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Voyager Therapeutics, Inc.

For the period ended Sep 30, 2025. Filed Nov 10, 2025.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Sep 30, 2025 · Filed Nov 10, 2025

VYGR$1.87+8.4%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-06-30; filed 2025-08-06.

0001558370-25-010511

ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

Current report: 10-Q

Period ended 2025-09-30; filed 2025-11-10.

0001104659-25-108846

ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

The baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.

Compared 115 previous paragraphs and 119 current paragraphs.

68 unchanged; 33 reworded; 14 added; 11 removed; 3 previous and 4 current paragraphs rearranged.

Tables are not compared: 67 previous and 64 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.

A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.

Added paragraphs (14)

Current report

In addition to our wholly-owned pipeline, we are advancing three programs with collaboration partners for which we retain options to participate in future development and commercialization. Two of these programs are in collaboration with Neurocrine: a glucosylceramidase beta 1, or GBA1, gene therapy program that will focus on Gaucher

Current report

and Parkinson’s disease, which we refer to as the GBA1 Program, and a frataxin (FXN) gene therapy program for FA, which we refer to as the FA Program. Neurocrine has indicated that they expect to provide an update on the IND filing timelines for their FA Program and GBA1 Program by the end of 2025. These filings could enable the initiation of clinical trials in 2026, pending supportive outcomes from the ongoing Good Laboratory Practice toxicology studies, acceptance of the INDs by the FDA, and Neurocrine’s internal strategic assessment.

Current report

We entered a collaboration with Transition Bio, Inc, or Transition Bio, to develop selective small molecules for the treatment of amyotrophic lateral sclerosis, or ALS, and frontotemporal dementia, or FTD, with TDP-43 pathology. TDP-43 pathology is commonly observed in many neurodegenerative diseases, including more than 90% of ALS cases, yet TDP-43 has historically been considered undruggable. Transition Bio’s approach targets biomolecular condensates within cells to correct the mislocalization of TDP-43 without abolishing its important functional activity. The collaboration provides us the exclusive option to license worldwide rights to any development candidate targeting TDP-43 in exchange for a single-digit million-dollar upfront payment and potential milestone payments totaling up to $500.0 million. Transition Bio is also eligible for tiered royalties on net sales if a product reaches the market.

Current report

In addition to the three partnered programs on which we retain opt-in rights, we have entered into multiple additional collaboration and licensing agreements with collaboration partners.

Current report

We have leveraged our knowledge of BBB receptors, discovered using TRACER, to develop a second platform called Voyager NeuroShuttle. Voyager NeuroShuttle is a nonviral delivery platform leveraging novel receptor-binding molecules to transport multiple modalities of neurotherapeutics across the blood-brain barrier. The first NeuroShuttle within the platform leverages the ALPL receptor. During the third quarter of 2025, we shared the results of initial murine proof-of-concept studies of ALPL-VYGR-NeuroShuttle demonstrating in the study sustained brain expression over three weeks, compared to less than one week for transferrin receptor shuttles, with no impact on circulating reticulocytes or downstream measurements of anemia. In addition to the initial murine proof-of-concept data provided for the ALPL-VYGR-NeuroShuttle, subsequent murine studies have demonstrated that the shuttle can deliver a therapeutic antibody to the brain with similar sustained exposure as demonstrated with the ALPL-VYGR-NeuroShuttle alone. We initiated a discovery program using ALPL-VYGR-NeuroShuttle to target an undisclosed neurological disease.

Current report

Under the terms of the 2022 Novartis Option and License Agreement, Novartis has the right to terminate the agreement, in whole or in part, for any or no reason upon ninety days’ written notice to us. In October 2025, Novartis notified us of its partial termination of the 2022 Novartis Option and License Agreement with respect to one Initial Novartis Target and the Novartis Direct License Target, in each case effective February 1, 2026, or the Effective Partial Termination Date.

Current report

Upon the termination of the two programs, the licenses we have granted to Novartis regarding the applicable Initial Novartis Target and the Novartis Direct License Target will expire, and all intellectual property rights that we have licensed to Novartis with respect to such programs will revert to us in accordance with the 2022 Novartis Option and License Agreement. As of the Effective Partial Termination Date, we will no longer be eligible to receive milestone payments or royalties in association with these programs. The 2022 Novartis Option and License Agreement remains in full force and effect for, and we remain eligible to receive milestone payments and royalties in connection with, the remaining program under the agreement.

Current report

On July 4, 2025, the One Big Beautiful Bill Act, or the Act, or OBBBA, was signed into law. Included in this legislation are provisions that allow for the immediate expensing of domestic research and development expenses, immediate expensing of certain capital expenditures, and other changes to the U.S. taxation of profits derived from foreign operations. The legislation has multiple effective dates, with certain provisions effective in the current fiscal period and others in the fiscal year ending December 31, 2026. The Company will continue to evaluate the impact of OBBBA, but does not expect it to have a material impact on its consolidated financial statements for fiscal year 2025.

Current report

Collaboration revenue was $13.4 million and $24.6 million for the three months ended September 30, 2025 and 2024, respectively. This decrease was primarily due to the recognition of a $15.0 million amendment fee under the 2022 Novartis Option and License Agreement during the three months ended September 30, 2024.

Current report

General and administrative expenses decreased by $0.1 million from $8.2 million for the three months ended September 30, 2024, to $8.1 million for the three months ended September 30, 2025. The consistent spend reflects continued disciplined expense management after the reduction in force during the second quarter of 2025.

Current report

Comparison of the nine months ended September 30, 2025 and 2024

Current report

During the nine months ended September 30, 2025, we recognized collaboration revenue in connection with the following agreements:

Current report

During the nine months ended September 30, 2024, we recognized collaboration revenue in connection with the following agreements:
Removed paragraphs (11)

Previous report

We are also working with our collaboration partners on multiple programs. We are advancing five gene therapy programs with Neurocrine. Development candidates were selected for three of these programs in 2024, resulting in milestone payments to us. We expect that Neurocrine will submit IND filings in 2025 for the two most advanced of these

Previous report

programs, a glucosylceramidase beta 1, or GBA1, gene therapy program, and a frataxin, or FXN, gene therapy program for FA, or the FA Program, provided the ongoing GLP toxicology studies support clinical development. These two programs are particularly significant to us for two reasons: the subsequent clinical trials of these programs have the potential to establish human proof-of-concept for the TRACER Capsids, and we have the option to opt-in on co-development and co-commercialization in the United States for both products. Initiation of the first clinical trials for the FA and GBA1 programs are expected in 2026, and the associated regulatory and clinical milestones that we could potentially realize related to these indications in 2025-2026 total up to $35 million.

Previous report

In the first half of 2025, Voyager restructured to focus resources on achieving critical clinical data while continuing to build an industry-leading multi-modality neurotherapeutics pipeline. Based on current operating plans, the anticipated cost savings from these actions are expected to extend the company’s cash runway into 2028, well beyond multiple clinical inflection points including:

Previous report

The restructuring plan includes cost-savings measures across multiple programs and research, reflecting reductions in force, streamlining clinical trial designs, introducing operational efficiencies, gating of certain activities, implementation of outsourcing in certain functions, and utilization of technology-based approaches as applicable. Changes to clinical trial design include streamlining the ongoing clinical trial of VY7523 and the planned clinical trial of VY1706 to focus on critical tau PET imaging data, and, in the case of the VY7523 trial, to review those data at 12-months versus 18-months.

Previous report

Employees impacted by a reduction in force were eligible to receive one-time severance benefits, including cash severance, temporary health care coverage, and transition support services, subject to each employee entering into a separation agreement, which included a general release of claims against the company. The Company recorded $2.4 million of restructuring expenses in the six months ended June 30, 2025, all of which are included in the total accrued expenses on the condensed consolidated balance sheets for the period.

Previous report

On July 4, 2025, the One Big Beautiful Bill Act, or the Act, was signed into law. The Act includes several tax changes, such as making certain provisions from the Tax Cuts and Jobs Act permanent, updating international tax rules, and reinstating immediate expensing for domestic research expenditures. The Act contains multiple effective dates, with certain provisions applicable beginning in 2025 and others in subsequent years. We are currently evaluating the potential impacts of the Act on our consolidated financial statements.

Previous report

General and administrative expenses increased by $0.3 million from $10.2 million for the three months ended June 30, 2024, to $10.5 million for the three months ended June 30,2025 due to increased employee related costs of $1.4 million associated with the reduction in force completed during the three months ended June 30, 2025.

Previous report

Collaboration revenue was $11.7 million and $49.1 million for the six months ended June 30, 2025 and 2024, respectively. This decrease was primarily due to the recognition of revenue under the percent complete model for up front work related to the 2023 Neurocrine Collaboration Agreement during the second quarter of 2024. During the six months ended June 30, 2025, we recognized collaboration revenue in connection with the following agreements:

Previous report

During the six months ended June 30, 2024, we recognized collaboration revenue in connection with the following agreements:
Reworded paragraphs (33)

Previous wording

We are advancing our own proprietary pipeline of drug candidates for neurological diseases, with a focus on AD, including programs against three critically important AD targets: tau, amyloid and apolipoprotein E (APOE). Our wholly-owned prioritized pipeline includes two tau targeting programs: VY7523 (which we formerly referred to as VY-TAU01), an anti-tau antibody for AD, and VY1706, a tau silencing gene therapy for AD. VY7523 is an IV-administered, recombinant, humanized IgG4 monoclonal antibody developed to inhibit the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in AD. The murine version of VY7523 reduced tau spread by approximately 70% in preclinical studies. VY7523 demonstrated an acceptable safety, tolerability, and immunogenicity profile as well as expected pharmacokinetic results in a Phase 1, single ascending dose, or SAD, clinical trial in healthy volunteers. In February 2025, we initiated a Phase 1 multiple ascending dose, or MAD, clinical trial of VY7523 in early AD patients. We believe this trial has the potential to generate initial data in the second half of 2026. VY1706 is a gene therapy that leverages an intravenously delivered TRACER Capsid containing a vectorized small interfering RNA specifically targeting tau mRNA. In a non-human primate, or NHP, study, a single 1.3E13 vector genomes per kilogram dose of VY1706 delivered intravenously resulted in reductions in tau mRNA levels of 50% to 73% across the cerebral cortex, including in areas of the brain where tau accumulates during progression of AD. We anticipate the submission of an investigational new drug, or IND, application and initiation of a clinical trial for the VY1706 program in 2026.

Current wording

We are advancing our own proprietary pipeline of drug candidates for neurological diseases, with a focus on AD. Our wholly-owned prioritized pipeline includes two tau targeting programs: VY7523, an anti-tau antibody for AD, and VY1706, a tau silencing gene therapy for AD. VY7523 is an IV-administered, recombinant, humanized IgG4 monoclonal antibody developed to inhibit the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in AD. The murine version of VY7523 reduced tau spread by approximately 70% in preclinical studies. VY7523 demonstrated an acceptable safety, tolerability, and immunogenicity profile as well as expected pharmacokinetic results in a Phase 1, single ascending dose, or SAD, clinical trial in healthy volunteers. In February 2025, we initiated a Phase 1 multiple ascending dose, or MAD, clinical trial of VY7523 in early AD patients. We believe this trial has the potential to generate initial data in the second half of 2026. VY1706 is a gene therapy that leverages an intravenously delivered TRACER Capsid containing a vectorized small interfering RNA specifically targeting tau mRNA. In a non-human primate, or NHP, study, a single 1.3E13 vector genomes per kilogram dose of VY1706 delivered intravenously resulted in reductions in tau mRNA levels of 50% to 73% across the cerebral cortex, including in areas of the brain where tau accumulates during progression of AD. We anticipate the submission of an investigational new drug, or IND, application and initiation of a clinical trial for the VY1706 program in 2026.

Previous wording

Our proprietary pipeline also includes early research initiatives to develop an APOE gene therapy program, which is designed to knock down APOE in APOE4 carriers while delivering APOE2 and maintaining total APOE levels, as well as to develop a vectorized anti-amyloid antibody for AD and a superoxide dismutase 1, or SOD1, gene therapy for SOD1 amyotrophic lateral sclerosis.

Current wording

Our proprietary pipeline also includes early research initiatives to develop an apolipoprotein E, or APOE, gene therapy program, which is designed to knock down APOE in APOE4 carriers while delivering APOE2 and maintaining total APOE levels, and a nonviral therapeutic that leverages our proprietary Voyager NeuroShuttle TM platform for the treatment of an undisclosed neurological disease. During the third quarter of 2025, investment in the SOD1-ALS gene therapy and anti-Aβ antibody gene therapy programs was deprioritized to focus on other discovery programs.

Previous wording

The GBA1 program, or the GBA1 Program, will focus on both Gaucher and Parkinson’s disease. In addition to the FA and GBA1 Programs, we are collaborating with Neurocrine on three gene therapy programs against undisclosed targets. In addition to the Neurocrine collaborations, we have partnered with Novartis on TRACER Capsid-based gene therapies for spinal muscular atrophy and Huntington’s disease. We have also licensed capsids to Novartis for three undisclosed CNS targets and to Alexion for one undisclosed rare neurological disease target. In total, our partnerships have delivered more than $500.0 million in non-dilutive funding to us to date, including upfront fees, development milestone payments, option exercise fees, license fees, and research and development expense reimbursement. Looking forward, we have the potential to earn up to $7.4 billion in milestone payments across the partnered portfolio, including $2.6 billion in potential development milestone payments, as well as royalties.

Current wording

In addition to the FA and GBA1 Programs, we are collaborating with Neurocrine on three gene therapy programs against undisclosed targets. Two of these three programs have development candidates selected and are advancing through preclinical toxicology studies. In addition to the Neurocrine collaborations, we have partnered with Novartis on TRACER Capsid-based gene therapies for spinal muscular atrophy and Huntington’s disease. We have also licensed capsids to Alexion for one undisclosed rare neurological disease target and to Novartis for three undisclosed CNS targets. Our capsid licenses with respect to two of the three undisclosed targets under our 2022 Novartis Option and License Agreement will terminate in the first quarter of 2026 as described below. In total, our partnerships have delivered more than $500.0 million in non-dilutive funding to us to date, including upfront fees, development milestone payments, option exercise fees, license fees, and research and development expense reimbursement. Looking forward, we have the potential to earn up to $6.8 billion in milestone payments across the partnered portfolio, including $2.4 billion in potential development milestone payments, as well as royalties. Additionally, Neurocrine initiated a preclinical toxicology study with the fourth development candidate in a gene therapy program partnered with us, triggering a $3.0 million milestone payment that is owed to us in the fourth quarter of 2025.

Previous wording

All of the gene therapies in our wholly-owned and collaborative pipeline are delivered intravenously leveraging TRACER capsids. TRACER is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier, or BBB, and enhanced CNS tropism in multiple species, including NHPs. We are also developing a second, non-viral therapeutics platform focused on non-viral receptor-mediated transport across the BBB.

Current wording

All of the gene therapies in our wholly-owned and collaborative pipeline are delivered intravenously leveraging TRACER capsids. TRACER is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier, or BBB, and enhanced CNS tropism in multiple species, including NHPs. We subsequently utilized these BBB-penetrant capsids to identify the receptors by which the capsids enter the BBB. We have identified multiple receptors, including alkaline phosphatase, or ALPL, that are capable of transporting a large biologic molecule, such as a capsid, across the BBB.

Previous wording

We and Novartis entered into the 2023 Novartis Stock Purchase Agreement, on December 28, 2023, for the sale and issuance of the Novartis Shares to Novartis at a price of $9.324 per share, for an aggregate purchase price of

Current wording

We and Novartis also entered into a stock purchase agreement on December 28, 2023, or the 2023 Novartis Stock Purchase Agreement, for the sale and issuance of 2,145,002 shares of our common stock, or the Novartis Shares, to Novartis at a price of $9.324 per share, for an aggregate purchase price of approximately $20.0 million. In accordance

Previous wording

approximately $20.0 million. In accordance with the terms and conditions of the 2023 Novartis Stock Purchase Agreement, we issued and sold these shares to Novartis on January 3, 2024.

Current wording

with the terms and conditions of the 2023 Novartis Stock Purchase Agreement, we issued and sold the Novartis Shares to Novartis on January 3, 2024.

Previous wording

The 2019 Neurocrine Collaboration Agreement provides for aggregate development milestone payments from Neurocrine to us for the research, development, manufacture, and commercialization of gene therapy products, or the 2019 Collaboration Products, under the FA Program of up to $195.0 million. We may be entitled to receive aggregate commercial milestone payments for each 2019 Collaboration Product of up to $275.0 million, subject to an aggregate cap on commercial milestone payments across all 2019 Neurocrine Programs of $1.1 billion. We are no longer eligible to receive milestone or royalty payments for the VY-AADC Program in light of the partial termination of the 2019 Neurocrine Collaboration Agreement with respect to the VY-AADC Program. The JSC’s selection of a lead development candidate for the FA Program in February 2024 triggered a $5.0 million milestone payment to us, which we received in March 2024. In April 2025, Neurocrine deprioritized both 2019 Discovery Programs. On April 30, 2025, the JSC agreed to discontinue these programs, so rights to these targets returned to us. The discontinuations were not due to any safety concerns. As a result, such programs are deemed terminated, and we are no longer eligible to receive the

Current wording

cap on commercial milestone payments across all 2019 Neurocrine Programs of $1.1 billion. We are no longer eligible to receive milestone or royalty payments for the VY-AADC Program in light of the partial termination of the 2019 Neurocrine Collaboration Agreement with respect to the VY-AADC Program. The JSC’s selection of a lead development candidate for the FA Program in February 2024 triggered a $5.0 million milestone payment to us, which we received in March 2024. In April 2025, Neurocrine deprioritized both 2019 Discovery Programs. On April 30, 2025, the JSC agreed to discontinue these programs, so rights to these targets returned to us. The discontinuations were not due to any safety concerns. As a result, such programs are deemed terminated, and we are no longer eligible to receive the milestones for either of the two 2019 Discovery Programs of up to $130.0 million per 2019 Discovery Program or any commercial milestones or royalties related thereto.

Previous wording

Pursuant to an amendment to the Alexion Agreement effective as of September 30, 2024, we agreed to extend the research term thereunder until April 1, 2025, during which period we may, at our sole discretion and expense, conduct further research activities to identify additional TRACER Capsids for potential use in the development of AAV gene therapies under the Alexion Agreement. Effective March 15, 2025, we agreed to further extend the research term to October 1, 2025. For a further description of the Alexion Agreement, refer to Note 9,Significant Agreements, to our consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, under the caption “Alexion Option and License Agreement (Formerly Pfizer Option and License Agreement).”

Current wording

Pursuant to an amendment to the Alexion Agreement effective as of September 30, 2024, we agreed to extend the research term thereunder until April 1, 2025, during which period we had the right to, at our sole discretion and expense, conduct further research activities to identify additional TRACER Capsids for potential use in the development of AAV gene therapies under the Alexion Agreement. Effective March 15, 2025, we agreed to further extend the research term to October 1, 2025. For a further description of the Alexion Agreement, refer to Note 9,Significant Agreements, to our consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, under the caption “Alexion Option and License Agreement (Formerly Pfizer Option and License Agreement).”

Previous wording

We have a history of incurring significant losses. We reported a net loss of $64.4 million for the six months ended June 30, 2025 and a net loss of $65.0 million for the fiscal year ended December 31, 2024. As of June 30, 2025, we had an accumulated deficit of $390.6 million. We expect to continue to incur significant expenses and operating losses for the foreseeable future. We anticipate that our expenses will increase substantially in connection with our ongoing activities, as we:

Current wording

We have a history of incurring significant losses. We reported a net loss of $92.3 million for the nine months ended September 30, 2025 and a net loss of $65.0 million for the fiscal year ended December 31, 2024. As of September 30, 2025, we had an accumulated deficit of $418.5 million. We expect to continue to incur significant expenses and operating losses for the foreseeable future. We anticipate that our expenses will increase substantially in connection with our ongoing activities, as we:

Previous wording

To date, we have not generated any revenue from product sales and do not expect to generate any revenue from product sales for the foreseeable future. For the three months ended June 30, 2025, we recognized $2.4 million of collaboration revenue from the 2019 Neurocrine Collaboration Agreement, $1.4 million of collaboration revenue in connection with the 2023 Novartis Collaboration Agreement, $1.3 million of collaboration revenue from the 2023 Neurocrine Collaboration Agreement, and $0.1 million of other collaboration revenue. For the six months ended June 30, 2025, we recognized $5.8 million of collaboration revenue from the 2023 Neurocrine Collaboration Agreement, $2.8 million of collaboration revenue from the 2019 Neurocrine Collaboration Agreement, $2.7 million of collaboration revenue in connection with the 2023 Novartis Collaboration Agreement, and $0.4 million of other collaboration revenue.

Current wording

To date, we have not generated any revenue from product sales and do not expect to generate any revenue from product sales for the foreseeable future. For the three months ended September 30, 2025, we recognized $11.1 million of collaboration revenue from the 2023 Neurocrine Collaboration Agreement, $1.5 million of collaboration revenue in connection with the 2023 Novartis Collaboration Agreement, $0.7 million of collaboration revenue from the 2019 Neurocrine Collaboration Agreement, and $0.1 million of other collaboration revenue. For the nine months ended September 30, 2025, we recognized $16.9 million of collaboration revenue from the 2023 Neurocrine Collaboration

Previous wording

For additional information about our revenue recognition policy related to collaborations pursuant to the 2023 and 2019 Neurocrine Collaboration Agreements and the 2023 Novartis Collaboration Agreement, refer to Note 9 of the December 31, 2024, consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.

Current wording

For additional information about our revenue recognition policy related to collaborations pursuant to the 2023 and 2019 Neurocrine Collaboration Agreements and the 2023 Novartis Collaboration Agreement, refer to Note 9,Significant Agreements of the December 31, 2024, consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024.

Previous wording

Research and development activities are central to our business model. We are in the early stages of development of our product candidates. During the six months ended June 30, 2025, our research and development expenses have increased as compared to the amounts recorded in the same period in the prior year. As our research and development programs progress and as we identify product candidates and initiate preclinical studies and clinical trials, including our MAD clinical trial to evaluate VY7523, we expect research and development costs to continue to increase. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development of our product candidates.

Current wording

Research and development activities are central to our business model. We are in the early stages of development of our product candidates. During the nine months ended September 30, 2025, our research and development expenses have increased as compared to the amounts recorded in the same period in the prior year. As our research and development programs progress and as we identify product candidates and initiate preclinical studies and clinical trials, including our MAD clinical trial to evaluate VY7523, we expect research and development costs to continue to increase. At this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development of our product candidates.

Previous wording

During the six months ended June 30, 2025, general and administrative expenses have increased as compared to the amount recorded in the same period in prior year primarily due to increased employee related costs including amounts associated with the reduction in force completed during the three months ended June 30, 2025.

Current wording

During the nine months ended September 30, 2025, general and administrative expenses have decreased as compared to the amount recorded in the same period in prior year primarily due to the reduction in force completed during the second quarter of 2025.

Previous wording

We believe that several accounting policies are important to understanding our historical and future performance. We refer to these policies as critical because these specific areas generally require us to make judgments and estimates about matters that are uncertain at the time we make the estimate. There were no changes to our critical accounting policies during the six months ended June 30, 2025, as compared to those identified in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024. It is important that the discussion of our operating results that follow be read in conjunction with the critical accounting policies disclosed in Item 7 “Management’s Discussion and Analysis of Financial Condition and Results of Operations –Critical Accounting Policies and Estimates” in our Annual Report on Form 10-K, as filed with the SEC on March 11, 2025.

Current wording

We believe that several accounting policies are important to understanding our historical and future performance. We refer to these policies as critical because these specific areas generally require us to make judgments and estimates about matters that are uncertain at the time we make the estimate. There were no changes to our critical accounting policies during the nine months ended September 30, 2025, as compared to those identified in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024. It is important that the discussion of our operating results that follow be read in conjunction with the critical accounting policies disclosed in Item 7 “Management’s Discussion and Analysis of Financial Condition and Results of Operations –Critical Accounting Policies and Estimates” in our Annual Report on Form 10-K, as filed with the SEC on March 11, 2025.

Previous wording

Research and development expenses decreased by $3.1 million from $34.5 million for the three months ended June 30, 2024, to $31.3 million for the three months ended June 30, 2025. The following table summarizes our research

Current wording

Research and development expenses increased by $5.7 million from $30.2 million for the three months ended September 30, 2024, to $35.9 million for the three months ended September 30, 2025. The following table summarizes our research and development expenses for the three months ended September 30, 2025 and 2024. All amounts for the three months ended September 30, 2024 have been reclassified to conform to the current year’s presentation.

Previous wording

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

Current wording

The increase in research and development expense for the three months ended September 30, 2025 was primarily attributable to the following:

Previous wording

Other income, net decreased by approximately $1.7 million. The decrease was primarily due to decreased interest income on our marketable securities balances, as our marketable securities balance decreased during the three months ended June 30, 2025, as compared to the three months ended June 30, 2024.

Current wording

Other income, net decreased by approximately $2.0 million. The decrease was primarily due to decreased interest income on our marketable securities balances, as our marketable securities balance decreased during the three months ended September 30, 2025, as compared to the three months ended September 30, 2024.

Previous wording

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:

Current wording

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

Previous wording

Collaboration revenue was $5.2 million and $29.6 million for the three months ended June 30, 2025 and 2024, respectively. This decrease was primarily due to the recognition of revenue under the percent complete model for up front work related to the 2023 Neurocrine Collaboration Agreement during the second quarter of 2024. During the three months ended June 30, 2025, we recognized collaboration revenue in connection with the following agreements:

Current wording

Collaboration revenue was $25.0 million and $73.7 million for the nine months ended September 30, 2025 and 2024, respectively. This decrease was primarily due to the $42.1 million of collaboration revenue recognized on the proportional performance model and the recognition of a $15.0 million amendment fee under the 2022 Novartis Option and License Agreement during the nine months ended September 30, 2024.

Previous wording

Research and development expense increased by $1.3 million from $61.5 million for the six months ended June 30, 2024 to $62.9 million for the six months ended June 30, 2025. The following table summarizes our research and

Current wording

Research and development expense increased by $6.9 million from $91.8 million for the nine months ended September 30, 2024 to $98.7 million for the nine months ended September 30, 2025. The following table summarizes

Previous wording

and development expenses for the three months ended June 30, 2025 and 2024. All amounts for the three months ended June 30, 2024 have been reclassified to conform to the current year’s presentation.

Current wording

our research and development expenses for the nine months ended September 30, 2025 and 2024. All amounts for the nine months ended September 30, 2024 have been reclassified to conform to the current year’s presentation.

Previous wording

The increase in research and development expense for the six months ended June 30, 2025 was primarily attributable to the following:

Current wording

The increase in research and development expense for the nine months ended September 30, 2025 was primarily attributable to the following:

Previous wording

General and administrative expenses increased by $1.4 million from $18.8 million for the six months ended June 30, 2024, to $20.1 million for the six months ended June 30, 2025, due to increased employee related costs including $1.4 million associated with the reduction in force completed during the six months ended June 30, 2025.

Current wording

General and administrative expenses increased by $1.3 million from $26.9 million for the nine months ended September 30, 2024, to $28.2 million for the nine months ended September 30, 2025, due to increased employee related costs, including $1.4 million associated with the reduction in force completed in the second quarter of 2025.

Previous wording

Other income, net decreased by approximately $2.8 million. The decrease was primarily due to decreased interest income on our marketable securities balances, as our marketable securities decreased during the six months ended June 30, 2025, as compared to the six months ended June 30, 2024.

Current wording

Other income, net decreased by approximately $4.8 million. The decrease was primarily due to decreased interest income on our marketable securities balances, as our marketable securities decreased during the nine months ended September 30, 2025, as compared to the nine months ended September 30, 2024.

Previous wording

During the six months ended June 30, 2024, we completed an underwritten public offering which resulted in net proceeds to us of approximately $93.5 million after deducting underwriting discounts and commissions and offering expenses.

Current wording

During the nine months ended September 30, 2024, we completed an underwritten public offering which resulted in net proceeds to us of approximately $93.5 million after deducting underwriting discounts and commissions and offering expenses.

Previous wording

We and Novartis also entered into a stock purchase agreement on December 28, 2023, or the 2023 Novartis Stock Purchase Agreement, for the sale and issuance of 2,145,002 shares of our common stock, or the Novartis Shares, to Novartis at a price of $9.324 per share, for an aggregate purchase price of approximately $20.0 million. In accordance with the terms and conditions of the 2023 Novartis Stock Purchase Agreement, we issued and sold the Novartis Shares to Novartis on January 3, 2024.

Current wording

We and Novartis entered into the 2023 Novartis Stock Purchase Agreement, on December 28, 2023, for the sale and issuance of the Novartis Shares to Novartis at a price of $9.324 per share, for an aggregate purchase price of approximately $20.0 million. In accordance with the terms and conditions of the 2023 Novartis Stock Purchase Agreement, we issued and sold these shares to Novartis on January 3, 2024.

Previous wording

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

Current wording

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

Previous wording

Net cash used in operating activities was $71.2 million during the six months ended June 30, 2025, compared to $27.2 million of net cash provided by operating activities during the six months ended June 30, 2024. The change was primarily due to a decrease in accounts receivable of $78.4 million during the 2024 period primarily from the receipt of the $80.0 million upfront payment under the 2023 Novartis Collaboration Agreement during the first quarter of 2024.

Current wording

Net cash used in operating activities was $102.2 million during the nine months ended September 30, 2025, compared to $0.8 million of net cash used in operating activities during the nine months ended September 30, 2024. The decrease was primarily due to our net loss during the nine months ended September 30, 2025 as compared to net loss during the nine months ended September 30, 2024. Our net loss increased in 2025, as we earned more revenue in 2024, primarily from the 2023 Neurocrine Collaboration Agreement.

Previous wording

Net cash provided by investing activities was $43.1 million during the six months ended June 30, 2025, compared to $132.0 million in cash used in investing activities during the six months ended June 30, 2024. The change was primarily due to decreased purchases of marketable securities during the six months ended June 30, 2025 as compared to the six months ended June 30, 2024.

Current wording

Net cash provided by investing activities was $78.0 million during the nine months ended September 30, 2025, compared to $67.7 million in cash used in investing activities during the nine months ended September 30, 2024. The increase was primarily due to decreased purchases of marketable securities during the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024, offset in part by lower proceeds from the sales and maturities of marketable securities.

Previous wording

Net cash provided by financing activities was $0.6 million during the six months ended June 30, 2025, compared to $113.4 million during the six months ended June 30, 2024. The change was primarily due to proceeds from the issuance of common stock and pre-funded warrants in connection with the underwritten public offering and the issuance of common stock in connection with the 2023 Novartis Stock Purchase Agreement during the first quarter of 2024.

Current wording

Net cash provided by financing activities was $0.7 million during the nine months ended September 30, 2025, compared to $113.4 million during the nine months ended September 30, 2024. The change was primarily due to proceeds from the issuance of common stock and pre-funded warrants in connection with the underwritten public offering and the issuance of common stock in connection with the 2023 Novartis Stock Purchase Agreement during the first quarter of 2024.

Previous wording

Our expenses increased duringthe six months ended June 30, 2025, as compared withthe six months ended June 30, 2024, as our development programs progressed, we advanced VY7523 into clinical development, and due to charges related to our reduction in force. We expect our expenses to continue to increase as we continue the research and development of, conduct clinical trials of, and seek marketing approval for our product candidates, including in our ongoing Phase 1 MAD clinical trial to evaluate VY7523 in 2025, and as we continue to enter into or conduct activities in connection with our collaboration agreements. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant expenses related to program sales, marketing, manufacturing and distribution to the extent that such sales, marketing and distribution are not the responsibility of potential collaborators. Furthermore, we expect to incur increasing costs associated with operating as a public company, executing financial statement controls, satisfying regulatory and quality standards, fulfilling healthcare compliance requirements, and maintaining product, clinical trial and directors’ and officers’ liability insurance coverage.

Current wording

Our expenses increased duringthe nine months ended September 30, 2025, as compared withthe nine months ended September 30, 2024, as our development programs progressed, we advanced VY7523 into clinical development, and due to charges related to our reduction in force. We expect our expenses to continue to increase as we continue the research and development of, conduct clinical trials of, and seek marketing approval for our product candidates, including in our ongoing Phase 1 MAD clinical trial to evaluate VY7523, and as we continue to enter into or conduct activities in connection with our collaboration agreements. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant expenses related to program sales, marketing, manufacturing and distribution to the extent that such sales, marketing and distribution are not the responsibility of potential collaborators. Furthermore, we expect to incur increasing costs associated with operating as a public company, executing financial

Previous wording

As of June 30, 2025, we had cash, cash equivalents, and marketable securities of $262.0 million. Based upon our current operating plans, we expect that our existing cash, cash equivalents, and marketable securities at June 30, 2025, along with amounts expected to be received as reimbursement for development costs under our collaboration and license agreements with Neurocrine and Novartis, and interest income, to be sufficient to meet our planned operating expenses and capital expenditure requirements into 2028. Our future capital requirements will depend on many factors, including:

Current wording

As of September 30, 2025, we had cash, cash equivalents, and marketable securities of $229.0 million. Based upon our current operating plans, we expect that our existing cash, cash equivalents, and marketable securities at September 30, 2025, along with amounts expected to be received as reimbursement for development costs under our collaboration and license agreements with Neurocrine and Novartis, and interest income, to be sufficient to meet our planned operating expenses and capital expenditure requirements into 2028. Our future capital requirements will depend on many factors, including:

Previous wording

Identifying potential product candidates and conducting preclinical studies and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete. We may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our product revenues, if any, and any commercial milestone payments or royalty

Current wording

Identifying potential product candidates and conducting preclinical studies and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete. We may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our product revenues, if any, and any commercial milestone payments or royalty payments under our collaboration agreements, if any, will be derived from sales of products that may not be commercially available for many years, if at all. Accordingly, we will need to continue to rely on additional financing and business development transactions to achieve our business objectives. Adequate additional financing may not be available to us on acceptable terms, or at all.
Rearranged current paragraphs (4)

Current report — wording carried from elsewhere

The 2019 Neurocrine Collaboration Agreement provides for aggregate development milestone payments from Neurocrine to us for the research, development, manufacture, and commercialization of gene therapy products, or the 2019 Collaboration Products, under the FA Program of up to $195.0 million. We may be entitled to receive aggregate commercial milestone payments for each 2019 Collaboration Product of up to $275.0 million, subject to an aggregate

Current report — wording carried from elsewhere

Agreement, $4.2 million of collaboration revenue in connection with the 2023 Novartis Collaboration Agreement, $3.5 million of collaboration revenue from the 2019 Neurocrine Collaboration Agreement, and $0.5 million of other collaboration revenue.

Current report — wording carried from elsewhere

During the three months ended September 30, 2024, we recognized collaboration revenue in connection with the following agreements:

Current report — wording carried from elsewhere

statement controls, satisfying regulatory and quality standards, fulfilling healthcare compliance requirements, and maintaining product, clinical trial and directors’ and officers’ liability insurance coverage.
Rearranged previous paragraphs (3)

Previous report — wording carried elsewhere

milestones for either of the two 2019 Discovery Programs of up to $130.0 million per 2019 Discovery Program or any commercial milestones or royalties related thereto.

Previous report — wording carried elsewhere

development expenses for the six months ended June 30, 2025 and 2024. All amounts for the six months ended June 30, 2024 have been reclassified to conform to the current year’s presentation.

Previous report — wording carried elsewhere

payments under our collaboration agreements, if any, will be derived from sales of products that may not be commercially available for many years, if at all. Accordingly, we will need to continue to rely on additional financing and business development transactions to achieve our business objectives. Adequate additional financing may not be available to us on acceptable terms, or at all.