VYGR on Nasdaq

Voyager Therapeutics (United States)

Logo: voyagertherapeutics.com3

In its 2026 annual report the company says: “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. We are advancing our own proprietary pipeline of drug candidates for neurological diseases, with a focus on AD.”6 It was founded in 2013.1 It is headquartered in Cambridge.1 Its shares trade on Nasdaq under the symbol VYGR.1

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Last updated Oct 21, 2026.

Overview

2013Founded1

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  • TickerVYGR on Nasdaq1
  • IndustryBiological Products, (No Diagnostic Substances)1
  • HeadquartersCambridge1
  • Websitewww.voyagertherapeutics.com
  • Incorporated inDE4
  • Business address75 Hayden Avenue, Lexington, Ma 024214
  • Filer categoryAccelerated filer, Smaller reporting company4
  • Legal formCorporation

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 …6 From its latest annual report

Financials

From the company’s reports to the SEC. Every number links to the filing it came from.

Key numbers, fiscal 2025

-326.6%Operating margin7
-296.5%Net margin7
-49.5%Revenue growth, 20257
-61.1%Return on equity7
-$135.1MFree cash flow7

Where the money goes

Where each dollar of revenue went, fiscal 2025

  • Research and development100¢$134.7M
  • Income tax0¢$144.0K

Out of $40.4M in revenue.7 The company lost money this year.

Financial statements

Revenue8910117$40.4M in 2025−61% since 2019
0$100.0M$200.0M$300.0M2019: $104.4M$104.4M20192020: $171.1M$171.1M2020+64%2021: $37.4M$37.4M2021−78%2022: $40.9M$40.9M2022+9%2023: $250.0M$250.0M2023+511%2024: $80.0M$80.0M2024−68%2025: $40.4M$40.4M2025−50%
Operating income8910117-$131.8M in 2025+155% since 2019
-$200.0M-$100.0M0$100.0M$200.0M2019: -$51.7M-$51.7M20192020: $27.4M$27.4M20202021: -$73.6M-$73.6M20212022: -$50.8M-$50.8M2022−31%2023: $122.0M$122.0M20232024: -$83.3M-$83.3M20242025: -$131.8M-$131.8M2025+58%
Net income8910117-$119.7M in 2025+175% since 2019
-$200.0M-$100.0M0$100.0M$200.0M2019: -$43.6M-$43.6M20192020: $36.7M$36.7M20202021: -$71.2M-$71.2M20212022: -$46.4M-$46.4M2022−35%2023: $132.3M$132.3M20232024: -$65.0M-$65.0M20242025: -$119.7M-$119.7M2025+84%

Every line of the income statement as filed: the last 3 fiscal years, latest first.7

Period ending2025-12-312024-12-312023-12-31
Collaboration revenue$40.4M$80.0M$250.0M
Research and development$134.7M$127.4M$92.2M
General and administrative$37.5M$35.9M$35.8M
Total operating expenses$172.2M$163.3M$128.0M
Operating (loss) income-$131.8M-$83.3M$122.0M
Interest income$10.6M$18.3M$11.7M
Other income$1.6M$622.0K$3.0K
Total other income, net$12.3M$18.9M$11.7M
(Loss) income before income taxes-$119.6M-$64.3M$133.7M
Income tax provision$144.0K$665.0K$1.4M
Net (loss) income-$119.7M-$65.0M$132.3M
Net unrealized gain (loss) on available-for-sale-securities$439.0K-$359.0K$171.0K
Total other comprehensive income (loss)$439.0K-$359.0K$171.0K
Comprehensive income (loss)-$119.3M-$65.4M$132.5M
Net (loss) income per share, basic (in dollars per share)-$2.04-$1.13$3.08
Net (loss) income per share, diluted (in dollars per share)-$2.04-$1.13$2.97
Weighted-average common shares outstanding, basic (in shares)58.7M shares57.7M shares43.0M shares
Weighted-average common shares outstanding, diluted (in shares)58.7M shares57.7M shares44.6M shares

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Corporate structure

Voyager Therapeutics (United States) sits at the top of its group. 2 entities, 1 country on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Voyager Therapeutics (United States)

  • ARMISTICE CAPITAL, LLC Filed SCHEDULE 13G/A on Voyager Therapeutics (United States), latest Aug 14, 2026 (11 filings since Feb 15, 2022)
  • INTEGRATED CORE STRATEGIES (US) LLC Filed SCHEDULE 13G/A on Voyager Therapeutics (United States), latest Jul 31, 2026 (2 filings since Jan 26, 2026)
  • EcoR1 Capital, LLC Filed SCHEDULE 13G/A on Voyager Therapeutics (United States), latest May 15, 2026 (8 filings since Oct 22, 2021)
  • VANGUARD GROUP INC Filed SCHEDULE 13G/A on Voyager Therapeutics (United States), latest Mar 27, 2026 (4 filings since Feb 10, 2022)
  • BlackRock Filed SCHEDULE 13G/A on Voyager Therapeutics (United States), latest Apr 24, 2025 (3 filings since Jan 29, 2024)
  • Third Rock Ventures III, L.P. Filed SC 13G/A on Voyager Therapeutics (United States), latest Feb 14, 2024 (2 filings since Feb 14, 2023)
  • Neurocrine Biosciences Filed SC 13D/A on Voyager Therapeutics (United States), latest Feb 24, 2023
  • BB Biotech Filed SC 13G/A on Voyager Therapeutics (United States), latest Feb 11, 2022
  • Voyager Therapeutics (United States) Cambridge
    • Voyager Securities Corporation Subsidiary · Massachusetts, United States

Built from subsidiaries listed in Exhibit 21 of annual reports filed with the SEC; major holders from Schedules 13D and 13G. Each entity in the chart links to its source.

What it owns

What Voyager Therapeutics (United States) reports owning in its annual reports to the SEC: buildings, land and equipment, real estate and locations, investments, intangible assets and inventory, at the end of its latest fiscal year and a year before.

Property and equipment

ItemLatestA year beforeChange
Property and equipment, before depreciation$32.3M7 Dec 31, 2025$30.3M7 Dec 31, 2024+6.7%
Accumulated depreciation$19.2M7 Dec 31, 2025$16.0M7 Dec 31, 2024+20.0%
Property and equipment, net$13.1M7 Dec 31, 2025$14.3M7 Dec 31, 2024−8.2%
Operating-lease assets (leased space)$28.5M7 Dec 31, 2025$33.3M7 Dec 31, 2024−14.6%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$65.3M7 Dec 31, 2025$71.4M7 Dec 31, 2024−8.5%
Marketable securities, current$131.1M7 Dec 31, 2025$195.3M7 Dec 31, 2024−32.9%
Marketable securities, non-current$5.2M7 Dec 31, 2025$65.7M7 Dec 31, 2024−92.0%

Properties, in its own words

Our corporate headquarters are located in Lexington, Massachusetts. We lease our office and laboratory space, which consist of approximately 26,148 square feet located in Cambridge, Massachusetts and 93,449 square feet located in Lexington, Massachusetts. Our lease in Cambridge expires in 2026 and our lease in Lexington expires in 2031. We have subleased our office and laboratory space in Cambridge to a third party.

Item 2 of the 10-K filed Mar 9, 2026.6

Leadership

Who has led Voyager Therapeutics (United States), and when. Pick a name to see everywhere that person has worked.

Board members and other officers (26)

Named in SEC Form D filings

People the company listed in its private-offering notices.

  • Executive OfficerJacquelyn Fahey Sandell named Jan 17, 202449
  • DirectorMichael Higgins named Mar 3, 2023 to Jan 17, 202449

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • Voyager Securities Corporation Massachusetts5

The subsidiaries the company lists in Exhibit 21 of its annual report, as filed.

Timeline

  1. Aug 6, 20268-K Results of operations50
  2. Jul 13, 20268-K Other events51
  3. Jun 10, 20268-K Bylaws or fiscal year changed; Shareholder vote52
  4. Jun 1, 20268-K Other events53
  5. May 7, 20268-K Results of operations54
  6. Apr 21, 20268-K Executive or director change55
  7. Mar 9, 20268-K Results of operations56
  8. Feb 12, 20268-K Executive or director change; Other events57
  9. Jan 8, 20268-K Regulation FD disclosure58
  10. Nov 10, 20258-K Results of operations59
  11. Sep 2, 20258-K Regulation FD disclosure60
  12. Aug 6, 20258-K Results of operations61
  13. Mar 23, 2022New chief executive Alfred Sandrock becomes CEO15
  14. Jul 18, 2018New chief executive Andre Turenne becomes CEO16
  15. 2013Founded Founded1

Calendar

Coming up

  1. Nov 9, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.47) a share, on Nov 10, 2025.62

When it reported results

  1. Aug 6, 2026Reported quarterly or annual results50
  2. May 7, 2026Reported quarterly or annual results54
  3. Mar 9, 2026Reported quarterly or annual results56
  4. Nov 10, 2025Reported quarterly or annual results59
  5. Aug 6, 2025Reported quarterly or annual results61
  6. May 6, 2025Reported quarterly or annual results63
  7. Mar 11, 2025Reported quarterly or annual results64
  8. Nov 12, 2024Reported quarterly or annual results65

Upcoming dates from Nasdaq's calendar; past results from the company's 8-K filings (Item 2.02). The full markets calendar

Filings

357 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 57 major events, 170 insider forms, 32 large holders, 2 private raises, 76 other. The latest 20:

  1. Aug 14, 2026SCHEDULE 13G/A EDGAR
  2. Aug 6, 20268-K EDGAR
  3. Aug 6, 202610-Q EDGAR
  4. Jul 31, 2026SCHEDULE 13G/A EDGAR
  5. Jul 13, 20268-K EDGAR
  6. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  7. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  8. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  9. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  10. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  11. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  12. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  13. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  14. Jun 11, 20264 OWNERSHIP DOCUMENT EDGAR
  15. Jun 10, 20268-K EDGAR
  16. Jun 1, 20268-K EDGAR
  17. May 18, 20263 OWNERSHIP DOCUMENT EDGAR
  18. May 15, 2026144 EDGAR
  19. May 15, 2026144 EDGAR
  20. May 15, 2026SCHEDULE 13G/A EDGAR

The 357 filings since October 2021 can be filtered by form type and year here when JavaScript is on.

Every filing from the last five years, summarized. The full record is on SEC EDGAR.

Insider trades

85 open-market trades by officers, directors and large owners since October 2021: 3 buys and 82 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Apr 2, 2026Sandrock AlfredPresident and CEOSale11,511$3.87$44.5KForm 4
Feb 24, 2026Swartz RobinCOO & CBOSale6,458$3.77$24.3KForm 4
Feb 24, 2026Sandrock AlfredPresident and CEOSale14,197$3.79$53.8KForm 4
Feb 24, 2026Jorgensen Nathan D.Chief Financial OfficerSale4,668$3.77$17.6KForm 4
Feb 24, 2026Carter Todd AlfredChief Scientific OfficerSale4,174$3.76$15.7KForm 4
Feb 18, 2026Swartz RobinCOO & CBOSale3,882$3.44$13.4KForm 4
Feb 18, 2026Carter Todd AlfredChief Scientific OfficerSale3,301$3.44$11.4KForm 4
Feb 18, 2026Sandrock AlfredPresident and CEOSale11,732$3.41$40.0KForm 4
Feb 10, 2026Swartz RobinCOO & CBOSale4,569$3.85$17.6KForm 4
Feb 10, 2026Sandrock AlfredPresident and CEOSale12,192$3.78$46.1KForm 4
Feb 10, 2026Carter Todd AlfredChief Scientific OfficerSale3,525$3.85$13.6KForm 4
Oct 3, 2025Jorgensen Nathan D.Chief Financial OfficerSale7,666$4.78$36.6KForm 4
Aug 18, 2025Ferguson TobyChief Medical OfficerSale19,000$3.73$70.9KForm 4
Apr 2, 2025Ferguson TobyChief Medical OfficerSale10,086$3.43$34.6KForm 4
Apr 2, 2025Sandrock AlfredPresident and CEOSale10,885$3.43$37.3KForm 4
Feb 20, 2025Carter Todd AlfredChief Scientific OfficerSale3,305$4.26$14.1KForm 4
Feb 20, 2025Sandrock AlfredPresident and CEOSale11,702$4.24$49.6KForm 4
Feb 20, 2025Swartz RobinCOO & CBOSale3,894$4.25$16.6KForm 4
Feb 11, 2025Carter Todd AlfredChief Scientific OfficerSale5,413$4.45$24.1KForm 4
Feb 11, 2025Swartz RobinCOO & CBOSale6,950$4.45$30.9KForm 4

All 85 trades can be filtered by person here when JavaScript is on.

From Forms 4 filed with the SEC.

Major holders

Investors who own more than 5% of the shares must tell the SEC: a 13D if they may seek to influence the company, a 13G if they hold passively.

Executive pay

What the company paid its top executives, from the summary compensation table in its proxy statement. Total pay includes salary, bonus, stock and option awards and other pay, as the company valued them.

YearExecutiveAgeSalaryTotal pay
2025Alfred Sandrock, M.D., Ph.D.Chief Executive Officer, President, and Director68$674.9K$2.5M74
2025Jacquelyn Fahey SandellFormer Chief Legal Officer$1.5M74
2025Robin SwartzChief Business Officer & Chief Operating Officer55$520.2K$1.5M74
2025Toby Ferguson, M.D., Ph.D.Former Chief Medical Officer$513.4K$1.4M74
2024Alfred Sandrock, M.D., Ph.D.Chief Executive Officer, President, and Director68$652.1K$3.4M74
2024Toby Ferguson, M.D., Ph.D.Former Chief Medical Officer$3.0M74
2024Robin SwartzChief Business Officer & Chief Operating Officer55$490.7K$1.6M74
2024Jacquelyn Fahey SandellFormer Chief Legal Officer$468.8K$1.2M74

Funding

Where the company's money came from, by calendar year: private offerings filed with the SEC, federal grants, loans, contracts and other assistance from USAspending.gov, and PPP loans from the SBA. Each bar links to the records below.

Money received by year$59.0M 2023–2024
Money received by yearMoney received by year, 2023–2024: $59.0M in all. Private raises (Form D) $59.0M.$0$10M$20M$30M$40M2023 · Private raises (Form D): $39.0M20232024 · Private raises (Form D): $20.0M2024
Figures by year
YearPrivate raises (Form D)Total
2024$20.0M$20.0M
2023$39.0M$39.0M

Private raises

Private offerings the company reported to the SEC on Form D. The amount is what the latest filing says was sold so far, charted in the year of the first sale. Covers filings since Oct 1, 2021.

  1. Jan 3, 2024Raised $20 million in a private offering of equity49 Form D: 1 investor so far Form D
  2. Feb 23, 2023Raised $39 million in a private offering of equity75 Form D: 1 investor so far Form D

Held by these funds

The largest holders among investment managers' 13F reports for the quarter ending Jun 30, 2026, the top 25 of 100. Change is in shares since the quarter before.

FundSharesValueShare of the fundChange
BlackRock, Inc.5,172,063$18.1M0.0%−396,318
Millennium Management LLC4,442,951$15.6M0.0%+1,014,380
Armistice Capital, LLC3,436,000$12.0M0.3%−64,000
Vestal Point Capital, LP2,625,000$9.2M0.2%−377,936
Vanguard Capital Management LLC2,345,674$8.2M0.0%+62,421
Geode Capital Management, LLC1,330,492$4.7M0.0%+280,514
Opaleye Management Inc.1,185,000$4.1M0.4%+35,000
State Street Corp1,076,422$3.8M0.0%+128,196
Two Sigma Investments, LP948,517$3.3M0.0%+332,857
Dimensional Fund Advisors LP838,184$2.9M0.0%−37,384
Vanguard Portfolio Management LLC810,091$2.8M0.0%+84,322
Citadel Advisors LLC784,067$2.7M0.0%+419,403
Morgan Stanley697,334$2.4M0.0%+15,623
Erste Asset Management GmbH638,900$2.3M0.0%−456,241
Renaissance Technologies LLC495,236$1.7M0.0%−82,788
Sphera Funds Management Ltd.427,554$1.5M0.2%−42,207
Diadema Partners LP413,642$1.4M0.5%−10,000
Northern Trust Corp411,803$1.4M0.0%+26,960
Aqr Capital Management LLC356,109$1.2M0.0%+123,428
Goldman Sachs Group Inc338,477$1.2M0.0%−89,120
Vanguard Fiduciary Trust Co337,325$1.2M0.0%+39,003
Fiduciary Trust Co334,397$1.2M0.0%No change
Jpmorgan Chase & Co276,211$988.8K0.0%−656
Point72 Asset Management, L.P.236,083$826.3K0.0%−32,140
Panagora Asset Management Inc231,077$808.8K0.0%New

13F reports cover disclosed long positions in U.S. stocks only.

SEC filer profile

How Voyager Therapeutics (United States) is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARVoyager Therapeutics, Inc.2
  • CIK0001640266
  • Employer ID (EIN)46-30031826
  • Filer statusAccelerated filer not an emerging growth company; not a smaller reporting company; not a shell company6
  • Incorporated inDelaware6
  • Fiscal year endsDecember 312
  • Industry code (SIC)2836 Biological Products, (No Diagnostic Substances)2
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance2
  • Business address75 Hayden Avenue, Lexington, MA 024212
  • Phone857-259-53402
  • Shares outstanding61,617,948 as of Jul 30, 202613
  • Public float$144.2M as of Jun 30, 20256
  • AuditorErnst & Young LLP, Boston, Massachusetts PCAOB ID 42; it also attests to the company’s internal control over financial reporting6
  • On EDGAR sinceApr 23, 20152
  • Filings678 in all: 4 (272), 8-K (112), 144 (42), SC 13G/A (40), 3 (38), 10-Q (33), SC 13G (18), S-8 (13)2
    All 36 form types

    SCHEDULE 13G/A (13), 10-K (11), DEF 14A (11), CORRESP (10), DEFA14A (7), UPLOAD (6), EFFECT (5), 424B5 (4), ARS (4), CT ORDER (4), DRSLTR (4), S-1/A (4), S-3 (4), 424B3 (3), D (3), DRS/A (3), SC 13D (2), SEC STAFF LETTER (2), 424B4 (1), 8-A12B (1), CERTNAS (1), DRS (1), POS EX (1), PRE 14A (1), S-1 (1), S-3/A (1), SC 13D/A (1), SCHEDULE 13G (1)

  • Latest filingsAnnual report (10-K) Mar 9, 2026
    Quarterly report (10-Q) Aug 6, 2026
    Proxy statement (DEF 14A) Apr 28, 2026
    Latest 8-K (8-K) Aug 6, 2026

Securities registered for trading

ClassSymbolExchange
Common Stock, $0.001 par valueVYGRNasdaq Global Select Market

From the cover page of the 10-K filed Mar 9, 2026 (Section 12(b) of the Exchange Act).6

Risk factors and legal proceedings

The risks Voyager Therapeutics (United States) names in Item 1A of its 10-K filed Mar 9, 2026: 70 risks under these headings, as written.6

  • Risks Related to Our Financial Position and Need for Capital
  • We have a history of incurring significant losses and anticipate that we will continue to incur losses for the foreseeable future and may never achieve or maintain consistent profitability.
  • We may not be able to generate sufficient revenue from the commercialization of our product candidates and may never be consistently profitable.
  • We will need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate certain of our product development efforts or other operations.
  • Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
  • The early stage of our development efforts may make it difficult for our stockholders to evaluate the success of our business to date and to assess our future viability.
  • Risks Related to the Development and Regulatory Approval of Our Product Candidates
  • Our AAV gene therapy, non-viral therapeutic, and other product candidates are based on proprietary technologies and, in several disease areas, unvalidated treatment approaches, which make it difficult and potentially infeasible to predict the duration and cost of development of, and subsequently obtaining regulatory approval for, our product candidates.
  • Regulatory requirements governing gene therapy, non-viral therapeutic, and other products have changed frequently and may continue to change in the future. Such requirements may lengthen the regulatory review process, require us to modify current studies or perform additional studies, or increase our development costs, which in turn may force us to delay, limit, or terminate certain of our programs.
  • Results from preclinical studies and early-stage clinical trials may not be indicative of efficacy in late-stage clinical trials.
  • We have conducted, and may in the future conduct, clinical trials for product candidates at sites outside the United States, and the FDA may not accept data from trials conducted in such locations.
  • Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their further development or regulatory approval, limit their commercial potential or result in significant negative consequences following any potential marketing approval.
The other 60 risks
  • A potential breakthrough therapy designation by the FDA for our product candidates may not lead to a faster development or regulatory review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.
  • A potential regenerative medicine advanced therapy designation by the FDA for our product candidates may not lead to a faster development or regulatory review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.
  • Fast Track designation by the FDA may not actually lead to a faster development or regulatory review or approval process and does not assure FDA approval of our product candidate.
  • Priority review designation by the FDA may not lead to a faster regulatory review or approval process and, in any event, does not assure FDA approval of our product candidate.
  • Even if we complete the necessary preclinical studies and clinical trials, the marketing approval process is expensive, time-consuming and uncertain and may prevent us from obtaining approvals for the commercialization of some or all of our product candidates. If we or any current or future collaborators are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we or they may not be able to commercialize our products, and our ability to generate revenue may be materially impaired.
  • Even if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory oversight.
  • Disruptions at the FDA and other government agencies from funding cuts, personnel losses, regulatory reform, government shutdowns and other developments could hinder our ability to obtain guidance from the FDA regarding our clinical development programs and develop and secure approval of our product candidates in a timely manner, which would negatively impact our business.
  • We face significant competition in an environment of rapid technological change and the possibility that our competitors may achieve regulatory approval before us or develop therapies that are more advanced or effective than ours, which may harm our business and financial condition and our ability to successfully market or commercialize our product candidates.
  • Even if we obtain and maintain approval for our product candidates from the FDA, we may never obtain approval for our product candidates outside of the United States, which would limit our market opportunities and adversely affect our business.
  • If approved, our product candidates that are licensed and regulated as biologics may face competition from biosimilars approved through an abbreviated regulatory pathway.
  • Risks Related to Third Parties
  • To date, substantially all of our revenue has been derived from our ongoing collaborations and licensing agreements with Neurocrine, Novartis, and Alexion, and from our prior collaborations with Sanofi Genzyme Corporation, AbbVie Biotechnology Ltd and AbbVie Ireland Unlimited Company. If any ongoing or future collaboration, option and license, or license agreements were to be terminated, our business financial condition, results of operations and prospects could be harmed.
  • We may seek to enter into collaborations and out-licensing transactions in the future with other third parties. If we are unable to enter into such collaborations or out-licensing transactions, or if these collaborations or out-licensing transactions are not successful, our business could be adversely affected.
  • We and our collaborators have relied, and we and our collaborators expect to continue to rely, on third parties to conduct, supervise and monitor our preclinical studies and clinical trials, and if these third parties perform in an unsatisfactory manner, our business could be harmed.
  • Risks Related to Manufacturing
  • Our gene therapies, non-viral therapeutics, and other biological modalities are novel, complex and difficult to manufacture. We could experience manufacturing problems that result in delays in the development or commercialization of our product candidates or otherwise harm our business.
  • Delays in obtaining regulatory approval of our or our collaborators’ manufacturing processes and facilities or disruptions in such manufacturing processes may delay or disrupt our commercialization efforts. Until recently, no cGMP gene therapy manufacturing facility in the United States had received approval from the FDA for the manufacture of an approved gene therapy product.
  • Failure to comply with ongoing regulatory requirements could cause us to suspend production or put in place costly or time-consuming remedial measures.
  • Any contamination in the manufacturing process for our products or product candidates, shortages of raw materials, cells or reagents, or failure of any of our key suppliers to deliver necessary components could result in delays in our clinical development or marketing schedules.
  • Failure to obtain access to or to protect intellectual property related to the manufacturing of our products or product candidates may result in changes, delays and/or inability to manufacture such products or product candidates.
  • Risks Related to Our Business Operations
  • We may not be successful in our efforts to identify or discover additional product candidates and may fail to capitalize on programs or product candidates that may be a greater commercial opportunity, or for which there is a greater likelihood of success.
  • Our future success depends on our ability to retain key members of our management and research and development teams, and to attract, retain and motivate qualified personnel.
  • In 2025 and 2026, we reduced the size of our organization in connection with restructurings. We may encounter difficulties in managing our business as a result of these restructurings, or as a result of the attrition that has occurred or may in the future occur due to these restructurings, which could disrupt our operations. In addition, we may not achieve anticipated benefits and savings from these restructurings.
  • If we are unable to manage expected growth in the scale and complexity of our operations, our performance may suffer.
  • Our employees, principal investigators, consultants and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
  • Current and future legislation may increase the difficulty and cost for us and any collaborators to obtain marketing approval of and commercialize our product candidates and affect the prices we, or they, may obtain.
  • The prices of prescription pharmaceuticals in the United States and foreign jurisdictions are subject to considerable legislative and executive actions and could impact the prices we obtain for our drug products, if and when approved.
  • Our relationships with healthcare providers, physicians and third-party payors will be subject, directly or indirectly, to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which, in the event of a violation, could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits and future earnings.
  • We are subject to stringent privacy laws, information security laws, regulations, policies and contractual obligations related to data privacy and security and changes in such laws, regulations, policies, and contractual obligations or our failure to comply with such requirements could subject us to significant fines and penalties, which may have a material adverse effect on our business, financial condition or results of operations.
  • Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any product candidates that we may develop.
  • If we, our collaborators, or any third-party manufacturers engaged by us or our collaborators fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could harm our business.
  • Risks Related to the Commercialization of Our Product Candidates
  • The affected populations for our product candidates may be smaller than we or third parties currently project, which may affect the addressable markets for our product candidates.
  • If we are unable to establish sales, medical affairs, market access and marketing capabilities or enter into agreements with third parties to market and sell our product candidates, we may be unable to generate any product revenue.
  • The insurance coverage and reimbursement status of newly-approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for our product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue.
  • The commercial success of any of our product candidates will depend upon its degree of market acceptance by physicians, patients, third-party payors and others in the medical community.
  • Our gene therapy approach utilizes vectors derived from viruses that are selectively engineered, which may be perceived as unsafe or may result in unforeseen adverse events. Adverse events related to other gene therapy companies and negative public opinion and increased regulatory scrutiny of gene therapy may damage public perception of the safety of our gene therapy product candidates and adversely affect our ability to conduct our business or obtain regulatory approvals for our gene therapy product candidates.
  • If we obtain approval to commercialize our product candidates outside of the United States, in particular in the United Kingdom or European Union, a variety of risks associated with international operations could harm our business.
  • Changes in and uncertainty surrounding U.S. and international trade policies may adversely impact our business and operating results.
  • Risks Related to Our Intellectual Property
  • Our rights to develop and commercialize our product candidates are subject to, in part, the terms and conditions of licenses granted to us by others.
  • If we are unable to obtain and maintain patent protection for our products and technology, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to successfully commercialize our products and technology may be adversely affected, which would have a material adverse impact on our business, results of operations, financial condition and prospects.
  • Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could impact the scope of our rights to the relevant intellectual property or technology, result in delays and increased costs associated with the resolution of the disagreements, result in termination of our access to such intellectual property, or increase our financial or other obligations to our licensors, licensees, or collaborators.
  • We may not be successful in obtaining necessary rights to our product candidates through acquisitions and in-licenses.
  • Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
  • We may not be able to protect our intellectual property rights throughout the world.
  • Issued patents covering our technology or product candidates could be found invalid or unenforceable if challenged in court. We may not be able to protect our trade secrets in court.
  • Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business.
  • Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
  • We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets or other proprietary confidential information or know-how of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.
  • Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
  • If we do not obtain patent term extension and regulatory exclusivity for our product candidates, our business may be harmed.
  • Intellectual property rights do not necessarily address all potential threats.
  • We may not be able to maintain sufficient control over our proprietary know-how or trade secrets when employees, consultants, advisors or persons with access to our proprietary information terminate their relationship with us.
  • Our reliance on third parties requires us to share our trade secrets, confidential information and know-how, which increases the possibility that a competitor will discover them or that our trade secrets, confidential information and/or know-how will be misappropriated or disclosed.
  • Risks Related to Ownership of Our Common Stock
  • Sales of a substantial number of shares of our common stock in the public market could cause our stock price to fall.
  • The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock.
  • We have been, and could in the future be, subject to legal actions and proceedings related to the decline in our stock price, which could distract our management and could result in substantial costs or large judgments against us.
  • We have broad discretion in how we apply our available funds, and we may not use these funds effectively, which could affect our results of operations and cause our stock price to decline.
  • Provisions in our amended and restated certificate of incorporation and bylaws and Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
  • Our amended and restated certificate of incorporation designates the Court of Chancery of the State of Delaware as the sole and exclusive forum for certain types of actions and proceedings that may be initiated by our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers or employees.
  • Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will be our stockholders’ sole source of gain.
  • General Risk Factors
  • We might not be able to utilize a significant portion of our net operating loss carryforwards.
  • Our internal computer systems, or those of our collaborators or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs.

Legal proceedings

In the ordinary course of business, we are from time to time involved in lawsuits, claims, investigations, proceedings, and threats of litigation relating to intellectual property, commercial arrangements and other matters. While the outcome of any such matters cannot be predicted with certainty, as of December 31, 2025, we were not party to any material pending proceedings. No material governmental proceedings are pending or, to our knowledge, contemplated against us. We are not a party to any material proceedings in which any director, member of senior management or affiliate of ours is either a party adverse to us or our subsidiaries or has a material interest adverse to us or our subsidiaries.

The opening of Item 3, Legal Proceedings.6

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