ADVM on NASDAQ

Avalanche Biotechnologies

American gene therapy company

Adverum Biotechnologies, Inc. (formerly known as Avalanche Biotechnologies) is a publicly traded clinical stage gene therapy company located in Redwood City, California. It is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD).3 Its shares trade on NASDAQ under the symbol ADVM.1 Wikipedia text CC BY-SA 4.0

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Last updated Oct 21, 2026.

Overview

Jan 1, 2006Founded1

Compare with Pluri Inc., PROKIDNEY CORP. or Compass Therapeutics, Inc., or pick any stock.

  • TickerADVM on NASDAQ1
  • Industrybiotechnology1
  • HeadquartersRedwood City1
  • Websiteadverum.com
  • Incorporated inDE4
  • Business address100 Cardinal Way, Redwood City, Ca 940634
  • Filer categoryNon-accelerated filer, Smaller reporting company4
  • Legal formCorporation

Adverum Biotechnologies, Inc. (formerly known as Avalanche Biotechnologies) is a publicly traded clinical stage gene therapy company located in Redwood City, California. It is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD).3

Financials

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

Key numbers, fiscal 2024

-13915.9%Operating margin7
-13092.7%Net margin7
-72.2%Revenue growth, 20247
-185.2%Return on equity7
-$92.8MFree cash flow7

Where the money goes

Where each dollar of revenue went, fiscal 2024

  • Research and development100¢$77.0M

Out of $1.0M in revenue.7 The company lost money this year.

Financial statements

Revenue8910117$1.0M in 2024−38% since 2018
0$2.0M$4.0M$6.0M$8.0M2018: $1.6M$1.6M20182019: $250.0K$250.0K2019−84%2020: $0$020202021: $7.5M$7.5M20212022: $0$020222023: $3.6M$3.6M20232024: $1.0M$1.0M2024−72%
Operating income8910117-$139.2M in 2024+78% since 2018
-$200.0M-$150.0M-$100.0M-$50.0M02018: -$78.1M-$78.1M20182019: -$68.5M-$68.5M2019−12%2020: -$118.0M-$118.0M2020+72%2021: -$146.1M-$146.1M2021+24%2022: -$157.1M-$157.1M2022+8%2023: -$128.9M-$128.9M2023−18%2024: -$139.2M-$139.2M2024+8%
Net income8910117-$130.9M in 2024+80% since 2018
-$200.0M-$150.0M-$100.0M-$50.0M02018: -$72.6M-$72.6M20182019: -$64.5M-$64.5M2019−11%2020: -$117.5M-$117.5M2020+82%2021: -$145.5M-$145.5M2021+24%2022: -$154.5M-$154.5M2022+6%2023: -$122.1M-$122.1M2023−21%2024: -$130.9M-$130.9M2024+7%
Fiscal year2018201920202021202220232024
Revenue$1.6M8$250.0K8$09$7.5M10$011$3.6M7$1.0M7
Cost of revenue$100.0K14
Research and development$50.1M8$40.4M8$73.3M9$89.2M10$99.3M11$77.5M7$77.0M7
Operating expenses$153.6M10$157.1M11$132.5M7$140.2M7
Operating income-$78.1M8-$68.5M8-$118.0M9-$146.1M10-$157.1M11-$128.9M7-$139.2M7
Interest
Income before tax-$145.5M10-$154.5M11-$123.2M7-$130.9M7
Income tax-$1.2M8$08$1.1M9$010$74.0K11-$1.1M7$07
Net income-$72.6M8-$64.5M8-$117.5M9-$145.5M10-$154.5M11-$122.1M7-$130.9M7
Earnings per share-$1.389-$1.4810-$1.5611-$12.117-$6.627
Earnings per share (diluted)-$1.389-$1.4810-$1.5611-$12.117-$6.627
Shares (weighted, basic)98.0M shares1099.3M shares1110.1M shares719.8M shares7
Shares (weighted, diluted)98.0M shares1099.3M shares1110.1M shares719.8M shares7

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Corporate structure

Avalanche Biotechnologies sits at the top of its group. 4 entities, 3 countries on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Avalanche Biotechnologies

  • BML Investment Partners, L.P. Filed SCHEDULE 13D/A on Avalanche Biotechnologies, latest Dec 9, 2025 (11 filings since May 10, 2022)
  • Frazier Life Sciences Public Fund, L.P. Filed SCHEDULE 13G/A on Avalanche Biotechnologies, latest Dec 9, 2025 (4 filings since Feb 9, 2024)
  • Franklin Templeton Investments Filed SCHEDULE 13G/A on Avalanche Biotechnologies, latest Aug 5, 2025 (2 filings since Oct 31, 2024)
  • BlackRock Filed SCHEDULE 13G/A on Avalanche Biotechnologies, latest Jul 16, 2025 (5 filings since Feb 3, 2022)
  • TCG Crossover GP II, LLC Filed SCHEDULE 13G/A on Avalanche Biotechnologies, latest May 15, 2025 (2 filings since Feb 15, 2024)
  • Venrock Healthcare Capital Partners III, L.P. Filed SCHEDULE 13G/A on Avalanche Biotechnologies, latest Feb 14, 2025 (3 filings since Jul 24, 2023)
  • Ryan Bradford Rush Filed SCHEDULE 13G on Avalanche Biotechnologies, latest Dec 17, 2024
  • Commodore Capital LP Filed SC 13G/A on Avalanche Biotechnologies, latest Nov 14, 2024 (3 filings since Apr 20, 2023)
  • Logos Global Management LP Filed SC 13G/A on Avalanche Biotechnologies, latest Nov 14, 2024 (2 filings since Feb 22, 2024)
  • Vivo Opportunity Fund Holdings, L.P. Filed SC 13G/A on Avalanche Biotechnologies, latest Nov 14, 2024 (2 filings since Feb 14, 2024)
  • FMR LLC Filed SC 13G/A on Avalanche Biotechnologies, latest Nov 12, 2024 (4 filings since Apr 11, 2022)
  • Morgan Stanley Filed SC 13G/A on Avalanche Biotechnologies, latest Nov 6, 2024 (3 filings since Feb 8, 2023)
  • Avalanche Biotechnologies Redwood City
    • Adverum NC, LLC Subsidiary · North Carolina, United States
    • Annapurna Therapeutics, LTD Subsidiary · Ireland
    • Avalanche Australia PTY LTD Subsidiary · Australia

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 Avalanche Biotechnologies 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$29.7M7 Dec 31, 2024$29.3M7 Dec 31, 2023+1.3%
Accumulated depreciation$18.0M7 Dec 31, 2024$14.5M7 Dec 31, 2023+24.3%
Property and equipment, net$11.6M7 Dec 31, 2024$14.8M7 Dec 31, 2023−21.4%
Operating-lease assets (leased space)$33.6M7 Dec 31, 2024$35.0M7 Dec 31, 2023−4.0%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$60.7M7 Dec 31, 2024$75.0M7 Dec 31, 2023−19.1%
Marketable securities, current$65.0M7 Dec 31, 2024$21.5M7 Dec 31, 2023+202.1%
Debt securities available for sale$65.0M7 Dec 31, 2024$21.5M7 Dec 31, 2023+202.1%

Properties, in its own words

Our corporate headquarters are located in Redwood City, California, consisting of approximately 79,675 square feet of office and laboratory space under a lease that will expire in December 2031.

In addition, we lease a manufacturing facility in North Carolina, consisting of approximately 173,820 square feet, under a lease that will expire in October 2037.

The rest of Item 2

We believe that our properties are adequate and suitable for our current needs; however, we are continuing to evaluate our real estate strategy in response to the changing needs of our in-office, hybrid and remote workforce.

Item 2 of the 10-K filed Apr 15, 2025.16

The story

Avalanche Biotechnologies was founded in 2006 by Tom Chalberg, Mark Blumenkranz, Mitchell Finer, and Steven Schwartz. In 2015, a year after raising $102 million when it went public through an IPO, Chalberg stepped down as CEO following Phase IIa trial results that were labeled as "iffy".

Upon acquiring Annapurna Therapeutics and changing its name to Adverum Biotechnologies in May 2016, it also changed its ticker symbol on Nasdaq from AAVL to ADVM. In October, former Annapurna CEO Amber Salzman became CEO of Adverum.

In May 2018, Salzman left Adverum, and Chief Medical Officer Athena Countouriotis resigned after less than one year in the job. In October 2018, Leone Patterson was named the new CEO of Adverum.

In September 2019, Adverum announced six-month data from the first cohort of its phase I trial. The stock price dropped 50% due to investor concerns over deterioration of vision that these trial subjects suffered, as well as several reports of occular inflammation.

In 2020, Laurent Fischer replaced Patterson as CEO of Adverum. In January 2022, the FDA granted Orphan Drug Designation to Adverum's ADVM-062, developed as a single intravitreal administration for blue-cone monochromacy by delivering a functional copy of the OPN1LW gene.

In September 2022, Adverum announced that its first patient was dosed in the LUNA phase 2 clinical trial; the trial evaluates development for the treatment of wet age-related macular degeneration.

In December 2025, it was announced that the company had been acquired by Eli Lilly and Company.

From Wikipedia’s article on Adverum Biotechnologies, shared under CC BY-SA 4.0.5

Leadership

Who has led Avalanche Biotechnologies, and when. Pick a name to see everywhere that person has worked.

Board members and other officers (41)

Named in SEC Form D filings

People the company listed in its private-offering notices.

  • Executive OfficerKishor Peter Soparkar named Feb 21, 202472
  • DirectorC. David Nicholson named Feb 21, 202472
  • DirectorDawn Gurses Svoronos named Feb 21, 202472
  • DirectorJames Gurses Scopa named Feb 21, 202472
  • DirectorPatrick Machado named Feb 21, 202472

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • Adverum NC, LLC North Carolina, USA6
  • Annapurna Therapeutics, LTD Ireland6
  • Avalanche Australia PTY LTD Australia6

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

Former names

  • Adverum Biotechnologies, Inc.May 12, 2016 to Dec 19, 20254
  • Avalanche Biotechnologies, Inc.Nov 26, 2013 to May 10, 20164

Timeline

  1. Dec 9, 20258-K Completed an acquisition or sale; Listing notice or transfer; Change to shareholders' rights73
  2. Dec 4, 20258-K Regulation FD disclosure; Other events74
  3. Nov 21, 20258-K Ended a material agreement; New debt or obligation; Other events75
  4. Oct 24, 20258-K Entered a material agreement; New debt or obligation; Regulation FD disclosure76
  5. Sep 22, 20258-K Regulation FD disclosure; Other events77
  6. Aug 12, 20258-K Results of operations78
  7. Jun 18, 20258-K Executive or director change; Shareholder vote79
  8. May 14, 20258-K Results of operations80
  9. Apr 15, 20258-K Results of operations81
  10. Mar 31, 20258-K Earlier financial statements no longer reliable; Other events82
  11. Nov 18, 20248-K Regulation FD disclosure; Other events83
  12. Nov 4, 20248-K Results of operations84
  13. Jun 17, 2020New chief executive Laurent Fischer becomes CEO17
  14. Jun 18, 2018New chief executive Leone D. Patterson becomes CEO18
  15. Feb 13, 2017New chief executive Amber Salzman becomes CEO19
  16. Dec 11, 2015New chief executive Paul B. Cleveland becomes CEO21
  17. Aug 25, 2015New chief executive Hans Hull becomes CEO20
  18. Jul 30, 2014New chief executive Thomas W. Chalberg becomes CEO22
  19. Jan 1, 2006Founded Founded1

Calendar

When it reported results

  1. Aug 12, 2025Reported quarterly or annual results78
  2. May 14, 2025Reported quarterly or annual results80
  3. Apr 15, 2025Reported quarterly or annual results81
  4. Nov 4, 2024Reported quarterly or annual results84
  5. Aug 12, 2024Reported quarterly or annual results85
  6. May 9, 2024Reported quarterly or annual results86
  7. Mar 18, 2024Reported quarterly or annual results87
  8. Nov 9, 2023Reported quarterly or annual results88

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

Filings

342 filings to the SEC since October 2021: 4 annual reports, 13 quarterly reports, 52 major events, 143 insider forms, 57 large holders, 1 private raises, 72 other. The latest 20:

  1. Dec 19, 202515-12G EDGAR
  2. Dec 11, 2025EFFECT EDGAR
  3. Dec 11, 2025EFFECT EDGAR
  4. Dec 11, 2025EFFECT EDGAR
  5. Dec 10, 20254 EDGAR
  6. Dec 10, 20254 EDGAR
  7. Dec 10, 20254 EDGAR
  8. Dec 10, 20254 EDGAR
  9. Dec 10, 20254 EDGAR
  10. Dec 10, 20254 EDGAR
  11. Dec 10, 20254 EDGAR
  12. Dec 10, 20254 EDGAR
  13. Dec 10, 20254 EDGAR
  14. Dec 10, 20254 EDGAR
  15. Dec 10, 20254 EDGAR
  16. Dec 10, 20254 EDGAR
  17. Dec 10, 20254 EDGAR
  18. Dec 10, 20254 EDGAR
  19. Dec 9, 2025SCHEDULE 13D/A EDGAR
  20. Dec 9, 2025SCHEDULE 13G/A EDGAR

The 342 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

52 open-market trades by officers, directors and large owners since October 2021: 35 buys and 17 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Dec 8, 2025Fischer LaurentCEO, PRESIDENT AND DIRECTORBuy117,614$4.31$506.9KForm 4
Dec 5, 2025Soparkar PeterCHIEF OPERATING OFFICERBuy50,000$4.22$211.0KForm 4
Dec 5, 2025Fischer LaurentCEO, PRESIDENT AND DIRECTORBuy11,085$4.24$47.0KForm 4
Dec 4, 2025Leonard Braden Michael10% ownerSale524,560$4.19$2.2MForm 4
Dec 3, 2025Leonard Braden Michael10% ownerSale222,925$4.17$929.6KForm 4
Dec 2, 2025Leonard Braden Michael10% ownerSale51,961$4.20$218.2KForm 4
Dec 1, 2025Leonard Braden Michael10% ownerSale300,000$4.20$1.3MForm 4
May 15, 2025Soparkar PeterCHIEF OPERATING OFFICERSale2,960$2.00$5.9KForm 4
May 15, 2025Seyedkazemi SetarehCHIEF DEVELOPMENT OFFICERSale924$2.00$1.8KForm 4
May 15, 2025Fischer LaurentCEO, PRESIDENT AND DIRECTORSale9,126$2.00$18.3KForm 4
Apr 23, 2025Leonard Braden Michael10% ownerBuy50,000$2.98$149.0KForm 4
Apr 22, 2025Leonard Braden Michael10% ownerBuy300,000$3.00$900.0KForm 4
Apr 2, 2025Leonard Braden Michael10% ownerBuy15,209$4.04$61.4KForm 4
Mar 31, 2025Leonard Braden Michael10% ownerBuy19,566$4.38$85.7KForm 4
Mar 27, 2025Leonard Braden Michael10% ownerBuy16,593$5.19$86.1KForm 4
Mar 27, 2025Leonard Braden Michael10% ownerBuy16,593$5.19$86.1KForm 4
Mar 27, 2025Leonard Braden Michael10% ownerBuy16,593$5.19$86.1KForm 4
Mar 26, 2025Leonard Braden Michael10% ownerBuy20,407$5.04$102.9KForm 4
Mar 26, 2025Leonard Braden Michael10% ownerBuy20,407$5.04$102.9KForm 4
Mar 26, 2025Leonard Braden Michael10% ownerBuy20,407$5.04$102.9KForm 4

All 52 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
2024Laurent FischerPresident and Chief Executive Officer61$691.8K$4.1M112
2024Kishor Peter SoparkarChief Operating Officer54$509.3K$1.8M112
2024Linda RubinsteinChief Financial Officer58$499.6K$1.6M112
2024Setareh SeyedkazemiChief Development Officer51$497.5K$1.6M112
2024Rabia Gurses OzdenChief Medical Officer57$281.2K$1.3M112
2023Linda RubinsteinChief Financial Officer58$198.4K$2.3M112
2023Laurent FischerPresident and Chief Executive Officer61$671.6K$1.5M112
2023Kishor Peter SoparkarChief Operating Officer54$494.5K$903.0K112
2023Setareh SeyedkazemiChief Development Officer51$483.0K$799.4K112
2022Laurent FischerPresident and Chief Executive Officer61$645.8K$2.9M112
2022Setareh SeyedkazemiChief Development Officer51$459.2K$1.5M112
2022Kishor Peter SoparkarChief Operating Officer54$473.2K$1.3M112
2022Linda RubinsteinChief Financial Officer58$111.9K112

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$127.8M 2024–2024
Money received by yearMoney received by year, 2024–2024: $127.8M in all. Private raises (Form D) $127.8M.$0$50M$100M$150M2024 · Private raises (Form D): $127.8M2024
Figures by year
YearPrivate raises (Form D)Total
2024$127.8M$127.8M

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. Feb 7, 2024Raised $127.8 million in a private offering of equity72 Form D: 20 investors so far Form D

SEC filer profile

How Avalanche Biotechnologies is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARAdverum Biotechnologies, Inc.2
  • CIK0001501756
  • Employer ID (EIN)20-525832716
  • Filer statusNon-accelerated filer not an emerging growth company; a smaller reporting company; not a shell company16
  • Incorporated inDelaware16
  • 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 address100 Cardinal Way, Redwood City, CA 940632
  • Phone(650) 649-10042
  • Shares outstanding22,077,467 as of Nov 7, 202512
  • Public float$136.7M as of Jun 30, 202416
  • AuditorErnst & Young LLP, San Francisco, California PCAOB ID 42; no auditor attestation of internal control over financial reporting16
  • On EDGAR sinceNov 26, 20132
  • Filings949 in all: 4 (359), 8-K (160), 3 (55), SC 13G/A (51), SC 13G (39), 10-Q (35), DEFA14A (34), S-8 POS (20)2
    All 59 form types

    S-8 (19), DFAN14A (17), 10-K (11), CORRESP (10), 4/A (9), 424B5 (9), DEF 14A (9), EFFECT (9), SCHEDULE 13G/A (9), UPLOAD (9), CT ORDER (7), S-1/A (7), SC 13D/A (7), 3/A (4), D (4), S-3 (4), ARS (3), POS AM (3), SC 13D (3), SCHEDULE 13D/A (3), 424B4 (2), 8-K/A (2), DEFC14A (2), PRE 14A (2), PREC14A (2), S-1 (2), S-1MEF (2), SC 14D9/A (2), SCHEDULE 13G (2), 10-K/A (1), 10-Q/A (1), 15-12G (1), 25-NSE (1), 424B3 (1), 8-A12B (1), CERTNAS (1), DEFM14A (1), DRS (1), DRS/A (1), NT 10-K (1), PREM14A (1), PRER14A (1), PRRN14A (1), S-3/A (1), S-3ASR (1), SC 14D9 (1), SC TO-C (1), SC TO-T (1), SC TO-T/A (1), SC14D9C (1), SCHEDULE 13D (1)

  • Latest filingsAnnual report (10-K) Apr 15, 2025
    Quarterly report (10-Q) Nov 12, 2025
    Proxy statement (DEF 14A) Apr 28, 2025
    Latest 8-K (8-K) Dec 9, 2025

Securities registered for trading

ClassSymbolExchange
Common Stock, par value $0.0001 per shareADVMNasdaq

From the cover page of the 10-K filed Apr 15, 2025 (Section 12(b) of the Exchange Act).16

Risk factors and legal proceedings

The risks Avalanche Biotechnologies names in Item 1A of its 10-K filed Apr 15, 2025: 72 risks under these headings, as written.16

  • You should consider carefully the risks and uncertainties described below, together with all of the other information in this Annual Report on Form 10-K. If any of the following risks are realized, our business, financial condition, results of operations and prospects could be materially and adversely affected. The risks described below are not the only risks facing us. Risks and uncertainties not currently known to us or that we currently deem to be immaterial also may materially adversely affect our business, financial condition, results of operations and prospects.
  • Risks Related to Our Financial Position and Need for Capital
  • There is substantial doubt regarding our ability to continue as a going concern. We will need to raise substantial additional funding to finance our operations through regulatory approval of our lead program and beyond, which may not be available on acceptable terms, or at all. If we fail to obtain additional capital necessary to fund our operations, we could be forced to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations, or to cease operations or liquidate our assets.
  • If we sell shares of our common stock or securities convertible into or exercisable for shares of our common stock in future financings or pursuant to licensing, collaboration or other arrangements, stockholders may experience immediate dilution and, as a result, our stock price may decline.
  • The report of our independent registered public accounting firm for the year ended December 31, 2024 contains an explanatory paragraph regarding substantial doubt about our ability to continue as a going concern.
  • We have incurred significant operating losses since inception, and we expect to incur significant losses for the foreseeable future. We may never become profitable or, if achieved, be able to sustain profitability.
  • We are subject to risks associated with subletting our leased premises, including risks associated with subtenant defaults, which have occurred and we expect to continue to occur.
  • Risks Related to the Discovery and Development of Our Product Candidates
  • Our business will depend substantially on the success of one or more of our product candidates. If we are unable to develop, obtain regulatory approval for, or successfully commercialize, any or all of our product candidates, our business will be materially harmed.
  • Drug development is a long, expensive and uncertain process, and delay or failure can occur at any stage of development, including after commencement of any of our clinical trials or any clinical trials using our proprietary viral vectors.
  • The occurrence of serious complications or side effects that outweigh the therapeutic benefit in connection with or during use of our product candidates, whether in nonclinical studies or clinical trials or post-approval, could lead to discontinuation of our clinical development program, refusal of regulatory authorities to approve our product candidates or, post-approval, revocation of marketing authorizations or refusal to approve new indications, which could severely harm our business prospects, financial condition and results of operations.
  • The results of nonclinical studies and early clinical trials are not always predictive of future results. Any product candidate we or any of our future development partners advance into clinical trials may not have favorable results in later clinical trials, if any, or receive regulatory approval.
The other 62 risks
  • Our gene therapy platform is based on a novel technology, which makes it difficult to predict the time and cost of product candidate development and the time, cost, and probability of subsequently obtaining regulatory approval.
  • If we encounter difficulties enrolling patients in our clinical trials, our clinical development activities could be delayed or otherwise adversely affected.
  • Our product candidates are subject to extensive regulation, compliance with which is costly and time consuming, and such regulation may cause unanticipated delays or prevent the receipt of the required approvals to commercialize our product candidates.
  • Preliminary and interim data from our clinical trials that we may announce or publish from time to time may change as each clinical trial progresses.
  • Fast Track and Regenerative Medicine Advanced Therapy (“RMAT”) designations by the FDA, PRIME designation by the EMA and the Innovation Passport by the MHRA for Ixo-vec may not lead to a faster development, regulatory review or approval, and they do not increase the likelihood that Ixo-vec will receive marketing approval in the U.S.
  • We may not be successful in our efforts to identify or discover additional product candidates.
  • Risks Related to Manufacturing
  • If we are unable to successfully develop and maintain robust and reliable manufacturing processes for our product candidates, we may be unable to advance clinical trials or licensure applications and may be forced to delay or terminate a program.
  • If we are unable to produce sufficient quantities of our products and product candidates at acceptable costs, we may be unable to meet clinical or potential commercial demand, face delayed timelines, lose potential revenue, have reduced margins, or be forced to terminate a program.
  • Changes in methods of manufacturing or formulation of our product candidates may result in additional costs or delays.
  • We and our contractors are subject to significant regulation with respect to manufacturing and testing our product candidates. We have a limited number of vendors on which we rely, including, in some cases, single source vendors, and the contract vendors on which we rely may not continue to meet regulatory requirements, may have limited capacity, or may have other factors limiting their ability to comply with their contracts with us.
  • We may face difficulties from changes to current regulations and future legislation.
  • The United Kingdom’s withdrawal from the EU may have a negative effect on global economic conditions, financial markets and our business.
  • We are subject to many manufacturing and distribution risks, any of which could substantially increase our costs and limit supply of our product candidates.
  • Risks Related to Our Reliance on Third Parties
  • We have relied, and expect to continue to rely, on third parties under contracts and partnerships to conduct some or all aspects of our research and development, including vector production, process development, assay development, product candidates and product manufacturing and testing, protocol development, clinical trials, product distribution, commercialization, nonclinical studies, research and related activities, and these third parties may not perform satisfactorily.
  • We rely and will continue to rely on third parties to conduct some nonclinical testing and all of our ongoing and planned clinical trials. If these third parties do not meet our deadlines or otherwise fail to conduct the trials as required, our clinical development programs could be delayed or unsuccessful and we may not be able to obtain regulatory approval for or commercialize our product candidates when expected or at all.
  • Risks Relating to Our Intellectual Property
  • Our success depends on our ability to protect our intellectual property and our proprietary technologies.
  • Our reliance on third parties requires us to share our trade secrets and other confidential information, which increases the possibility that a competitor will discover them or that our confidential information, including trade secrets, will be misappropriated or disclosed.
  • Claims by third parties that we infringe their proprietary rights may result in liability for damages or prevent or delay our developmental and commercialization efforts.
  • We may not be successful in obtaining or maintaining necessary rights to our product candidates through acquisitions and in-licenses.
  • Our rights to develop and commercialize our product candidates are subject in part to the terms and conditions of licenses granted to us by other companies and universities.
  • The patent protection and patent prosecution for some of our product candidates are dependent on third parties.
  • We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
  • Third-party patent rights could delay or otherwise adversely affect our planned development and sale of product candidates of our programs.
  • We may not be able to obtain intellectual property rights or protect our intellectual property rights throughout the world.
  • Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our product candidates.
  • If we do not obtain patent term extensions for patents covering our product candidates, our business may be materially harmed.
  • We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming, and unsuccessful. Further, our issued patents could be found invalid or unenforceable if challenged administratively or in court.
  • Some intellectual property that we have in-licensed or may in-license may have been discovered through government funded programs and thus may be subject to federal regulations such as “march-in” rights, certain reporting requirements and a preference for U.S.-based companies. Compliance with such regulations may limit our exclusive rights and limit our ability to contract with non-U.S. manufacturers.
  • We may fail to comply with any of our obligations under existing agreements pursuant to which we license or have otherwise acquired intellectual property rights or technology, which could result in the loss of rights or technology that are material to our business.
  • Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
  • We may be subject to claims that we have wrongfully hired an employee from a competitor or that we or our employees have wrongfully used or disclosed alleged confidential information or trade secrets of their former employers.
  • If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets of interest and our business may be adversely affected.
  • Risks Related to Commercialization of Our Product Candidates
  • Any suspension of, or delays in the commencement or completion of, clinical trials for our product candidates could result in increased costs to us, delay or limit our ability to generate revenue and adversely affect our commercial prospects.
  • Final marketing approval for our product candidates by the FDA or other regulatory authorities outside the U.S. for commercial use may be delayed, limited or denied, any of which would adversely affect our ability to generate operating revenue.
  • Even if we receive regulatory approval, we still may not be able to successfully commercialize any of our product candidates, and the revenue that we generate from product sales, if any, could be limited.
  • If our competitors develop treatments for the target indications of our product candidates that are approved, marketed more successfully, or demonstrated to be safer or more effective or easier to administer than our product candidates, our commercial opportunity will be reduced or eliminated.
  • Even if we obtain marketing approval for any of our product candidates, they could be subject to restrictions or withdrawal from the market, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our product candidates, when and if any of them are approved.
  • Coverage and reimbursement may be limited or unavailable in certain market segments for our product candidates, which could make it difficult for us to sell our product candidates profitably.
  • Healthcare and other reform legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and, if approved, may affect the prices we may obtain.
  • If the market for Ixo-vec, if approved, in the treatment of wet AMD or any other indication we seek to treat is smaller than we believe it is, or if our product candidate is approved with limitations that reduce the market size, or if this occurs for any of our other product candidates, our future revenue may be adversely affected, and our business may suffer.
  • If we do not achieve our projected development goals in the time frames we announce and expect, the commercialization of our product candidates, if approved, may be delayed and the credibility of our management team may be adversely affected and, as a result, our stock price may decline.
  • Due to the novel nature of our technology and the potential for our product candidates to offer therapeutic benefit in a single administration, we face uncertainty related to pricing and reimbursement for these product candidates.
  • We may not be successful in establishing and maintaining development, commercialization or other strategic collaborations, which could adversely affect our ability to develop and commercialize product candidates and receive milestone and/or royalty payments.
  • Governments may impose price controls, which may adversely affect our future profitability.
  • We may form strategic alliances in the future, and we may not realize the benefits of such alliances.
  • We have no sales, marketing, distribution, or market access and reimbursement capabilities, and we would have to establish a partnering arrangement and/or invest significant resources to develop these capabilities.
  • Risks Related to Our Business Operations
  • Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage public perception of our product candidates or adversely affect our ability to conduct our business or obtain marketing approvals for our product candidates.
  • We are dependent on the services of our key executives and clinical and scientific staff, and if we are not able to retain these members of our management or recruit additional management, clinical and scientific personnel, our business will suffer.
  • We may encounter difficulties in managing our growth and expanding our operations successfully.
  • If our information technology systems or those third parties with whom we work, or our data, are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; material disruption of our product development programs; and other adverse consequences.
  • If we fail to comply with applicable state and federal healthcare laws and regulations, we may be subject to civil or criminal penalties and/or exclusion from federal and/or state healthcare programs, or foreign equivalents.
  • If product liability lawsuits are brought against us, we may incur substantial liabilities and may be required to limit commercialization of our product candidates.
  • We are subject to certain U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions, and other trade laws and regulations (collectively, “Trade Laws”). We can face serious consequences for violations.
  • We and our development partners, third-party manufacturers and suppliers use biological materials and use or may use hazardous materials, and any claims relating to improper handling, storage or disposal of these materials could be time consuming or costly.
  • We and any of our development partners will be required to report to regulatory authorities if any of our approved products cause or contribute to adverse medical events, and any failure to do so would result in sanctions that would materially harm our business.
  • Our employees, independent contractors, principal investigators, CROs, CMOs, consultants and vendors may engage in misconduct or other improper activities, including noncompliance with our code of conduct or regulatory standards and requirements.
  • Our ability to use net operating loss carryforwards and other tax attributes may be limited by the Code.
  • Potential natural disasters, some possibly related to the increasing effects of climate change, could damage, destroy or disrupt operations at our office and laboratories, CMO premises, study sites, and/or other third-party vendor sites, which could have a significant negative impact on our operations.
  • Risks Related to Our Common Stock
  • The trading price of the shares of our common stock has been and could continue to be highly volatile, and purchasers of our common stock could incur substantial losses.
  • Anti-takeover provisions in our charter documents and under 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.
  • We have identified a material weakness in our internal control over financial reporting. If we are unable to remedy the material weakness, or if we fail to maintain an effective system of internal control over financial reporting in the future, we may not be able to accurately report our financial condition, results of operations or cash flows, which may adversely affect investor confidence in our company and, as a result, the value of our common stock.
  • Our quarterly operating results may fluctuate significantly.

Legal proceedings

In October 26, 2021, we entered into a sublease agreement with Jaguar, who subsequently assigned the sublease to AMP, as the subtenant for the NC Premises, for the remainder of the underlying lease term (ending in October 2037). Pursuant to the sublease and the assignment agreement, Jaguar remained obligated for AMP’s proper performance under the sublease. In February 2025, a lien in the amount of $4.8 million was filed by a third-party contractor against the subtenant’s interest in the NC Premises. We discharged the lien in March 2025 by depositing cash in the full amount with the applicable court in order to avoid a default under the head lease. Further liens by third-party subcontractors or other contractors have been or may in the future be filed against the subtenant’s interest in the NC Premises and we may need to also discharge such liens to avoid a default under the head lease. The subtenant and Jaguar failed to remit March 2025 rent and subsequent rent payments and defaulted, and failed to cure such defaults, under the sublease and the assignment agreement for the NC Premises. As a result, we assumed responsibility for such payments in March 2025. As of April 7, 2025, we have made payments in the total amount of $1.9 million to satisfy outstanding rent and common area management fee obligation, and we remain obligated under the head lease, including for all future remaining rent payments in an aggregate amount of up to $119.7 million for the remainder of the head lease term, of which $7.1 million is due between April 8 and December 31, 2025. As a result of the uncured defaults, we terminated the sublease and initiated a lawsuit against the subtenant and Jaguar in the Superior Court of Wake County, North Carolina to enforce our rights under the sublease and seek recovery of losses and damages due to the defaults by the subtenant and Jaguar.

The opening of Item 3, Legal Proceedings.16

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