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Item 2. Management’s Discussion and Analysis

2025-05-12 compared with 2024-11-04 · 143 added, 189 removed, 148 unchanged (69% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations.

The following discussion and analysis of our financial condition and results of operations should be read together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the fiscal year ended December 31, 2023, which was filed with the Securities and Exchange Commission (“SEC”) on February 28, 2024 (the “Annual Report”).

The following discussion and analysis of our financial condition and results of operations should be read together with our condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which was filed with the Securities and Exchange Commission (“SEC”) on March 5, 2025 (the “Annual Report”).

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties.

The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

All statements addressing our future operating performance and clinical development and regulatory timelines that we expect or anticipate will occur in the future, as well as expectations for cash runway, are forward-looking statements.

There are a number of important risks and uncertainties that could cause our actual results to differ materially from those indicated by forward-looking statements, including uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of our product candidates;

availability and timing of results from pre-clinical studies and clinical trials;

whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials;

All statements addressing our future operating performance and development timelines that we expect or anticipate will occur in the future, as well as expectations for cash runway, are forward-looking statements.

availability and timing of results from pre-clinical studies;

expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements.

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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 clinical stage 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 potentially 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.

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 advancing gene editing medicines to treat hemoglobinopathies, beginning with the continued development of our current ex vivo renizgamglogene autogedtemcel (“reni-cel”) (formerly known as EDIT-301) program and leveraging the insights gained from this program to pursue next generation in vivo gene editing medicines targeting hematopoietic stem cells (“HSCs”).

We are also pursuing the development of in vivo gene editing medicines for other organs and tissues that we believe will significantly differentiate our gene editing approach from the current standards of care for serious diseases.

As part of these efforts, we are using strategic partnerships and collaborations and pursuing further opportunities to extend the reach of our intellectual property portfolio and access complementary technologies to expedite our drug discovery and clinical execution objectives.

Our lead program, reni-cel, is an experimental ex vivo gene-edited medicine to treat sickle cell disease (“SCD”), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (“TDT”), the most severe form of beta-thalassemia, another inherited blood disorder characterized by severe anemia.

We are investigating reni-cel in a single Phase 1/2/3 clinical trial, referred to as the RUBY trial, for the treatment of severe SCD.

In 2022, we dosed the first patient in the RUBY trial, and after completing sequential dosing of the first two patients, we commenced concurrent patient dosing in the first quarter of 2023.

We continue to dose adult patients and have dosed 28 patients as of the date of this Quarterly Report on Form 10-Q.

We previously completed enrollment of the adult and adolescent cohorts and continue to manufacture drug product and schedule dosing for the initial adolescent cohort patients.

Based on our discussions to date with the U.S.

Food and Drug Administration (the “FDA”), we expect any future biologics license application package for reni-cel for the treatment of severe SCD to be similar in number of patients and duration of follow-up to the package submitted for a previously FDA-approved gene editing medicine for severe SCD.

In December 2021, the FDA cleared our Investigational New Drug (“IND”) application for a Phase 1/2 clinical trial of reni-cel for the treatment of TDT, which we refer to as our EdiTHAL trial.

We dosed the first patient in this trial in the first quarter of 2023 and commenced concurrent patient dosing in the second quarter of 2023.

We have completed enrollment of the adult cohort in the trial and continue to dose patients.

In June 2024, we presented new safety and efficacy data as of a May 8, 2024 data cutoff date in 18 patients treated with reni-cel in the RUBY clinical trial and in seven patients treated with reni-cel in the EdiTHAL clinical trial.

We are on track to present additional safety and efficacy clinical data, including hematology parameters, vaso-occlusive events, and patient reported outcomes, from 28 patients in the RUBY trial in December 2024 at the American Society of Hematology Annual Meeting and Exposition, as well as additional clinical data from the EdiTHAL trial by year-end 2024.

In October 2024, we announced that we have initiated a global process to partner or out-license reni-cel to focus our resources on in vivo pipeline development.

We are also pursuing the development of next generation in vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body.

We are initially focused on editing HSCs through targeted delivery of our AsCas12a enzyme to our clinically validated HBG1 and HBG2 promotor site.

Our internal development efforts leverage our indel CRISPR technology we use to upregulate the expression of the gamma globin gene, a functional homolog of the beta globin gene, through direct editing of the HBG1/2 promotor site in our ex vivo reni-cel program.

Using this approach, we are pursuing an in vivo strategy aimed at driving functional upregulation of gene expression to address loss of function or deleterious mutations in genetic diseases in rare and orphan disease patient populations, from which we intend to expand to diseases with larger patient populations.

We are utilizing lipid nanoparticles for delivery of gene editing cargo into multiple tissue types, while also evaluating additional, next generation delivery technology.

In October 2024, we announced that we achieved in vivo preclinical proof of concept of HBG1/2 editing in hematopoietic stem and progenitor cells (“HSPC”) and fetal hemoglobin (“HbF”) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.

Utilizing a hematopoietic stem cell targeting strategy and a novel, Editas-proprietary targeted lipid nanoparticle (“tLNP”) for extrahepatic tissue delivery, we enabled an editing level of 29% of HSPCs after a single dose.

Editing with our proprietary tLNP formulation resulted in the functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month.

We expect to provide an update on our in vivo progress and pipeline development in the first quarter of 2025.

We are pursuing the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies, while also leveraging our intellectual property portfolio to drive potential out-licensing and partnership discussions that can accelerate the achievement of our goal of delivering lifesaving medicines to patients with previously untreatable diseases.

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 CASGEVYTM (exagamglogene autotemcel).

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 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, leveraging 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 LNP (“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 an in vivo development candidate via gene upregulation in HSCs 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 tLNP.

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 an in vivo development candidate via gene upregulation 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 preclinical HSC data and in vivo preclinical data in one liver indication at the American Society of Gene and Cell Therapy annual meeting in May 2025.

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.

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.

(“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).

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 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 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.

We are required to pay The Broad Institute, Inc.

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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.

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 Cas9 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.

In cellular therapy medicines, we are leveraging partnerships to progress engineered cell medicines to treat various cancers.

We are advancing alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors in 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, has resulted in 13 total programs.

In March 2024, we entered into an amendment to extend the collaboration to November 2026, with options to extend the collaboration for up to an additional two years, and provided BMS the ability to select up to three new gene targets for research.

We are also party to a non-exclusive collaboration and licensing agreement with Immatics N.V.

to combine gamma-delta T cell adoptive cell therapies and gene editing to develop medicines for the treatment of cancer.

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.

Except for reni-cel, 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.

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, our former strategic alliance with Allergan Pharmaceuticals International Limited (together with its affiliates, “Allergan”), which was terminated in August 2020, and payments received in connection with our license agreement with Vertex.

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 $191.7 million and $134.3 million for the nine months ended September 30, 2024 and 2023, respectively.

As of September 30, 2024, we had an accumulated deficit of $1.4 billion.

Our net losses were $76.1 million and $62.0 million for the three months ended March 31, 2025 and 2024, respectively.

As of March 31, 2025, we had an accumulated deficit of $1.5 billion.

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

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;

progress the clinical development of reni-cel;

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

nominate development candidates and initiate clinical trials for our current preclinical programs;

seek to identify additional research programs and additional product candidates;

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

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;

further develop our gene editing platform;

hire additional clinical, quality control, and scientific personnel;

hire additional personnel;

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

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We no longer receive research funding support.

During the three months ended September 30, 2024 and three months ended September 30, 2023, we did not recognize revenue related to our collaboration with BMS.

As of September 30, 2024, we had $56.7 million of deferred revenue in relation to our collaboration with BMS, of which $6.5 million was classified as short-term deferred revenue and $50.2 million was classified as long-term deferred revenue on our consolidated balance sheet.

During the three months ended March 31, 2025 we did not recognize revenue related to our collaboration with BMS.

As of March 31, 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.

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 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.

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 and 2025 annual license fees of $10.0 million in each of the first quarters of 2024 and 2025.

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

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 our license agreement with Vertex, collaboration with BMS, and any other collaborations or license agreements we may enter into.

Operating Expenses

Research and Development Expenses

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

• costs incurred under clinical trial agreements with investigative sites;

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

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

• costs incurred for research and development activities under our collaboration agreements ;

• costs incurred for the research and development activities under our collaboration agreements;

• facility costs including rent, depreciation, and maintenance expenses;

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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, IND-enabling studies and natural history studies;

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

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

• 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 progress our clinical trials as well as support preclinical studies for our other research programs.

We expect research and development costs 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.

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 increase in the future to support continued research and development activities and potential commercialization of any product candidates we identify and develop.

These increases will include increased costs related to the hiring of additional personnel and fees to outside consultants.

We also anticipate increased expenses related to reimbursement of third-party patent-related expenses and 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 relations costs.

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 will continue to be significant.

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.

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 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 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 nine months ended September 30, 2024 and September 30, 2023, other income, net consisted primarily of interest income and the amortization of premiums or discounts on marketable securities.

For the three months ended March 31, 2025 and March 31, 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.

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.

There have been no material changes to our critical accounting policies from those described in Part II, “Item 7.

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.

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

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 income.

Actual results may differ from these estimates.

Results of Operations

Comparison of the Three Months ended September 30, 2024 and 2023

The following table summarizes our results of operations for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Comparison of the Three Months ended March 31, 2025 and 2024

The following table summarizes our results of operations for the three months ended March 31, 2025 and 2024, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended

September 30, Dollar Change Percentage Change

Collaboration and other research and development revenues $ 61 $ 5,336 $ (5,275) (99) %

March 31, Dollar Change Percentage Change

Collaboration and other research and development revenues $ 4,658 $ 1,135 $ 3,523 n/m

Operating expenses:

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General and administrative 13,375 19,339 (5,964) (31) %

Restructuring and impairment charges 40,853 — 40,853 100 %

Total operating expenses 80,821 68,126 12,695 19 %

Other income, net

Other expense, net (5) — (5) 100 %

Other expense, net (425) 6 (431) n/m

Interest related to sale of future revenues (2,216) — (2,216) 100 %

Interest income, net 2,716 5,035 (2,319) (46) %

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Collaboration and Other Research and Development Revenues

Collaboration and other research and development revenues were $0.1 million for the three months ended September 30, 2024 compared to $5.3 million for the same period in 2023.

The decrease from the three months ended September 30, 2023 is primarily attributable to the receipt of the upfront payment for the non-exclusive license to Vor Bio in the third quarter of 2023.

Collaboration and other research and development revenues were $4.7 million for the three months ended March 31, 2025 compared to $1.1 million for the same period in 2024.

The increase from the three months ended March 31, 2024 is primarily attributable to the recognition of the remaining deferred revenue upon the closing out of a collaboration agreement with a strategic partner.

Research and Development Expenses

Research and development expenses increased by $7.1 million to $47.6 million for the three months ended September 30, 2024 compared to $40.5 million for the same period in 2023.

The following table summarizes our research and development expenses for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Research and development expenses decreased by $22.2 million to $26.6 million for the three months ended March 31, 2025 compared to $48.8 million for the same period in 2024.

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

Three Months Ended

September 30, Dollar Change Percentage Change

March 31, Dollar Change Percentage Change

Employee related expenses $ 11,322 $ 13,683 $ (2,361) (17) %

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Sublicense and license fees 17 5,896 (5,879) (100) %

Other expenses 3,511 1,689 1,822 n/m

Total research and development expenses $ 47,639 $ 40,512 $ 7,127 18 %

The increase in research and development expenses for the three months ended September 30, 2024 compared to the three months ended September 30, 2023 was primarily attributable to:

• approximately $3.3 million in increased employee-related expenses related to increased headcount to support manufacturing, quality, and clinical work related to our reni-cel program;

• approximately $2.5 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our reni-cel program as well as costs attributable to in vivo research and discovery;

• approximately $2.2 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 $1.8 million in increased other expenses related to external manufacturing cost savings realized in the third quarter of 2023.

These increases were partially offset by the following:

• approximately $2.1 million in decreased sublicense and license fees related to reduced licensing activity in the third quarter of 2024 compared to 2023;

• approximately $0.5 million in decreased stock-based compensation expense.

General and Administrative Expenses

General and administrative expenses increased by $3.1 million to $18.1 million for the three months ended September 30, 2024 compared to $15.0 million for the three months ended September 30, 2023.

The following table summarizes our general and administrative expenses for the three months ended September 30, 2024 and 2023, together with the changes in those items in dollars (in thousands) and the respective percentages of change:

Three Months Ended

September 30, Dollar Change Percentage Change

Employee related expenses $ 5,902 $ 3,910 $ 1,992 51 %

Professional service expenses 4,121 3,684 437 12 %

Intellectual property and patent related fees 3,291 2,769 522 19 %

Stock-based compensation expenses 2,754 2,160 594 28 %

Facility and other expenses 2,020 2,464 (444) (18) %

Total general and administrative expenses $ 18,088 $ 14,987 $ 3,101 21 %

The increase in general and administrative expenses for the three months ended September 30, 2024 compared to the three months ended September 30, 2023 was primarily attributable to:

• approximately $2.0 million in increased employee related expenses related to increased headcount to support business operations due to the progression of our reni-cel program;

• approximately $0.6 million in increased stock-based compensation expenses related to increased headcount;

• approximately $0.5 million in increased intellectual property and patent related fees;

• approximately $0.4 million in increased professional service expenses.

These increases were partially offset by approximately $0.4 million in decreased facility and other expenses.

Other Income, Net

For the three months ended September 30, 2024, and September 30, 2023 other income, net was $3.5 million and $5.1 million, respectively, which primarily relates to interest income and accretion of discounts and premiums associated with marketable securities.

The decrease is attributable to a reduction in our investments for the three months ended September 30, 2024 compared to the three months ended September 30, 2023.

Comparison of the Nine Months ended September 30, 2024 and 2023

The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023, 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 $ 1,710 $ 18,074 $ (16,364) (91) %

Operating expenses:

Research and development 150,636 108,095 42,541 39 %

General and administrative 55,633 55,198 435 1 %

Total operating expenses 206,269 163,293 42,976 26 %

Other income, net

Other income (expense), net — (1,590) 1,590 (100) %

Interest income, net 12,861 12,464 397 3 %

Total other income, net 12,861 10,874 1,987 18 %

Net loss $ (191,698) $ (134,345) $ (57,353) 43 %

Collaboration and Other Research and Development Revenues

Collaboration and other research and development revenues decreased by $16.4 million to $1.7 million for the nine months ended September 30, 2024 compared to $18.1 million for nine months ended September 30, 2023.

The decrease was primarily related to the sale of our wholly owned oncology assets and licenses in January 2023 and the receipt of the upfront payment for the non-exclusive license to Vor Bio in the third quarter of 2023, for which there was no equivalent revenue in 2024 due to the decrease of collaboration license activity.

Research and Development Expenses

Research and development expenses increased by $42.5 million to $150.6 million for the nine months ended September 30, 2024 compared to $108.1 million for the nine months ended September 30, 2023.

The following table summarizes our research development expenses for the nine months ended September 30, 2024 and 2023, 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 $ 41,830 $ 34,970 $ 6,860 20 %

External research and development expenses 64,670 37,110 27,560 74 %

Facility expenses 19,818 16,053 3,765 23 %

Stock-based compensation expenses 7,081 7,182 (101) (1) %

Sublicense and license fees 7,060 4,700 2,360 50 %

Other expenses 1,511 3,493 (1,982) (57) %

Total research and development expenses $ 26,593 $ 48,787 $ (22,194) (45) %

The increase in research and development expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily attributable to:

• approximately $27.6 million in increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our 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 to support manufacturing, quality, and clinical work related to our reni-cel program;

• approximately $3.8 million in increased facility expenses primarily related to increased rent expense incurred in connection with a lease commencement for manufacturing space;

• approximately $2.4 million in increased sublicense and license fees paid in connection with licensing activity in 2024;

• approximately $2.1 million in increased other expenses related to increased consulting fees to support clinical and quality initiatives in connection with our reni-cel program.

These increases were partially offset by $0.1 million in decreased stock-based compensation expense.

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

• approximately $10.1 million in decreased external research and development expenses primarily related to clinical and manufacturing costs related to our reni-cel program due to the Discontinuation, partially offset by costs attributable to in vivo research and discovery;

• approximately $5.9 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 achievement in the first quarter of 2025;

• approximately $2.4 million in decreased employee-related expenses related to reduced headcount associated with the Reduction;

• approximately $2.0 million in decreased other expenses related to professional services to support our reni-cel program due to the Discontinuation;

• approximately $2.0 million in decreased stock-based compensation expense related to expense in connection with the achievement of certain performance-based vesting of restricted stock units recognized in the first quarter of 2024 for which there was no equivalent expense in the first quarter of 2025.

These decreases were partially offset by approximately $0.1 million in increased facility expenses.

General and Administrative Expenses

General and administrative expenses increased by $0.4 million to $55.6 million for the nine months ended September 30, 2024 compared to $55.2 million for the nine months ended September 30, 2023.

The following table summarizes our general and administrative expenses for the nine months ended September 30, 2024 and 2023, 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

General and administrative expenses decreased by $6.0 million to $13.4 million for the three months ended March 31, 2025 compared to $19.3 million for the three months ended March 31, 2024.

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

Three Months Ended

March 31, Dollar Change Percentage Change

Employee related expenses $ 4,441 $ 4,889 $ (448) (9) %

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Total general and administrative expenses $ 13,375 $ 19,339 $ (5,964) (31) %

The increase in general and administrative expenses for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily attributable to:

• approximately $3.0 million in increased stock-based compensation expenses due primarily to the achievement of certain performance-based vesting of restricted stock units in 2024;

• approximately $2.9 million in increased employee related expenses related to increased headcount to support business operations and progression of reni-cel program;

• approximately $0.4 million in increased intellectual property and patent related fees;

• approximately $0.3 million in increased facility and other expense;

These increases were partially offset by approximately $6.3 million in decreased professional service expenses primarily related to one-time expenses to support strategic initiatives and business development activities in the nine months ended September 30, 2023.

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

• approximately $2.6 million in decreased stock-based compensation expenses related to expense in connection with the achievement of certain performance-based vesting of restricted stock units recognized in the first quarter of 2024 for which there was no equivalent expense in the first quarter of 2025;

• approximately $1.4 million in decreased intellectual property and patent related legal fees;

• approximately $0.9 million in decreased facility and other expenses related to the expiration of a lease;

• approximately $0.6 million in decreased professional service expenses related to reduced licensing and strategic business activities in 2025 relative to 2024;

• approximately $0.4 million in decreased employee related expenses related to reduced headcount associated with the Reduction.

Restructuring and Impairment Charges

Restructuring charges were $40.9 million for the three months ended March 31, 2025, with no equivalent charges for the three months ended March 31, 2024.

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

Three Months Ended March 31, Dollar Change Percentage Change

Employee termination benefits $ 3,509 $ — $ 3,509 100 %

Costs for ongoing contracts and terminated contracts 28,875 — 28,875 100 %

Acceleration of expense for change in useful life estimate 4,745 — 4,745 100 %

Impairment charges 3,724 — 3,724 100 %

Total restructuring and impairment charges $ 40,853 $ — $ 40,853 100 %

During the three months ended March 31, 2025, we recorded $3.5 million, $28.9 million, $4.7 million and $3.7 million related to employee termination benefits, program-related ongoing contract and terminated 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 nine months ended September 30, 2024 and September 30, 2023, other income, net was $12.9 million and $10.9 million, respectively, which was primarily attributable to interest income, partially offset by accretion of discounts associated with other marketable securities.

The increase for the nine months ended September 30, 2024 is attributable to increased invested balances throughout the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 and favorable market rates.

For the three months ended March 31, 2025, and March 31, 2024 other income, net was $0.1 million and $5.0 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 is attributable to interest accretion related to the DRI Purchase and Sale Agreement off-setting our investment income along with a reduction in our investments for the three months ended March 31, 2025 compared to the three months ended March 31, 2024

Liquidity and Capital Resources

Sources of Liquidity

As of September 30, 2024, 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, our former strategic alliance with Allergan, and payments received under the license agreement with Vertex.

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $265.1 million.

Subsequently, in October 2024, we received the $57.0 million payment under the DRI Agreement.

In May 2021, we entered into a common stock sales agreement with Cowen and Company, LLC (“Cowen”), under which we from time to time can issue and sell shares of our common stock through Cowen in at-the-market offerings for aggregate gross sale proceeds of up to $300.0 million (the “ATM Facility”).

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

As of September 30, 2024, we have not sold any shares of our common stock under the ATM Facility.

As of March 31, 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 March 31, 2025, we had cash, cash equivalents and marketable securities of $221.0 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.

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 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 March 31, 2025, we have sold 706,236 shares of our common stock under the ATM Facility at a weighted average price of $2.03 per share for aggregate gross proceeds of $1.4 million and have $148.6 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.

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 September 30, 2024, our right to contingent payments under our collaboration agreement with BMS and our license agreement with 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.

The following table provides information regarding our cash flows for the nine months ended September 30, 2024 and 2023 (in thousands):

Nine Months Ended

September 30,

March 31, 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 three months ended March 31, 2025 and 2024 (in thousands):

Three Months Ended

Net cash (used in) provided by:

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Financing activities (1,437) 192

Net decrease in cash, cash equivalents, and restricted cash $ (27,820) $ (58,984)

Net increase (decrease) in cash, cash equivalents, and restricted cash $ 7,151 $ (54,425)

Net Cash Used in Operating Activities

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 $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 the reni-cel program, sublicense and license payments, and supporting business operations.

Net cash used in operating activities was approximately $109.9 million for the nine months ended September 30, 2023, which primarily consisted of operating expenses that related to increasing our research efforts, the focused progression of clinical and manufacturing activities in support of reni-cel, and supporting business operations.

Net cash used in operating activities was approximately $47.8 million for the three months ended March 31, 2025, 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 $49.9 million for the three months ended March 31, 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 $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 purchases of marketable securities of $86.2 million and purchases of property and equipment of $8.3 million.

Net cash used in investing activities was approximately $66.6 million for the nine months ended September 30, 2023, primarily related to the purchases of marketable securities of $219.8 million, partially offset by the proceeds from the maturities of marketable securities of $156.6 million.

Net Cash Provided by Financing Activities

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 the 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 $117.5 million for the nine months ended September 30, 2023, primarily related to proceeds from offering of common stock of $117.1 million after deducting underwriting discounts and commissions and other offering costs, and proceeds received from issuance of common stock under the employee stock purchase plan of $0.4 million

Net cash provided by investing activities was approximately $56.4 million for the three months ended March 31, 2025, primarily related to the proceeds from the maturities of marketable securities of $56.5 million.

Net cash used in investing activities was approximately $4.7 million for the three months ended March 31, 2024, primarily related to the purchases of marketable securities of $86.2 million, partially offset by the proceeds from the maturities of marketable securities of $83.4 million.

Net Cash Used in Financing Activities

Net cash used in financing activities was approximately $1.4 million for the three months ended March 31, 2025, primarily related to the repayment of our liability for the sale of future revenues with DRI of $2.9 million.

This was offset by the proceeds from the issuance of common stock from our ATM Facility of $1.4 million .

Net cash provided by financing activities was approximately $0.2 million for the three months ended March 31, 2024, related to the proceeds received from exercises of options for our common stock.

Funding Requirements

We expect our expenses to increase in connection with our ongoing activities, particularly as we progress the clinical development of reni-cel;

further advance our research programs and our preclinical development activities;

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;

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;

hire additional clinical, quality control, and scientific personnel;

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.

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.

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.

We do not expect to generate significant recurring revenue unless and until we obtain regulatory approval for and commercialize a product candidate.

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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 September 30, 2024, together with the upfront cash payment from DRI in October 2024, 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 2026 .

We expect that our existing cash, cash equivalents and marketable securities on March 31, 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 .

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 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.

We could utilize our available capital

resources sooner than we currently expect.

We could utilize our available capital resources sooner than we currently expect.

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

• the costs of progressing the clinical development of reni-cel to treat SCD and TDT;

• 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 restructuring costs associated with the Discontinuation and Reduction;

• 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.

• the outcome of our process to partner or out-license reni-cel to focus our resources on in vivo pipeline development.

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.

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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.

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 September 30, 2024, we had operating leases with future minimum lease payments for a total of $44.0 million, of which $5.9 million will be payable in 2024.

As of March 31, 2025, we had operating leases with future minimum lease payments for a total of $30.0 million, of which $9.6 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.

During the three months ended June 30, 2023, we entered into a license and service agreement pursuant to which we will lease manufacturing space for our continued research and development activities.

In 2023, we entered into a license and service agreement pursuant to which we leased manufacturing space for our continued research and development activities.

The lease commenced April 1, 2024.

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.

The termination of the license and service agreement continues to remain at our discretion.

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.

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

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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