative expenses for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024 was primarily attributable to:

• approximately $5.5 million in decreased employee related expenses related to reduced headcount associated with the Reduction;

• approximately $2.0 million in decreased intellectual property and patent related fees due to reduced legal activity related to intellectual property defense;

• approximately $4.8 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;

• approximately $3.9 million in decreased professional service expenses related to reduced licensing and strategic business activities in 2025 relative to 2024;

• approximately $3.5 million in decreased facility and other expense primarily related to the end of a lease.

The decreases were partially offset by approximately $0.8 million in increased intellectual property and patent related fees.

Restructuring and Impairment Charges

Restructuring charges were $66.9 million for the six months ended June 30, 2025, with no equivalent charges for the six months ended June 30, 2024.

The following table summarizes our restructuring charges for the six months ended June 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Six Months Ended June 30, Dollar Change Percentage Change

Restructuring charges were $66.9 million for the nine months ended September 30, 2025, with no equivalent charges for the nine months ended September 30, 2024.

The following table summarizes our restructuring charges for the nine months ended September 30, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Nine Months Ended September 30, Dollar Change Percentage Change

Employee termination benefits $ 4,322 $ — $ 4,322 100 %

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Total restructuring and impairment charges $ 66,935 $ — $ 66,935 100 %

During the six months ended June 30, 2025, we recorded $4.3 million, $52.3 million, $6.5 million and $3.7 million related to employee termination benefits, reni-cel-related contract costs, acceleration in expense due to changes in useful life estimates for leasehold improvements, software and a right of use asset, and impairment charges, respectively, due to the Discontinuation and the Reduction.

Other Income, Net

For the six months ended June 30, 2025 and June 30, 2024, other (expense) income, net was $(1.6) million and $9.3 million, respectively, which primarily relates to interest income and accretion of discounts and premiums associated with marketable securities and interest accretion related to the liability for the sale of future revenues.

The decrease for the six months ended June 30, 2025 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.

During the nine months ended September 30, 2025, we recorded $4.3 million, $52.3 million, $6.5 million and $3.7 million related to employee termination benefits, reni-cel-related contract costs, acceleration in expense due to changes in

useful life estimates for leasehold improvements, software and a right of use asset, and impairment charges, respectively, due to the Discontinuation and the Reduction.

Other (Expense) Income, Net

For the nine months ended September 30, 2025 and September 30, 2024, other (expense) income, net was $(2.2) million and $12.9 million, respectively, which primarily relates to interest income and accretion of discounts and premiums associated with marketable securities and interest accretion related to the liability for the sale of future revenues.

The decrease for the nine months ended September 30, 2025 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

Sources of Liquidity

As of June 30, 2025, we have raised an aggregate of $1.0 billion in net proceeds through the sale of shares of our common stock in public offerings and at-the-market offerings .

As of September 30, 2025, we have raised an aggregate of $1.0 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.

As of June 30, 2025, we had cash, cash equivalents and marketable securities of $178.5 million.

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $165.6 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.

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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 405 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 June 30, 2025, we have sold 4,126,072 shares of our common stock under the ATM Facility at a weighted average price of $2.14 per share for aggregate gross proceeds of $8.6 million and have $141.4 million of shares of our common stock remaining available for issuance and sale under 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.

As of September 30, 2025, we have sold 9,925,250 shares of our common stock under the ATM Facility at a weighted average price of $2.66 per share for aggregate gross proceeds of $26.4 million and have $123.6 million of shares of our common stock remaining available for issuance and sale under the ATM Facility.

As of November 10, 2025, the issuance date of the condensed consolidated financial statements, we have sold an incremental 4,327,483 shares of our common stock under the ATM Facility at a weighted average price of $4.00 per share for gross proceeds of $17.3 million and have $106.3 million of shares of our common stock remaining available for issuance and sale under 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 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 June 30, 2025, our right to contingent payments under our collaboration agreement with BMS, 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.

The following table provides information regarding our cash flows for the six months ended June 30, 2025 and 2024 (in thousands):

Six Months Ended

As of September 30, 2025, our right to contingent payments under our collaboration agreement with BMS, 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.

The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024 (in thousands):

Nine Months Ended

September 30,

Net cash (used in) provided by:

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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 $98.0 million for the six months ended June 30, 2025, which primarily consisted of operating expenses that related to our ongoing research efforts, the wind-down 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 $106.9 million for the six months ended June 30, 2024, which primarily consisted of operating expenses that related to increasing our research efforts, the focused 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 $128.9 million for the nine months ended September 30, 2025, which primarily consisted of operating expenses that related to our ongoing research and pre-clinical efforts, the wind-down 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 $159.5 million for the nine months ended September 30, 2024, which primarily consisted of operating expenses that related to increasing our research efforts, the focused 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 $99.0 million for the six months ended June 30, 2025, primarily related to the proceeds from the maturities of marketable securities of $99.0 million.

Net cash provided by investing activities was approximately $47.2 million for the six months ended June 30, 2024, primarily related to the proceeds from the maturities of marketable securities of $138.8 million, and partially offset by the purchase of marketable securities of $86.2 million, and the purchase of property and equipment of $5.4 million.

Net cash provided by investing activities was approximately $138.6 million for the nine months ended September 30, 2025, primarily related to the proceeds from the maturities of marketable securities of $139.0 million and proceeds from the sale of property and equipment of $0.2 million, partially offset by the purchase of property and equipment of $0.5 million.

Net cash provided by investing activities was approximately $131.2 million for the nine months ended September 30, 2024, primarily related to the proceeds from the maturities of marketable securities of $225.7 million, and partially offset by the purchase of marketable securities of $86.2 million and the purchase of property and equipment of $8.3 million.

Net Cash Provided by Financing Activities

Net cash provided by financing activities was approximately $6.0 million for the six months ended June 30, 2025, primarily related to proceeds from the issuance of common stock from our at the market offering program of $8.6 million and proceeds received from issuance of common stock under our employee stock purchase plan of $0.2 million.

This was offset by the repayment of our liability for the sale of future revenues with DRI of $2.9 million.

Net cash provided by financing activities was approximately $0.5 million for the six months ended June 30, 2024, related to the proceeds received from issuance of common stock under our employee stock purchase plan of $0.3 million and exercises of options for our common stock of $0.2 million.

Net cash provided by financing activities was approximately $23.1 million for the nine months ended September 30, 2025, primarily related to net proceeds from the issuance of common stock from our at-the-market offering program of $25.8 million and proceeds received from issuance of common stock under our employee stock purchase plan of $0.2 million.

This was partially offset by the repayment of our liability for the sale of future revenues with DRI of $2.9 million.

Net cash provided by financing activities was approximately $0.5 million for the nine months ended September 30, 2024, related to the proceeds received from issuance of common stock under our employee stock purchase plan of $0.3 million and exercises of options for our common stock of $0.2 million.

Funding Requirements

We expect our expenses to increase 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;

seek to identify product candidates and additional research programs;

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

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;

and incur costs

associated with operating as a public company.

and incur costs associated with operating as a public company.

In addition, if we obtain marketing approval for any product candidate that we identify and develop, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing, and distribution to the extent that such sales, marketing, and distribution are not the responsibility of a collaborator.

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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 June 30, 2025, and the retained portions of the payments payable under our license agreement with Vertex, will enable us to fund our operating expenses and capital expenditure requirements into the second quarter of 2027 .

We expect that our existing cash and cash equivalents on September 30, 2025, together with the proceeds of sales of shares of common stock under our ATM Facility after September 30, 2025 through the date of this report and the retained portions of the payments payable under our license agreement with Vertex, 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 and investments will be adequate to support our operations is a forward-looking statement and involves significant risks and uncertainties.

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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, and licensing arrangements.

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.

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.

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Contractual Obligations

As of June 30, 2025, we had operating leases with future minimum lease payments for a total of $24.4 million, of which $3.9 million will be payable in 2025.

As of September 30, 2025, we had operating leases with future minimum lease payments for a total of $22.4 million, of which $2.0 million will be payable in 2025.

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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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-Q

Item 2. Management’s Discussion and Analysis