What changed in this filing

/stocks — the workspaceLOADING

Item 2. Management’s Discussion and Analysis

2025-08-12 compared with 2025-05-12 · 137 added, 72 removed, 219 unchanged (49% of the section changed)

20 unchanged sentences

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 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.

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 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.

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 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.

1 unchanged sentence

(“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.

This collaboration, which leverages our Cas9 and Cas12a 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).

10 unchanged sentences

Since inception, we have incurred significant operating losses.

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.

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.

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

4 unchanged sentences

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;

6 unchanged sentences

We no longer receive research funding support.

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.

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.

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

36 unchanged sentences

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.

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

Restructuring and Impairment Charges

1 unchanged sentence

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.

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.

1 unchanged sentence

Other Income, Net

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.

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.

Critical Accounting Policies and Estimates

17 unchanged sentences

Results of Operations

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:

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:

Three Months Ended

March 31, Dollar Change Percentage Change

June 30, Dollar Change Percentage Change

Collaboration and other research and development revenues $ 3,578 $ 513 $ 3,065 n/m

4 unchanged sentences

Total operating expenses 55,122 72,416 (17,294) (24) %

Other income, net

Other (expense) income, net

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

1 unchanged sentence

Interest income, net 2,087 4,297 (2,210) (51) %

Total other income, net 75 5,041 (4,966) (99) %

Total other (expense) income, net (1,691) 4,296 (5,987) n/m

Net loss $ (53,235) $ (67,607) $ 14,372 (21) %

1 unchanged sentence

Collaboration and Other Research and Development Revenues

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.

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.

Research and Development Expenses

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:

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:

Three Months Ended

March 31, Dollar Change Percentage Change

June 30, Dollar Change Percentage Change

Employee related expenses $ 8,067 $ 13,661 $ (5,594) (41) %

5 unchanged sentences

Total research and development expenses $ 16,181 $ 54,210 $ (38,029) (70) %

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;

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;

• approximately $5.6 million in decreased employee-related expenses related to reduced headcount associated with the Reduction;

• approximately $4.0 million in decreased facility expense primarily due to the end of leases for manufacturing space;

• 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.

• 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.

General and Administrative Expenses

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:

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:

Three Months Ended

March 31, Dollar Change Percentage Change

June 30, Dollar Change Percentage Change

Employee related expenses $ 3,132 $ 4,800 $ (1,668) (35) %

4 unchanged sentences

Total general and administrative expenses $ 12,859 $ 18,206 $ (5,347) (29) %

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;

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:

• approximately $1.7 million in decreased employee related expenses related to reduced headcount associated with the Reduction;

• approximately $1.6 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 $0.9 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 $0.3 million in decreased professional service expenses.

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

Collaboration and other research and development revenues $ 8,236 $ 1,649 $ 6,587 n/m

Operating expenses:

Research and development 42,774 102,997 (60,223) (58) %

General and administrative 26,234 37,545 (11,311) (30) %

Restructuring and impairment charges 66,935 — 66,935 100 %

Total operating expenses 135,943 140,542 (4,599) (3) %

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 %

Interest income, net 4,803 9,331 (4,528) (49) %

Total other (expense) income, net (1,616) 9,336 (10,952) n/m

Net loss $ (129,323) $ (129,557) $ 234 — %

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.

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.

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

Employee related expenses $ 19,389 $ 27,344 $ (7,955) (29) %

External research and development expenses 10,066 44,613 (34,547) (77) %

Facility expenses 8,755 12,589 (3,834) (30) %

Stock-based compensation expenses 1,770 5,033 (3,263) (65) %

Sublicense and license fees 112 6,753 (6,641) (98) %

Other expenses 2,682 6,665 (3,983) (60) %

Total research and development expenses $ 42,774 $ 102,997 $ (60,223) (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;

• approximately $8.0 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 $3.8 million in decreased facility expenses primarily due to the end of leases for manufacturing space;

• approximately $3.3 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

Employee related expenses $ 7,574 $ 9,690 $ (2,116) (22) %

Professional service expenses 5,902 6,808 (906) (13) %

Intellectual property and patent related fees 5,767 7,790 (2,023) (26) %

Stock-based compensation expenses 3,873 7,562 (3,689) (49) %

Facility and other expenses 3,118 5,695 (2,577) (45) %

Total general and administrative expenses $ 26,234 $ 37,545 $ (11,311) (30) %

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;

• approximately $2.1 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 $0.9 million in decreased professional service expenses related to reduced licensing and strategic business activities in 2025 relative to 2024.

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

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

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

Costs for ongoing contracts and terminated contracts 52,341 — 52,341 100 %

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

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

Impairment charges 3,724 — 3,724 100 %

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

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.

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 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

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.

Liquidity and Capital Resources

Sources of Liquidity

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 .

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 .

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.

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

1 unchanged sentence

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.

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.

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.

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

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

Net cash (used in) provided by:

5 unchanged sentences

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 $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 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 Provided by Investing Activities

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.

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 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.

Funding Requirements

3 unchanged sentences

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.

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.

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

1 unchanged sentence

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 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 .

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 .

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.

19 unchanged sentences

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.

2 unchanged sentences

Contractual Obligations

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.

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.

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.

12 unchanged sentences

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