Voyager Therapeutics, Inc.

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q


☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2026


☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from ______ to ______

Commission file number: 001-37625

Voyager Therapeutics, Inc.

(Exact name of Registrant as specified in its charter)


Delaware		46-3003182
(State or other jurisdiction of incorporation or organization)		(I.R.S. Employer Identification No.)

75 Hayden Avenue, Lexington, Massachusetts		02421
(Address of principal executive offices)		(Zip Code)

(857) 259-5340

(Registrant’s telephone number, including area code)

Not Applicable

(Former name, former address and former fiscal year, if changed since last report)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, $0.001 par value	VYGR	Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ⌧ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ⌧ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.



Large accelerated filer	☐	Accelerated filer	☒
Non-accelerated filer	☐	Smaller reporting company	☒
		Emerging growth company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

The number of outstanding shares of the registrant’s common stock, par value $0.001 per share, as of April 30, 2026, was 60,421,287.

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Forward-Looking Statements

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and expected market growth, are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.

The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “contemplate,” “anticipate,” “goals,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, among other things, statements about:

●

our plans to develop and commercialize our proprietary adeno-associated virus, or AAV, gene therapy, antibody, non-viral therapeutic, and small molecule product candidates;

●

our ability to continue to develop our proprietary gene therapy platform technologies, including our TRACER^TM(Tropism Redirection of AAV by Cell-type-specific Expression of RNA) discovery platform, our non-viral therapeutics platform, including VOYAGER NEUROSHUTTLE^TM, and our proprietary antibody, small molecule, gene therapy, shuttled or vectorized antibody, and non-viral therapeutic programs;

●

our ability to identify and optimize product candidates, proprietary AAV capsids, and non-viral blood-brain-barrier shuttles;

●

our strategic collaborations and licensing agreements with, and funding from, our collaboration partners Neurocrine Biosciences, Inc. and Novartis Pharma AG and our licensee Alexion, AstraZeneca Rare Disease (successor-in-interest to former licensee Pfizer Inc.);

●

our collaboration with Transition Bio, Inc. to advance small molecules targeting TDP-43 to treat amyotrophic lateral sclerosis and frontotemporal dementia with TDP-43 pathology;

●

our ongoing and planned clinical trials, preclinical development efforts, related timelines and studies, including our plans to generate initial data from the Phase 1 multiple ascending dose clinical trial of VY7523 in early Alzheimer’s disease patients in the second half of 2026, complete the investigational new drug, or IND, application process for the VY1706 program in the second quarter of 2026, and, subject to successful IND clearance, initiate a clinical trial for the VY1706 program in the second half of 2026;

●

our ability to enter into future collaborations, strategic alliances, or option and license arrangements;

●

the timing of and our ability to submit applications and obtain and maintain regulatory approvals for our product candidates, including the ability to submit IND applications for our programs;

●

our belief as to potential outcomes of our clinical development activities, and plans and potential outcomes with respect to interactions with regulatory authorities;

●

our estimates regarding future revenue, expenses, contingent liabilities, existing cash resources, capital requirements and cash runway;

●

our intellectual property position and our ability to obtain, maintain and enforce intellectual property protection for our proprietary assets;

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●

our estimates regarding the size of the potential markets for our product candidates and our ability to serve those markets;

●

our need for and the timing of additional funding and our plans and ability to raise additional capital, including through equity offerings, debt financings, collaborations, strategic alliances, and option and license arrangements;

●

our competitive position and the success of competing products that are or might become available for the indications that we are pursuing;

●

the impact of government laws and regulations, including in the United States, the European Union, and other important geographies such as Japan; and

●

our ability to control costs and prioritize our product candidate pipeline and platform development objectives successfully in connection with our strategic initiatives.

These forward-looking statements are only predictions, and we may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements. You should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. We have based these forward-looking statements largely on our current expectations and projections about future events and trends that we believe may affect our business, financial condition and operating results. We have included important factors in the cautionary statements included in our Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 9, 2026, particularly in “Part I, Item 1A — Risk Factors,” and, if applicable, this Quarterly Report on Form 10-Q, particularly in “Part II, Item 1A — Risk Factors,” that could cause actual future results or events to differ materially from the forward-looking statements that we make. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, strategic collaborations, licenses, joint ventures or investments we may make or enter into.

You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q with the understanding that our actual future results may be materially different from what we expect. 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 obtained the statistical and other industry and market data in this Quarterly Report on Form 10-Q and the documents we have filed as exhibits to the Quarterly Report on Form 10-Q from our own internal estimates and research, as well as from industry and general publications and research, surveys, studies and trials conducted by third parties. Some data is also based on our good faith estimates, which are derived from management’s knowledge of the industry and independent sources. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, while we believe the market opportunity information included in this Quarterly Report on Form 10-Q and the documents we have filed as exhibits to the Quarterly Report on Form 10-Q is reliable and is based upon reasonable assumptions, such data involves risks and uncertainties and are subject to change based on various factors, including those discussed under “Risk Factors” and in the documents we have filed as exhibits to the Quarterly Report on Form 10-Q. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this Quarterly Report on Form 10-Q, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain, and investors are cautioned not to unduly rely upon these statements.

We own various U.S. federal trademark registrations and applications and unregistered trademarks, including our corporate logo. This Quarterly Report on Form 10-Q and the documents filed as exhibits to the Quarterly Report on Form 10-Q contain references to trademarks, service marks and trade names referred to in this Quarterly Report on Form 10-Q and the information incorporated herein, including logos, artwork, and other visual displays, that may appear

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PART I. FINANCIAL INFORMATION

Voyager Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(amounts in thousands, except share and per share data)

(unaudited)


	March 31,		December 31,
	2026		2025
Assets
Current assets:
Cash and cash equivalents	$	43,314	$	65,300
Marketable securities		98,165		131,147
Accounts receivable		1,494		1,761
Related party collaboration receivable		151		151
Prepaid expenses and other current assets		3,857		4,328
Total current assets		146,981		202,687
Property and equipment, net		12,218		13,136
Restricted cash		2,736		2,736
Marketable securities, non-current		30,181		5,244
Operating lease, right-of-use assets		27,209		28,478
Total assets	$	219,325	$	252,281
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable	$	2,723	$	5,013
Accrued expenses		6,972		12,098
Operating lease liabilities		7,363		7,840
Deferred revenue		395		1,590
Total current liabilities		17,453		26,541
Operating lease liabilities, net of current portion		27,203		28,659
Other non-current liabilities		1,000		1,000
Total liabilities		45,656		56,200
Commitments, contingencies, and other liabilities (see note 8)
Stockholders’ equity:
Preferred stock, $0.001 par value: 5,000,000 shares authorized at March 31, 2026 and December 31, 2025; no shares issued and outstanding at March 31, 2026 and December 31, 2025		—		—
Common stock, $0.001 par value: 120,000,000 shares authorized at March 31, 2026 and December 31, 2025; 60,310,526 and 59,047,860 shares issued and outstanding at March 31, 2026 and December 31, 2025, respectively		60		59
Additional paid-in capital		647,714		641,895
Accumulated other comprehensive (loss) income		(263)		32
Accumulated deficit		(473,842)		(445,905)
Total stockholders’ equity		173,669		196,081
Total liabilities and stockholders’ equity	$	219,325	$	252,281

The accompanying notes are an integral part of these condensed consolidated financial statements.

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Voyager Therapeutics, Inc.

Condensed Consolidated Statements of Operations and Comprehensive Loss

(amounts in thousands, except share and per share data)

(unaudited)


	Three Months Ended
	March 31,
	2026		2025
Collaboration revenue	$	2,593	$	6,473
Operating expenses:
Research and development		24,602		31,526
General and administrative		8,261		9,640
Total operating expenses		32,863		41,166
Operating loss		(30,270)		(34,693)
Other income, net:
Interest income		1,912		3,291
Other income		435		418
Total other income, net		2,347		3,709
Loss before income taxes		(27,923)		(30,984)
Income tax provision		14		37
Net loss	$	(27,937)	$	(31,021)
Other comprehensive (loss) income:
Net unrealized (loss) gain on available-for-sale securities		(295)		208
Total other comprehensive (loss) income		(295)		208
Comprehensive loss	$	(28,232)	$	(30,813)

Net loss per share, basic and diluted	$	(0.47)	$	(0.53)

Weighted-average common shares outstanding, basic and diluted		59,496,329		58,349,769

The accompanying notes are an integral part of these condensed consolidated financial statements.

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Voyager Therapeutics, Inc.

Condensed Consolidated Statements of Stockholders’ Equity

(amounts in thousands, except share data)

(unaudited)


						Accumulated
				Additional		Other
	Common Stock			Paid-In		Comprehensive		Accumulated		Stockholders'
	Shares	Amount		Capital		Loss		Deficit		Equity
Balance at December 31, 2024	54,731,316	$	55	$	626,296	$	(407)	$	(326,184)	$	299,760
Exercises of stock options	27,613		—		82		—		—		82
Vesting of restricted stock units	450,556		—		—		—		—		—
Stock-based compensation expense	—		—		3,673		—		—		3,673
Unrealized gain on available-for-sale securities, net of tax	—		—		—		208		—		208
Net loss	—		—		—		—		(31,021)		(31,021)
Balance at March 31, 2025	55,209,485	$	55	$	630,051	$	(199)	$	(357,205)	$	272,702

Balance at December 31, 2025	59,047,860		59		641,895		32		(445,905)		196,081
Exercises of stock options	87,081		—		294		—		—		294
Vesting of restricted stock units	545,015		—		—		—		—		—
Issuance of common stock from at-the-market sales agreement, net of issuance costs	630,570		1		3,037		—		—		3,038
Stock-based compensation expense	—		—		2,488		—		—		2,488
Unrealized gain on available-for-sale securities, net of tax	—		—		—		(295)		—		(295)
Net loss	—		—		—		—		(27,937)		(27,937)
Balance at March 31, 2026	60,310,526	$	60	$	647,714	$	(263)	$	(473,842)	$	173,669

The accompanying notes are an integral part of these condensed consolidated financial statements.

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Voyager Therapeutics, Inc.

Condensed Consolidated Statements of Cash Flows

(amounts in thousands)

(unaudited)


	Three Months Ended
	March 31,
	2026		2025
Cash flow from operating activities
Net loss	$	(27,937)	$	(31,021)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense		2,488		3,673
Depreciation		932		1,014
Amortization of premiums and discounts on marketable securities		(365)		(998)
Loss on disposal of fixed assets		—		30
Non-cash lease expense		1,269		1,221
Changes in operating assets and liabilities:
Accounts receivable		267		(119)
Related party collaboration receivable		—		189
Prepaid expenses and other current assets		471		868
Accounts payable		(2,290)		(1,362)
Accrued expenses		(5,126)		(5,118)
Operating lease liabilities		(1,933)		(1,730)
Deferred revenue		(1,195)		(4,542)
Net cash used in operating activities		(33,419)		(37,895)
Cash flow from investing activities
Purchases of property and equipment		(14)		(658)
Purchases of marketable securities		(43,441)		(76,924)
Proceeds from sales and maturities of marketable securities		51,556		118,783
Net cash provided by investing activities		8,101		41,201
Cash flow from financing activities
Proceeds from the exercise of stock options		294		82
Proceeds from the issuance of common stock from at-the-market sales agreement, net of issuance costs		3,038		—
Net cash provided by financing activities		3,332		82
Net (decrease) increase in cash, cash equivalents, and restricted cash		(21,986)		3,388
Cash, cash equivalents, and restricted cash, beginning of period		68,036		74,241
Cash, cash equivalents, and restricted cash, end of period	$	46,050	$	77,629

The accompanying notes are an integral part of these condensed consolidated financial statements.

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VOYAGER THERAPEUTICS INC.

NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

1. Nature of business

Voyager Therapeutics, Inc. (the “Company”) is a biotechnology company whose mission is to leverage the power of human genetics to modify the course of and ultimately cure neurological diseases. Its pipeline includes programs for Alzheimer’s disease; Friedreich’s ataxia; Parkinson’s disease; and multiple other diseases of the central nervous system (“CNS”). Its pipeline includes programs it wholly owns and programs it is advancing with licensees and collaborators, including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG (“Novartis”); Neurocrine Biosciences, Inc. (“Neurocrine”); and Transition Bio, Inc.

Many of the Company’s programs are derived from its TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) adeno-associated virus (“AAV”) capsid discovery platform, which it has used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. TRACER is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier (“BBB”) and enhanced CNS tropism in multiple species, including non-human primates. The Company is also developing a second, non-viral therapeutics platform focused on non-viral receptor-mediated transport across the BBB, the Voyager NeuroShuttle^TM platform.

The Company has a history of incurring annual net operating losses. As of March 31, 2026, the Company had an accumulated deficit of $473.8 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities and funding from fees, milestone payments, and cost reimbursements associated with its prior and current collaborations and license agreements.

As of March 31, 2026, the Company had cash, cash equivalents, and marketable securities of $171.7 million. Based upon its current operating plan, the Company expects that its existing cash, cash equivalents, and marketable securities at March 31, 2026, to be sufficient to meet the Company’s planned operating expenses and capital expenditure requirements for at least twelve months from the issuance of these condensed consolidated financial statements.

There can be no assurance that the Company will be able to obtain additional debt or equity financing on terms acceptable to the Company or generate product revenue or revenue from collaboration partners, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition.

2. Summary of significant accounting policies and basis of presentation

Basis of Presentation

The accompanying unaudited condensed consolidated financial statements of the Company have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. For further information, refer to the consolidated financial statements and footnotes included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025, as filed with the Securities and Exchange Commission on March 9, 2026. These interim condensed consolidated financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the Company’s financial position and results of operations for the periods presented. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP, which can be found in the Accounting Standards Codification and Accounting Standards Updates of the Financial Accounting Standards Board.

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Principles of Consolidation

The unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiary as disclosed in Note 2, Summary of Significant Accounting Policies and Basis of Presentation, within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025. Intercompany balances and transactions have been eliminated.

Use of Estimates

The preparation of condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the condensed consolidated financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, estimates related to revenue recognition, research and development accrued expenses, stock-based compensation expense, and income taxes. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions.

Summary of Significant Accounting Policies

There have been no changes in the Company’s significant accounting policies as described in Note 2, Summary of Significant Accounting Policies and Basis of Presentation, within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025.

3. Fair value measurements

Assets and liabilities measured at fair value on a recurring basis as of March 31, 2026 and December 31, 2025, are as follows:


			Quoted Prices		Significant
			in Active		Other		Significant
			Markets for		Observable		Unobservable
			Identical Assets		Inputs		Inputs
Assets	Total		(Level 1)		(Level 2)		(Level 3)

March 31, 2026	(in thousands)
Cash equivalents:
Money market funds	$	29,992	$	29,992	$	—	$	—
Marketable securities:
U.S. Treasury notes		57,393		57,393		—		—
U.S. Government agency securities		18,976		18,976		—		—
Corporate bonds		47,035		—		47,035		—
Commercial paper		4,942		—		4,942		—
Total cash equivalents and marketable securities	$	158,338	$	106,361	$	51,977	$	—
December 31, 2025
Cash equivalents:
Money market funds	$	51,161	$	51,161	$	—	$	—
Commercial paper		3,477		—		3,477		—
Marketable securities:
U.S. Treasury notes		72,805		72,805		—		—
U.S. Government agency securities		15,387		15,387		—		—
Corporate bonds		44,260		—		44,260		—
Commercial paper		3,939		—		3,939		—
Total cash equivalents and marketable securities	$	191,029	$	139,353	$	51,676	$	—

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The Company measures the fair value of money market funds, U.S. Treasury notes and U.S. Government agency securities based on quoted prices in active markets for identical securities. The Company measures the fair value of the Level 2 securities, including corporate bonds and commercial paper, based on recent trades of securities in inactive markets or based on quoted market prices of similar instruments and other significant inputs derived from or corroborated by observable market data.

4. Cash equivalents and available-for-sale marketable securities

Cash equivalents and available-for-sale marketable securities included the following as of March 31, 2026 and December 31, 2025:


					Unrealized Losses
	Amortized Cost		Unrealized Gains		Less than 12 months			Greater than 12 months	Fair Value

	(in thousands)
As of March 31, 2026
Cash equivalents:
Money market funds	$	29,992	$	—	$	—	$	—	$	29,992
Marketable securities:
U.S. Treasury notes		57,381		58		(6)		(40)		57,393
U.S. Government agency securities		19,024		7		(5)		(50)		18,976
Corporate bonds		47,134		1		(55)		(45)		47,035
Commercial paper		4,942		—		—		—		4,942
Total money market funds and marketable securities	$	158,473	$	66	$	(66)	$	(135)	$	158,338
As of December 31, 2025
Cash equivalents:
Money market funds	$	51,161	$	—	$	—	$	—	$	51,161
Commercial paper		3,477		—		—		—		3,477
Marketable securities:
U.S. Treasury notes		72,665		141		(1)		—		72,805
U.S. Government agency securities		15,376		18		—		(7)		15,387
Corporate bonds		44,251		19		(10)		—		44,260
Commercial paper		3,939		—		—		—		3,939
Total cash equivalents and marketable securities	$	190,869	$	178	$	(11)	$	(7)	$	191,029

All of the Company’s marketable securities as of March 31, 2026 and December 31, 2025, have a contractual maturity of two years or less.

The Company reviews investments whenever the fair value of an investment is less than the amortized cost and evidence indicates that an investment’s carrying amount is not recoverable within a reasonable period of time. In connection with these investments, the Company evaluates whether the decline in fair value has resulted from credit losses or other factors, considering the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security, among other factors. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security is compared to the amortized cost basis of the security. If the present value of cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss on the condensed consolidated balance sheet, limited by the amount that the fair value is less than the amortized cost basis. Any impairment that is not related to credit is recognized in other comprehensive (loss) income. Changes in the allowance for credit losses are recorded as a provision for (or reversal of) credit loss expense in other income, net within the condensed consolidated statement of operations and comprehensive (loss) income. Losses are charged against the allowance when the Company believes the uncollectability of an available-for-sale security is confirmed or when either of the criteria regarding intent or requirement to sell is met. No credit losses were recorded during the periods presented.

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As of March 31, 2026 and December 31, 2025, the Company held 57 and 20 marketable securities, respectively, that were in an unrealized loss position, representing $73.5 million and $26.6 million in fair value, respectively. The unrealized losses as of March 31, 2026 and December 31, 2025, were attributable to changes in interest rates and do not represent credit losses. The Company does not intend to sell these securities, and it is not more likely than not that it will be required to sell them before recovery of their amortized cost basis.

The following table provides a reconciliation of cash, cash equivalents, and restricted cash within the condensed consolidated balance sheets that sum to the total of the same amounts shown in the condensed consolidated statements of cash flows:


		As of March 31,		As of December 31,
	2026		2025

	(in thousands)
Cash and cash equivalents	$	43,314	$	65,300
Restricted cash included in deposits and other non-current assets		2,736		2,736
Total cash, cash equivalents, and restricted cash	$	46,050	$	68,036

Restricted cash balances for both periods presented are held in the form of money market accounts and represent collateral for the Company’s facility lease obligations.

5. Accrued expenses

Accrued expenses as of March 31, 2026 and December 31, 2025, consist of the following:


	As of March 31,		As of December 31,
	2026		2025

	(in thousands)
Employee compensation costs	$	3,307	$	9,091
Research and development costs		2,621		2,069
Accrued other		511		549
Professional services		533		389
Total	$	6,972	$	12,098

Employee compensation costs include $1.3 million in accrued severance costs related to a restructuring that occurred and was recognized in December 2025 but that remain partially unpaid as of March 31, 2026.

6. Lease obligation

Operating Leases

As of March 31, 2026, the Company has a lease for laboratory and office space at 75 Hayden Avenue in Lexington, Massachusetts (the “Lexington Facility”) through January 31, 2031, and a lease for additional office and laboratory space at 64 Sidney Street in Cambridge, Massachusetts (the “Cambridge Facility”) through November 30, 2026.

In August 2023, the Company entered into a first amendment (the “First Amendment”) to its existing lease for the Lexington Facility, pursuant to which the Company agreed to lease approximately 61,307 square feet of additional office and laboratory space. The commencement date for the First Amendment occurred on February 1, 2024. The expected contractual obligation under the First Amendment to the Company’s existing lease for the Lexington Facility is approximately $35.4 million, to be paid over the 7-year term of the lease.

The Company’s lease agreements require the Company to maintain a cash deposit or irrevocable letter of credit in the aggregate amount of $2.7 million payable to its landlords as security for the performance of its obligations under the leases. These amounts are recorded as restricted cash in the accompanying condensed consolidated balance sheets.

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In June 2024, the Company vacated the Cambridge Facility. The Company recorded an impairment charge of $2.8 million to operating expenses during the second quarter of 2024 as a result of the carrying value of the leased office and laboratory space asset group exceeding the undiscounted cash flows projected from a planned sublease of the facility. The impairment charge reduced the carrying value of the leased office and laboratory space asset group by $2.8 million. In August 2024, the Company executed the sublease of the Cambridge Facility (the “Sublease Agreement”). Payments received under the Sublease Agreement are included in other income in the condensed consolidated statements of operations and comprehensive (loss) income. Subject to certain conditions set forth therein, the Sublease Agreement terminates simultaneously with the Company’s lease for the Cambridge Facility.

Total lease cost for operating leases of approximately $1.9 million was incurred during each of the three months ended March 31, 2026 and 2025. As of March 31, 2026, the weighted average remaining lease term was 3.5 years and the weighted average incremental borrowing rate used to determine the operating lease liability was 6.5%.

The following table summarizes the operating sublease income generated under the Sublease Agreement for the three months ended March 31, 2026 and 2025:


	Three Months Ended
	March 31,
	2026			2025

		(in thousands)
Operating sublease income	$	402	$	440

Future minimum lease payments due under operating leases are as follows:


		Total Minimum
Year Ending December 31,		Lease Payments
		(in thousands)
Remainder of 2026		$	7,461
2027			7,763
2028			7,996
2029			8,235
2030			8,483
Thereafter			227
Total future minimum lease payments		$	40,165
Less: imputed interest			(5,599)
Total operating lease liabilities		$	34,566
Reported as:
Operating lease liabilities		$	7,363
Operating lease liabilities, net of current portion			27,203
Total operating lease liabilities		$	34,566

7. Commitments, contingencies and other liabilities

Other Agreements

In 2016, the Company entered into a research and development funding arrangement with a non-profit organization that provided up to $4.0 million in funding to the Company upon the achievement of clinical and development milestones. The agreement, as amended in 2022, provides that the Company is obligated to repay amounts received under certain circumstances, including termination of the agreement, and to pay an amount up to 2.6 times the funding received upon successful development and commercialization of any products developed. In 2017, the Company earned a milestone payment of $1.0 million. The Company evaluated the arrangement and concluded that it represents a research and development financing arrangement as it is probable that the Company will repay amounts received under

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the arrangement. As a result, the $1.0 million is recorded as a non-current liability in the condensed consolidated balance sheet.

Litigation

The Company was not a party to any material legal matters or claims as of March 31, 2026 or December 31, 2025. The Company did not have contingency reserves established for any litigation liabilities as of March 31, 2026 or December 31, 2025.

8. Significant agreements

The Company’s significant agreements are described in Note 9, Significant Agreements, within the “Notes to Consolidated Financial Statements” accompanying the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025. During the three months ended March 31, 2026, there were no material changes to the Company’s collaboration agreements or option and license agreements, and the Company did not enter into any new collaboration or license agreements. The Company recorded collaboration revenue of $2.6 million and $6.5 million during the three months ended March 31, 2026 and 2025, respectively.

Related Party Collaboration Receivable and Deferred Revenue

The following table presents changes in the balances of the Company’s related party collaboration receivable and contract liabilities (reflected as deferred revenue in the condensed consolidated balance sheets) for the collaboration and license agreement the Company entered into with Neurocrine in January 2023 (the “2023 Neurocrine Collaboration Agreement”) and the collaboration and license agreement the Company entered into with Neurocrine in January 2019 (the “2019 Neurocrine Collaboration Agreement”) during the three months ended March 31, 2026:


	Balance at						Balance at
	December 31, 2025		Additions		Deductions		March 31, 2026

		(in thousands)
Related party collaboration receivables	$	151	$	151	$	(151)	$	151
Contract liabilities:
Deferred revenue	$	1,590	$	—	$	(1,195)	$	395

The change in the related party collaboration receivable balance for the three months ended March 31, 2026, is primarily driven by amounts owed to the Company for research and development services provided, offset by amounts collected during the period, under the 2023 and 2019 Neurocrine Collaboration Agreements. Deferred revenue activity for the period includes the recording of $1.2 million of collaboration revenue recognized on the proportional performance model during the period for the 2023 and 2019 Neurocrine Collaboration Agreements.

9. Stock-based compensation

Stock-Based Compensation Expense

Total compensation cost recognized for all stock-based compensation awards in the condensed consolidated statements of operations and comprehensive (loss) income was as follows:


	Three Months Ended
	March 31,
	2026		2025

	(in thousands)
Research and development	$	660	$	1,485
General and administrative		1,828		2,188
Total stock-based compensation expense	$	2,488	$	3,673

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Employee Stock Purchase Plan

In October 2015, the Company’s board of directors (the “Board”) and stockholders approved the 2015 Employee Stock Purchase Plan (the “2015 ESPP”). Under the 2015 ESPP, all full-time employees of the Company are eligible to purchase common stock of the Company twice per year, at the end of each six-month payment period. During each payment period, eligible employees who so elect, may authorize payroll deductions in an amount of 1% to 10% (whole percentages only) of the employee’s base pay for each payroll period. At the end of each payment period, the accumulated deductions are used to purchase shares of common stock from the Company at a discount. A total of 262,362 shares of common stock were initially authorized for issuance under the 2015 ESPP.

On March 18, 2025, the Board adopted, and on June 3, 2025, the stockholders approved, the Amended and Restated 2015 Employee Stock Purchase Plan (together with the 2015 ESPP, the “ESPP”) to (i) eliminate the evergreen provision in the 2015 ESPP and (ii) expand the class of eligible employees by eliminating the requirement that participants must have completed six months of employment.

As of March 31, 2026, there were 1,809,493 shares available for future purchase under the ESPP.

2025 Stock Incentive Plan

On March 18, 2025, the Board adopted, and on June 3, 2025, the stockholders approved, the 2025 Stock Incentive Plan (the “2025 Plan”). Under the 2025 Plan, the Company may issue awards for up to a number of shares of common stock equal to the sum of: (i) 3,631,952 shares of common stock; and (ii) such additional number of shares of common stock (up to 14,190,976 shares) as is equal to the sum of (x) the number of shares of common stock reserved for issuance under the Company’s 2015 Stock Option and Incentive Plan (the “2015 Plan”) that remained available for grant thereunder immediately prior to the date that the 2025 Plan was approved by our stockholders and (y) the number of

shares of common stock subject to awards granted under the 2015 Plan that were outstanding as of the date that the 2025 Plan was approved by our stockholders and which awards expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right (subject, however, in the case of incentive stock options, to any limitations under the Internal Revenue Code of 1986, as amended, and any regulations thereunder).

The Company intends to utilize the 2025 Plan to grant equity awards to the Company’s employees, non-employee directors, consultants, and advisors in order to recruit, incentivize, retain and reward those who are critical to the Company’s success. No new awards will be granted under the 2015 Plan.

As of March 31, 2026, there were 4,799,738 shares available under the 2025 Plan.

Restricted Stock Units and Performance Restricted Stock Units

A summary of the status of and changes in unvested restricted stock unit (“RSU”) activity and performance-based restricted stock unit (“PRSU”) activity under the Company’s equity award plans for the three months ended March 31, 2026, was as follows:


		Weighted Average Grant
	Units	Date Fair Value Per Unit
Unvested as of December 31, 2025	2,296,920	$	5.39
Granted	708,348		3.68
Vested	(545,015)		6.05
Forfeited	(351,626)		5.89
Outstanding as of March 31, 2026	2,108,627	$	4.56

All RSUs granted in the three months ended March 31, 2026, vest in equal amounts, annually over three years. Stock-based compensation for RSUs is based on the fair value of the Company’s common stock on the date of grant and is recognized over the vesting period.

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As of March 31, 2026, the Company had unrecognized stock-based compensation expense related to its unvested RSUs (excluding PRSUs) of $6.1 million, which is expected to be recognized over the remaining average vesting period of 2.1 years.

During the three months ended March 31, 2025, in connection with the Company’s annual compensation cycle, the Company granted PRSUs to members of the Company’s senior leadership team to more closely align with long-term performance incentives. The awards vest as to a certain number of PRSUs upon the achievement of specified clinical performance milestones over multiple years. Stock-based compensation for PRSUs is based on the fair value of the Company’s common stock on the date of grant and will be recognized at the time the achievement of the specified performance milestones becomes probable. The Company did not recognize any stock-based compensation expense in connection with the PRSUs during the three months ended March 31, 2026.

As of March 31, 2026, the Company had unrecognized stock-based compensation expense related to its unvested PRSUs of $2.3 million. The Company will recognize a cumulative catch-up adjustment in the period in which the specified performance condition is considered probable.

Stock Options

The following is a summary of stock option activity for the three months ended March 31, 2026:


		Weighted		Remaining	Aggregate
		Average		Contractual	Intrinsic Value
	Shares	Exercise Price		Life (in years)	(in thousands)
Outstanding as of December 31, 2025	9,613,881	$	7.26	6.8	$	1,251
Granted	2,328,200		3.72
Exercised	(87,450)		3.39
Cancelled or forfeited	(756,056)		6.14
Outstanding as of March 31, 2026	11,098,575	$	6.63	6.8	$	1,452
Exercisable as of March 31, 2026	6,603,226	$	7.93	5.3	$	880

As of March 31, 2026, the Company had unrecognized stock-based compensation expense related to its unvested stock options of $13.3 million, which is expected to be recognized over the remaining weighted-average vesting period of 2.8 years.

10. Net loss per share

For purposes of the diluted net loss per share, unvested restricted common stock awards, unvested RSUs and PRSUs, and outstanding stock options are considered to be potentially dilutive securities. Unvested RSUs and PRSUs and outstanding stock options were excluded from the calculation of diluted net loss per share in the three months ended March 31, 2026 and 2025, because their effect would be anti-dilutive and therefore, basic and diluted net loss per share were the same for the three months ended March 31, 2026 and 2025.

The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share because to include them would be anti-dilutive:


	As of March 31,
	2026	2025
Unvested RSUs and PRSUs	2,108,627	2,849,278
Outstanding stock options	11,098,575	10,522,083
Total	13,207,202	13,371,361

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11. Related-party transactions

During the three months ended March 31, 2026, the Company received scientific advisory board and other scientific advisory services from one of its prior executives, Dinah Sah, Ph.D., the Company’s former Chief Scientific Officer. The total amount of fees paid to Dr. Sah for services provided during the three months ended March 31, 2026 and 2025 was $0.1 million and 0.2 million, respectively.

Under both the 2019 Neurocrine Collaboration Agreement and the 2023 Neurocrine Collaboration Agreement, the Company and Neurocrine have agreed to conduct research, development and commercialization activities for certain of the Company’s AAV gene therapy product candidates. Any amounts due from Neurocrine are reflected as related party collaboration receivable. Amounts received from Neurocrine that have not yet been recognized as revenue are reflected as deferred revenue. Refer to Note 8 for further description of these account balances.

For the three months ended March 31, 2026 and 2025, the Company recognized collaboration revenue of $1.4 million and $5.0 million, respectively, related to the 2023 and 2019 Neurocrine Collaboration Agreements.

12. Segment Information

The Company’s Chief Operating Decision Maker (“CODM”), the Company’s Chief Executive Officer, views the Company’s operations and manages its business as a single operating segment, which is the business of developing and commercializing genetic medicines. The Company therefore has one reportable segment.

The CODM reviews the segment’s profit or loss based on net loss reported on the condensed consolidated statement of operations and comprehensive loss and considers forecast-to-actual variances quarterly for expenses that are deemed significant in making resource allocation decisions and assessing performance. Further, the CODM reviews the segment’s assets based on total assets reported on the condensed consolidated balance sheets. All long-lived assets are held in the United States.

The CODM views specific categories within research and development expenses and general and administrative expenses in totality as significant. Further, the CODM views the external research and development expenses of specific programs as significant given the impact on achieving the Company’s corporate goals. The following table reconciles reported collaboration revenue and segment expenses to net loss under the significant expense principle for the three months ended March 31, 2026 and 2025:


	Three Months Ended March 31,
	2026		2025
Collaboration revenue	$	2,593	$	6,473
External research and development ⁽¹⁾:
Anti-tau antibody program (VY7523)		2,377		4,249
Tau silencing gene therapy program (VY1706)		5,636		3,174
SOD1 silencing gene therapy program		—		1,886
Partnered programs ⁽²⁾		672		991
Other programs and platforms ⁽³⁾		1,699		3,181
Internal research and development ⁽⁴⁾		8,093		9,958
Facilities and other research and development ⁽⁵⁾		6,125		8,087
General and administrative ⁽⁵⁾		8,261		9,640
Interest income		1,912		3,291
Other income		435		418
Income tax provision		14		37
Net loss	$	(27,937)	$	(31,021)

(1)

External research and development is allocated to the Company’s programs and platforms and includes laboratory supplies and non-employee consultant and contractor costs.

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ITEM 2. 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 in conjunction with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial information and the notes thereto included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, which was filed with the Securities and Exchange Commission, or the SEC, on March 9, 2026.

Our actual results and timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this Quarterly Report on Form 10-Q. The following information and any forward-looking statements should be considered in light of factors discussed in Part I, Item 1A, "Risk Factors" of our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, and, if applicable, those included under Part II, Item 1A of our Quarterly Reports on Form 10-Q, that could cause actual future results or events to differ materially from the forward-looking statements that we make.

Overview

We are a biotechnology company whose mission is to leverage the power of human genetics to modify the course of and ultimately cure neurological diseases. Our pipeline includes programs for Alzheimer’s disease, or AD; Friedreich’s ataxia, or FA; Parkinson’s disease, or PD; and multiple other diseases of the central nervous system, or CNS. Many of our programs are derived from our TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) adeno-associated virus, or AAV, capsid discovery platform, which we have used to generate novel capsids, or TRACER Capsids, and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous, or IV, dosing. Some of our programs are wholly-owned, and some are advancing with licensees and collaborators, including Alexion, AstraZeneca Rare Disease, or Alexion; Novartis Pharma AG, or Novartis; Neurocrine Biosciences, Inc., or Neurocrine; and Transition Bio, Inc., or Transition Bio.

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: VY1706, a tau silencing gene therapy for AD, and VY7523, an anti-tau antibody for AD. 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, or vg/kg, 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 the progression of AD. We completed investigational new drug-enabling good laboratory practices, or GLP, toxicology in the first quarter of 2026. Having had a Type C communication with the FDA in the first quarter of 2026, we anticipate completion of the investigational new drug, or IND, application process for the VY1706 program in the second quarter of 2026 and, subject to successful IND clearance, initiation of a clinical trial for the VY1706 program in the second half of 2026. 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 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. Enrollment in this MAD clinical trial was completed in the fourth quarter of 2025, and we expect initial tau positron emission tomography imaging efficacy data in the second half of 2026.

Our proprietary pipeline also includes an early research initiative to develop a non-viral therapeutic that leverages our proprietary Voyager NeuroShuttle^TM platform for the treatment of an undisclosed neurological disease.

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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 frataxin (FXN) gene therapy program for FA, or the FA Program, and a glucosylceramidase beta 1 gene therapy program that will focus on Gaucher disease and PD, or the GBA1 Program. Neurocrine has stated that, pending successful FDA IND clearance, it intends to initiate a clinical trial with NBIB-’223 for FA in the second half of 2026. In preclinical studies, IV-administered NBIB-’223 resulted in protein expression in the brain and heart, and GLP toxicology studies for NBIB-’223 are now complete. In May 2026, FDA granted orphan drug designation to NBIB-’223 for the treatment of FA. Neurocrine has informed us that it continues to progress the GBA1 Program in addition to three gene therapy programs for which we do not have opt-in rights. We maintain options to co-develop and co-commercialize the FA Program, under which profits and losses of the program would be allocated 60% to Neurocrine and 40% to us, and the GBA1 Program, under which profits and losses of the program would be allocated 50% to Neurocrine and 50% to us, in each case in the U.S.

Our third opt-in program is our collaboration with Transition Bio to develop selective small molecules for the treatment of amyotrophic lateral sclerosis, or ALS, and frontotemporal dementia with TDP-43 pathology. We have an option for an exclusive license to the worldwide rights to develop and commercialize this program, which is in the research stage.

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

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, or SMA, and Huntington’s disease, or HD. We have also licensed capsids to Novartis for one undisclosed CNS target 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 $6.8 billion in milestone payments across the partnered portfolio, including $2.4 billion in potential development milestone payments, as well as royalties.

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.

We have leveraged our knowledge of BBB receptors, discovered using TRACER, to develop a second delivery platform called Voyager NeuroShuttle^TM. Voyager NeuroShuttle is a non-viral delivery platform leveraging novel receptor-binding molecules to transport multiple modalities of neurotherapeutics across the BBB. 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, or TfR, 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, a proof-of-concept study using anti-amyloid antibodies demonstrated that the ALPL-NeuroShuttle showed similar target engagement to a TfR shuttle. We initiated a discovery program using ALPL-VYGR-NeuroShuttle to target an undisclosed neurological disease.

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the Novartis License Options, to license TRACER Capsids, or the Novartis Licensed Capsids, for exclusive use in programs targeting three specified genes, or the Initial Novartis Targets, to develop and commercialize AAV gene therapy candidates comprised of Novartis Licensed Capsids and payloads directed to the Initial Novartis Targets, or the Novartis Initial Licensed Products. Effective as of March 1, 2023, Novartis exercised its Novartis License Options to license TRACER Capsids for use in gene therapy programs against two undisclosed Initial Novartis Targets. Upon Novartis’ exercise of the two Novartis License Options for Initial Novartis Targets, we granted Novartis a target-exclusive, worldwide license, with the right to sublicense, under certain of our intellectual property, the rights to develop and commercialize the applicable Novartis Licensed Capsid as incorporated into Novartis Initial Licensed Products. We also agreed to provide certain additional know-how to enable Novartis to exploit the Novartis Licensed Capsid and a payload directed to the applicable Initial Novartis Target for use in a Novartis Initial Licensed Product. Novartis elected not to license a capsid for one Initial Novartis Target prior to the expiration of the applicable Novartis License Option. As a result, the non-exclusive research license that we granted to Novartis in connection with this third Initial Novartis Target terminated, and all capsid rights with respect to that Initial Novartis Target returned to us.

On September 3, 2024, we entered into an amendment, or the Novartis Amendment, to the 2022 Novartis Option and License Agreement. Pursuant to the Novartis Amendment, we agreed to amend the 2022 Novartis Option and License Agreement to incorporate the grant to Novartis of a direct license, or a Novartis Direct License, to a TRACER Capsid, or the Novartis Direct Licensed Capsid, for exclusive use with a certain gene, or the Novartis Direct License Target, to develop and commercialize the Novartis Direct Licensed Capsid as incorporated into AAV gene therapy candidates comprised of the Novartis Direct Licensed Capsid and a payload directed to the Novartis Direct License Target, or the Novartis Direct Licensed Products. We refer to (a) the two Initial Novartis Targets for which Novartis has exercised its Novartis License Options and the Novartis Direct License Target collectively as Novartis Licensed Targets, and (b) the Novartis Initial Licensed Products and Novartis Direct Licensed Products collectively as Novartis Licensed Products. As a result of the Novartis Amendment, the Novartis Direct License Target was deemed a Licensed Target under the 2022 Novartis Option and License Agreement, as such term is defined therein, and the Novartis Direct License became subject to all other terms and conditions applicable to other licenses granted to Novartis under the 2022 Novartis Option and License Agreement. In connection with the Novartis Amendment, the parties acknowledged that Novartis’ prior rights to exercise options for any initial targets and additional targets as described in the 2022 Novartis Option and License Agreement, other than those that had previously been exercised, had expired as of the effective date of the Novartis Amendment.

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.

Following the termination of the two programs, the licenses we granted to Novartis regarding the applicable Initial Novartis Target and the Novartis Direct License Target expired, and all intellectual property rights that we licensed to Novartis with respect to such programs reverted to us in accordance with the 2022 Novartis Option and License Agreement. As of the Effective Partial Termination Date, we are no longer eligible to receive the milestone payments or royalties associated 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 Initial Novartis Target.

We are eligible to receive specified development, regulatory, and commercialization milestone payments of up to an aggregate of $125.0 million for the first Novartis Initial Licensed Product for the remaining Initial Novartis Target for which a Novartis License Option has been exercised to achieve the corresponding milestone. We are eligible to receive (a) specified sales milestone payments of up to an aggregate of $175.0 million per Novartis Licensed Product directed to the remaining Initial Novartis Target and (b) tiered, escalating royalties in the mid-to high-single-digit percentages based on annual net sales of each Novartis Licensed Product directed to the remaining Initial Novartis Target. The remaining Initial Novartis Target is distinct from targets in our wholly-owned and partnered pipeline. For a further description of the 2022 Novartis Option and License Agreement, refer to Note 9, Significant Agreements, to our

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consolidated financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, under the caption “2022 Novartis Option and License Agreement.”

2023 Neurocrine Collaboration Agreement

On January 8, 2023, we entered into a collaboration and license agreement with Neurocrine, or the 2023 Neurocrine Collaboration Agreement, for the research, development, manufacture and commercialization of the GBA1 Program, and three new programs focused on the research, development, manufacture and commercialization of gene therapies designed to address CNS diseases or conditions associated with rare genetic targets, or the 2023 Discovery Programs, and, collectively with the GBA1 Program, the 2023 Neurocrine Programs. With the exception of one preclinical study where we agreed to share costs, Neurocrine has agreed to be responsible for all costs we incur in conducting preclinical development activities for each 2023 Neurocrine Program, in accordance with workplans and budgets agreed upon by a Joint Steering Committee, or the JSC.

The 2023 Neurocrine Collaboration Agreement provides for aggregate development milestone payments from Neurocrine to us for the gene therapy products, or the 2023 Collaboration Products, under (a) the GBA1 Program of up to $985.0 million; and (b) each of the three 2023 Discovery Programs of up to $175.0 million for each 2023 Discovery Program. We may be entitled to receive aggregate commercial milestone payments for up to two 2023 Collaboration Products under the GBA1 Program of up to $950.0 million per 2023 Collaboration Product and for one 2023 Collaboration Product under each 2023 Discovery Program of up to $275.0 million per 2023 Discovery Program. We agreed to forfeit certain milestones and royalties on net sales in the United States if we exercise our option to co-develop and co-commercialize 2023 Collaboration Products in the GBA1 Program in the United States upon the occurrence of a specified event.

Neurocrine has also agreed to pay us tiered royalties, based on future net sales of the 2023 Collaboration Products. Such royalty percentages, for net sales in and outside the United States, range from (a) for the GBA1 Program, the low double-digits to twenty and the high single-digits to mid-teens, respectively, and (b) for each 2023 Discovery Program, high single-digits to mid-teens and mid-single digits to low double-digits, respectively. For a further description of the 2023 Neurocrine Collaboration 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, 2025, under the caption “2023 Neurocrine Collaboration Agreement.”

2019 Neurocrine Collaboration Agreement

In January 2019, we entered into the 2019 Neurocrine Collaboration Agreement for the research, development and commercialization of certain of our AAV gene therapy products. Under the 2019 Neurocrine Collaboration Agreement, we agreed to collaborate on the conduct of four collaboration programs, which we refer to collectively as the 2019 Neurocrine Programs: the NBIb-1817 (VY-AADC) program for the treatment of PD, or the VY-AADC Program; the FA Program; and two other programs, or the 2019 Discovery Programs. Effective August 2, 2021, Neurocrine terminated the VY-AADC Program under the 2019 Neurocrine Collaboration Agreement, and the license granted by us to Neurocrine expired and we regained worldwide intellectual property rights regarding such program.

On April 30, 2025, as a result of Neurocrine no longer prioritizing the two 2019 Discovery Programs, the JSC mutually agreed to discontinue the activities associated with such programs, and to return the rights to those targets to us. As a result of the discontinuation, the licenses granted by us to Neurocrine thereunder regarding the 2019 Discovery Programs terminated and we regained worldwide intellectual property rights regarding such programs. The 2019 Neurocrine Collaboration Agreement remains in full force and effect for the FA Program.

The 2019 Neurocrine Collaboration Agreement provides for aggregate development milestone payments from Neurocrine to us for the 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, for a total of up to $550.0 million under the FA Program. Neurocrine has also agreed to pay us royalties, based on future net sales of the 2019 Collaboration Products. Such royalty percentages, for net sales in and outside the United States, as applicable, range from the low-teens to high-teens and high-single digits to mid-teens,

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for the FA Program. For a further description of the 2019 Neurocrine Collaboration 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, 2025 under the caption “2019 Neurocrine Collaboration Agreement.”

Alexion Option and License Agreement

On October 1, 2021, we entered into an option and license agreement, or the Alexion Agreement, with Pfizer Inc., or Pfizer, pursuant to which we granted Pfizer options to receive an exclusive license to certain TRACER Capsids to develop and commercialize certain AAV gene therapy candidates comprised of a capsid and specified transgene. Effective as of September 30, 2022, Pfizer exercised one option with respect to a capsid for a specified transgene for the potential treatment of a rare neurological disease, and we granted Pfizer an exclusive, worldwide license, with the right to sublicense, under certain of our intellectual property, the rights to develop and commercialize rare neurological disease products utilizing the capsid candidate and incorporating the specified transgene.

Effective upon the closing of a transaction on September 20, 2023, Alexion (via Alexion Pharma International Operations Limited, or APIO) acquired all of Pfizer’s rights under the Alexion Agreement and became the successor-in-interest to Pfizer thereunder. APIO subsequently assigned the Alexion Agreement to its affiliate AstraZeneca Ireland Limited, or AstraZeneca. Neither the acquisition by Alexion nor the subsequent assignment from APIO to AstraZeneca impacted the material terms of the Alexion Agreement.

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. On October 1, 2025, Alexion exercised its right to substitute another TRACER Capsid for the TRACER Capsid that was originally licensed under the Alexion Agreement. 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, 2025, under the caption “Alexion Option and License Agreement (Formerly Pfizer Option and License Agreement).”

License Agreement with Transition Bio

In June 2025, we entered into a research collaboration, option and license agreement with Transition Bio, or the Transition Bio Agreement. The Transition Bio Agreement superseded a prior agreement between us and Transition Bio dated December 24, 2024. Under the terms of the agreement, Transition Bio is responsible for the discovery and optimization of small molecules targeting TDP-43 until nomination of a development candidate, upon which we will have an option to license the worldwide exclusive rights to develop and commercialize the program. The Transition Bio Agreement contains a termination for convenience clause that allows for either party to terminate the arrangement for any reason or no reason upon sixty days prior written notice. In the event that we terminate the Transition Bio Agreement due to a change in control, we are obligated to pay Transition Bio up to $2.0 million in termination fees.

We paid Transition Bio a low single-digit million dollar upfront payment during the year ended December 31, 2024 in connection with a prior agreement. During the fourth quarter of 2025, we paid Transition Bio an additional low single-digit million dollar payment for the achievement of a research milestone. Transition Bio is eligible to receive potential research, development, commercial and net sales milestone payments totaling up to $500.0 million. Transition Bio is also eligible for high single-digit to low double-digit royalties on net sales during the royalty term of up to ten years. For a further description of the Transition Bio 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, 2025, under the caption “Other Agreements.”

License Agreement with Touchlight IP Limited

In November 2022, we and Touchlight IP Limited, or Touchlight, entered into a license agreement, or the Touchlight License Agreement, to authorize historical use by us of a certain DNA preparation process, or the Subject

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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 three months ended March 31, 2026, as compared to those identified in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025. 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 9, 2026.

Results of Operations

Comparison of the three months ended March 31, 2026 and 2025

The following table summarizes our results of operations for the three months ended March 31, 2026, and 2025:


	Three Months Ended
	March 31,
	2026		2025		Change

	(in thousands)
Collaboration revenue	$	2,593	$	6,473	$	(3,880)
Operating expenses:
Research and development		24,602		31,526		(6,924)
General and administrative		8,261		9,640		(1,379)
Total operating expenses		32,863		41,166		(8,303)
Other income, net:
Interest income		1,912		3,291		(1,379)
Other income		435		418		17
Total other income, net		2,347		3,709		(1,362)
Loss before income taxes		(27,923)		(30,984)		3,061
Income tax provision		14		37		(23)
Net loss	$	(27,937)	$	(31,021)	$	3,084

Collaboration Revenue

Collaboration revenue was $2.6 million and $6.5 million for the three months ended March 31, 2026 and 2025, respectively. During the three months ended March 31, 2026, we recognized $1.3 million of collaboration revenue under the 2023 Neurocrine Collaboration Agreement, $0.1 million of collaboration revenue under the 2019 Neurocrine Collaboration Agreement, and $1.2 million of collaboration revenue under the 2023 Novartis Collaboration Agreement. During the three months ended March 31, 2025, we recognized $4.5 million of collaboration revenue under the 2023 Neurocrine Collaboration Agreement, $0.5 million of collaboration revenue under the 2019 Neurocrine Collaboration Agreement, $1.2 million of collaboration revenue under the 2023 Novartis Collaboration Agreement, and $0.3 million in other collaboration revenue.

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Research and Development Expense

The following table summarizes our research and development expenses for the three months ended March 31, 2026 and 2025:


	Three Months Ended
	March 31,
	2026		2025		Change

	(in thousands)
Internal research and development	$	8,093	$	9,958	$	(1,865)
External research and development:
Anti-tau antibody program (VY7523)		2,377		4,249		(1,872)
Tau silencing gene therapy program (VY1706)		5,636		3,174		2,462
SOD1 silencing gene therapy program		—		1,886		(1,886)
Partnered programs		672		991		(319)
Other programs and platforms		1,699		3,181		(1,482)
Facilities and other		6,125		8,087		(1,962)
Total research and development expenses	$	24,602	$	31,526	$	(6,924)

Research and development expense was $24.6 million and $31.5 million for the three months ended March 31, 2026 and March 31, 2025, respectively. The $6.9 million decrease was primarily due to the discontinuation of the SOD1 program, reduced spend on the anti-tau antibody program (VY7523) as enrollment in planned clinical trials was completed, and lower employee-related expenses as we had lower headcount due to cost-cutting initiatives. The decrease was partially offset by an increase in the tau silencing gene therapy program (VY1706) in preparation for the planned clinical trial initiation.

General and Administrative Expense

General and administrative expense decreased by $1.4 million from $9.6 million for the three months ended March 31, 2025 to $8.3 million for the three months ended March 31, 2026. The decrease in general and administrative expense was primarily attributable to lower legal fees and reduced employee-related expenses as a result of lower headcount year-over-year due to cost-cutting and efficiency initiatives.

Other Income, Net

Other income, net decreased by $1.4 million from $3.7 million for the three months ended March 31, 2025 to $2.3 million for the three months ended March 31, 2026. 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 March 31, 2026, as compared to the three months ended March 31, 2025.

Liquidity and Capital Resources

Sources of Liquidity

We have funded our operations primarily through private placements of redeemable convertible preferred stock, public offerings and private placements of our common stock, strategic collaborations and option and license arrangements, including our 2019 Neurocrine Collaboration Agreement, 2023 Neurocrine Collaboration Agreement, 2022 Novartis Option and License Agreement, 2023 Novartis Collaboration Agreement, Alexion Agreement, and our prior collaboration agreements.

In November 2025, we entered into a sales agreement with TD Securities (USA) LLC, as the sales agent, pursuant to which we may from time to time, issue and sell common stock with an aggregate value of up to $100.0 million in an at-the-market offering, or the ATM Facility. We sold 630,570 shares of our common stock under the ATM Facility during the three months ended March 31, 2026. These shares were sold at a weighted average price per share of

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$4.92 for aggregate net proceeds of $3.0 million, after deducting commissions and offering costs. As of March 31, 2026, $96.9 million remained available to be sold under the ATM Facility.

Cash Flows

The following table provides information regarding our cash flows for the three months ended March 31, 2026, and 2025:


	Three Months Ended March 31,
	2026		2025

	* (in thousands)*
Net cash (used in) provided by:
Operating activities	$	(33,419)	$	(37,895)
Investing activities		8,101		41,201
Financing activities		3,332		82
Net increase in cash, cash equivalents, and restricted cash	$	(21,986)	$	3,388

Net Cash Used in Operating Activities

Net cash used in operating activities was $33.4 million during the three months ended March 31, 2026, compared to $37.9 million during the three months ended March 31, 2025. The decrease in net cash used in operating activities is primarily attributed to a lower net loss. Net cash used in operating activities during the three months ended March 31, 2026, was primarily due to our net loss of $27.9 million, combined with changes in our working capital accounts, including decreases in accrued expenses and accounts payable and deferred revenue during the three months ended March 31, 2026.

Net cash used in operating activities during the three months ended March 31, 2025, was primarily due to our net loss of $31.0 million, combined with changes in our working capital accounts, including a decrease in accrued expenses and deferred revenue during the three months ended March 31, 2025.

Net Cash Provided by Investing Activities

Net cash provided by investing activities was $8.1 million during the three months ended March 31, 2026, compared to $41.2 million during the three months ended March 31, 2025. The decrease was primarily due to a decrease in proceeds from sales and maturities of marketable securities of $67.2 million, partially offset by decreases in purchases of marketable securities of $33.5 million during the three months ended March 31, 2026.

Net Cash Provided by Financing Activities

Net cash provided by financing activities was $3.3 million during the three months ended March 31, 2026, compared to $0.1 million during the three months ended March 31, 2025. The increase was primarily due to $3.0 million in proceeds during the three months ended March 31, 2026, from the issuance of common stock from the ATM Facility.

Funding Requirements

Our expenses decreased during the three months ended March 31, 2026, as compared with the three months ended March 31, 2025, as a result of continued portfolio prioritization and alignment of operating expenditures with our current capital resources. We expect we will continue to incur research and development expenses as we continue our research and development activities, conduct clinical trials, including completing the Phase 1 MAD clinical trial for VY7523 and initiating the clinical trial for VY1706, pending successful IND clearance, and seek marketing approval of our product candidates, 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 continue to

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●

the costs of establishing or contracting for sales, manufacturing, marketing, distribution, and other commercialization capabilities if we obtain regulatory approvals to market our product candidates.

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 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.

Until such time, if ever, as we can generate product revenues sufficient to achieve consistent profitability, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and option and license arrangements. We do not have any committed external source of funds other than the amounts we are entitled to receive from our collaboration partners and licensees for reimbursement of certain research and development expenses, potential option exercises, the achievement of specified regulatory and commercial milestones, and royalty payments under our collaboration, and option and license agreements, as applicable. To the extent that we raise additional capital through the sale of equity or equity-linked securities, including convertible debt, our stockholders’ ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect our existing stockholders’ rights as holders of our common stock. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, obtaining additional capital, acquiring or divesting businesses, making capital expenditures or declaring dividends.

If we raise additional funds through collaborations, strategic alliances, or option and license arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.

Contractual Obligations

We enter into agreements in the normal course of business with clinical research organizations, contract manufacturing organizations, and institutions to license intellectual property. These contracts generally are cancelable at any time by us upon 30 to 90 days prior written notice.

Our agreements to license intellectual property generally include potential milestone payments that are dependent upon the development of products using the intellectual property licensed under the agreements and contingent upon the achievement of clinical trial or regulatory approval milestones. We may also be required to pay annual maintenance fees or minimum amounts payable ranging from low-four digits to low five-digits depending upon the terms of the applicable agreement. In certain instances, we are also obligated to pay our licensors royalties based on sales of products, if approved, using the intellectual property licensed under the applicable agreement.

We also have non-cancelable operating lease commitments arising from our leases of office and laboratory space at our facilities in Cambridge and Lexington, Massachusetts. For more information, refer to Note 6 to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Off-Balance Sheet Arrangements

We did not have, during the periods presented, and we do not currently have, any off-balance sheet arrangements, as defined under applicable SEC rules.

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ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK

We are exposed to market risk related to changes in interest rates. We have policies requiring us to invest in high-quality issuers, limit our exposure to any individual issuer, and ensure adequate liquidity. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates, particularly because our investments, including cash equivalents, are in the form of money market funds and marketable securities and are invested in U.S. Treasury notes or government obligations and corporate securities. Due to the short-term duration of our investment portfolio and the low risk profile of our investments, we believe an immediate 100 basis point change in interest rates would not have a material effect on the fair market value of our portfolio.

We are not currently exposed to market risk related to changes in foreign currency exchange rates; however, we may contract with vendors that are located in Asia and Europe in the future and may be subject to fluctuations in foreign currency rates at that time.

Inflation generally affects us by increasing our costs of labor, goods, and services. We do not believe that inflation had a material effect on our business, financial condition, or results of operations during the three months ended March 31, 2026.

ITEM 4. CONTROLS AND PROCEDURES

Management’s Evaluation of Disclosure Controls and Procedures

We maintain “disclosure controls and procedures,” as defined in Rules 13a-15(e) or 15d-15(e) under the Exchange Act of 1934, or the Exchange Act, to mean controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized, and reported within the time periods specified in the SEC’s rules and forms. Our disclosure controls and procedures include, without limitation, controls and other procedures designed to ensure that information required to be disclosed by us in the reports we file or submit under the Exchange Act is accumulated and communicated to our management, including our principal executive officer and principal financial officer, as appropriate, to allow timely decisions regarding required disclosure.

Our management, with the participation of our principal executive officer and principal financial officer, evaluated the effectiveness of our disclosure controls and procedures as of March 31, 2026. Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Our principal executive officer and principal financial officer have concluded based upon the evaluation described above that, as of March 31, 2026, our disclosure controls and procedures were effective at the reasonable assurance level.

We continue to review and document our disclosure controls and procedures and may from time to time make changes aimed at enhancing their effectiveness and to ensure that our systems evolve with our business.

Changes in Internal Control over Financial Reporting

During the first quarter of 2026, we completed the implementation of a new enterprise resource planning, or ERP, system to replace our previous ERP system. As a result of this implementation, we modified certain existing internal controls over financial reporting and implemented application controls related to the new ERP system. The changes in process and procedures under the new ERP system continue to be subject to our evaluation of the operating effectiveness of internal control over financial reporting. During the three months ended March 31, 2026, there have been no other changes in our internal control over financial reporting, as such term is defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act, that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

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ITEM 6.EXHIBITS

The exhibits filed or furnished as part of this Quarterly Report are set forth on the Exhibit Index, which is incorporated herein by reference.

INDEX TO EXHIBITS

Incorporated by Reference to:

Exhibit
No.

Description

Form or
Schedule

Exhibit
No.

Filing
Date with
SEC

SEC File
Number

Filed
Herewith

10.1#

Employment Agreement, by and between the Registrant and Gregory Shiferman, dated as of December 4, 2025.

10.2#

Form of Stock Option Agreement under 2025 Stock Incentive Plan

10.3#

Form of Non-Employee Director Non-Statutory Stock Option Agreement under 2025 Stock Incentive Plan

10.4#

Form of Restricted Stock Unit Agreement under 2025 Stock Incentive Plan

31.1

Certification of Principal Executive Officer pursuant to Exchange Act Rules 13a-14 or 15d-14.

31.2

Certification of Principal Financial Officer pursuant to Exchange Act Rules 13a-14 or 15d-14.

32.1+

Certification of Principal Executive Officer and Principal Financial Officer pursuant to Exchange Act Rules 13a-14(b) or 15d-14(b) and 18 U.S.C. Section 1350.

101.INS

Inline XBRL Instance Document – the instance document does not appear in the Interactive Data File because XBRL tags are embedded within the Inline XBRL document.

101.SCH

Inline XBRL Taxonomy Extension Schema Document.

101.CAL

Inline XBRL Taxonomy Extension Calculation Linkbase Document.

101.DEF

Inline XBRL Taxonomy Extension Definition Linkbase Document.

101.LAB

Inline XBRL Taxonomy Extension Label Linkbase Document.

101.PRE

Inline XBRL Taxonomy Extension Presentation Linkbase Document.

104

Cover Page Interactive Data File – The cover page interactive data file does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document


Name (Title)	Action Taken (Date of Action)	Type of Trading Arrangement	Nature of Trading Arrangement	Duration of Trading Arrangement	Aggregate Number of Securities
Gregory Shiferman (Senior Vice President, General Counsel & Secretary)	Adoption (January 5, 2026)	Durable Rule 10b5-1 trading arrangement for sell-to-cover transactions related to restricted stock units, or RSUs	Sale	Until final settlement of any covered RSU	Indeterminable ⁽¹⁾


Date: May 7, 2026	VOYAGER THERAPEUTICS, INC.

	By:	/s/ Alfred Sandrock, M.D., Ph.D.
		Alfred Sandrock, M.D., Ph.D.
		Chief Executive Officer, President, and Director
		(Principal Executive Officer)

	By:	/s/ Nathan Jorgensen, Ph.D.
		Nathan Jorgensen, Ph.D.
		Chief Financial Officer
		(Principal Financial and Accounting Officer)


		Page
PART I. FINANCIAL INFORMATION
ITEM 1.	CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED)
	CONDENSED CONSOLIDATED BALANCE SHEETS	6
	CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS	7
	CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY	8
	CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS	9
	NOTES TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS	10
ITEM 2.	MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS	20
ITEM 3.	QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK	34
ITEM 4.	CONTROLS AND PROCEDURES	34
PART II. OTHER INFORMATION
ITEM 1.	LEGAL PROCEEDINGS	35
ITEM 1A.	RISK FACTORS	35
ITEM 5.	OTHER INFORMATION	35
ITEM 6.	EXHIBITS	37
	SIGNATURES	39