Sign in

TSHA on Nasdaq

Taysha Gene Therapies, Inc.

In its 2026 annual report the company says: “We are a clinical-stage biotechnology company focused on advancing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system, or CNS. Our lead clinical program TSHA-102 is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease.”3 It is headquartered in Dallas, Texas.1

Price unavailable

Last updated Oct 21, 2026.

Overview

Compare with CURIS INC, Alpha Cognition Inc. or TScan Therapeutics, Inc., or pick any stock.

  • TickerTSHA on Nasdaq1
  • IndustryBiological Products, (No Diagnostic Substances)1
  • HeadquartersDallas, Texas1
  • Incorporated inDE2
  • Business address3000 Pegasus Park Drive, Suite 1430, Dallas, Tx 752472
  • Filer categoryNon-accelerated filer, Smaller reporting company2
  • Legal formCorporation

See how the biggest companies compare on our leaderboards

We are a clinical-stage biotechnology company focused on advancing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system, or CNS. Our lead clinical program TSHA-102 is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, we aim to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. Our management team has proven experience in gene therapy development and commercialization. We leverage this experience, our manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside.3 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

-1130.6%Operating margin4
-1115.3%Net margin4
17.3%Revenue growth, 20254
-44.1%Return on equity4
0.20×Debt to equity4
-$93.8MFree cash flow4

Where the money goes

Where each dollar of revenue went, fiscal 2025

  • Research and development100¢$86.4M

Out of $9.8M in revenue.4 The company lost money this year.

Financial statements

Revenue564$9.8M in 2025+291% since 2022
0$5.0M$10.0M$15.0M$20.0M2022: $2.5M$2.5M20222023: $15.5M$15.5M2023+518%2024: $8.3M$8.3M2024−46%2025: $9.8M$9.8M2025+17%
Operating income78564-$110.5M in 2025+157% since 2020
-$200.0M-$150.0M-$100.0M-$50.0M02020: -$43.0M-$43.0M20202021: -$173.3M-$173.3M2021+303%2022: -$162.4M-$162.4M2022−6%2023: -$72.4M-$72.4M2023−55%2024: -$91.5M-$91.5M2024+26%2025: -$110.5M-$110.5M2025+21%
Net income78564-$109.0M in 2025+82% since 2020
-$200.0M-$150.0M-$100.0M-$50.0M02020: -$60.0M-$60.0M20202021: -$174.5M-$174.5M2021+191%2022: -$166.0M-$166.0M2022−5%2023: -$111.6M-$111.6M2023−33%2024: -$89.3M-$89.3M2024−20%2025: -$109.0M-$109.0M2025+22%

Every line of the income statement as filed: the last 2 fiscal years, latest first.4

Period ending2025-12-312024-12-31
Revenue$9.8M$8.3M
Research and development$86.4M$66.0M
General and administrative$33.9M$29.0M
Impairment of long-lived assets$0$4.8M
Total operating expenses$120.3M$99.8M
Loss from operations-$110.5M-$91.5M
Change in fair value of warrant liability-$1.2M$16.0K
Change in fair value of term loan-$6.2M-$4.6M
Interest income$9.2M$6.9M
Interest expense-$63.0K-$102.0K
Other expense-$291.0K-$110.0K
Total other income, net$1.5M$2.2M
Net loss-$109.0M-$89.3M
Net loss per common share, basic-$0.34-$0.36
Net loss per common share, diluted-$0.34-$0.36
Weighted average common shares outstanding, basic319.7M shares250.1M shares
Weighted average common shares outstanding, diluted319.7M shares250.1M shares

Compare Taysha Gene Therapies, Inc. with another company

Corporate structure

Taysha Gene Therapies, Inc. sits at the top of its group. 1 entity, 1 country on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Taysha Gene Therapies, Inc.

  • RA CAPITAL MANAGEMENT, L.P. Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest Aug 14, 2026 (6 filings since Aug 25, 2023)
  • Morgan Stanley Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest Aug 13, 2026 (3 filings since Feb 5, 2025)
  • RTW INVESTMENTS, LP Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest May 15, 2026 (6 filings since Feb 14, 2024)
  • Vestal Point Capital, LP Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest May 15, 2026 (2 filings since Feb 17, 2026)
  • FMR LLC Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest Feb 5, 2026 (9 filings since Feb 9, 2022)
  • BlackRock Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest Jan 21, 2026 (6 filings since Feb 7, 2022)
  • Octagon Capital Advisors LP Filed SCHEDULE 13G/A on Taysha Gene Therapies, Inc., latest Nov 14, 2025 (2 filings since Feb 5, 2025)
  • Goldman Sachs Filed SCHEDULE 13G on Taysha Gene Therapies, Inc., latest Jul 3, 2025
  • Paul B. Manning Filed SCHEDULE 13D/A on Taysha Gene Therapies, Inc., latest Jun 3, 2025 (3 filings since Nov 2, 2022)
  • Avoro Capital Advisors LLC Filed SC 13G on Taysha Gene Therapies, Inc., latest Nov 14, 2024
  • Venrock Healthcare Capital Partners III, L.P. Filed SC 13G/A on Taysha Gene Therapies, Inc., latest Nov 14, 2024 (3 filings since Aug 28, 2023)
  • TCG Crossover GP I, LLC Filed SC 13G/A on Taysha Gene Therapies, Inc., latest Feb 9, 2024 (2 filings since Aug 25, 2023)
  • Taysha Gene Therapies, Inc. Dallas, Texas

Built from major holders from Schedules 13D and 13G. Each entity in the chart links to its source.

What it owns

What Taysha Gene Therapies, Inc. 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$11.4M4 Dec 31, 2025$11.1M4 Dec 31, 2024+3.2%
Accumulated depreciation$4.7M4 Dec 31, 2025$3.6M4 Dec 31, 2024+30.7%
Property and equipment, net$6.7M4 Dec 31, 2025$7.5M4 Dec 31, 2024−10.0%
Operating-lease assets (leased space)$9.4M4 Dec 31, 2025$8.4M4 Dec 31, 2024+12.6%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$319.8M4 Dec 31, 2025$139.0M4 Dec 31, 2024+130.0%

Properties, in its own words

In January 2021, we entered into a lease agreement for 15,000 square feet of administrative space in Dallas, Texas, pursuant to a lease agreement that expires approximately ten years after the lease commenced in May 2021. This lease was amended in December 2021 to lease an additional 18,000 square feet of administrative space in Dallas, TX, pursuant to a lease amendment that expires approximately ten years after the lease commencement date. In December 2020, we entered into a lease agreement for approximately 187,500 square feet of a manufacturing facility in Durham, North Carolina, pursuant to a lease agreement that expires in September 2036. We have two options to extend the term of that lease, each for a period of an additional five years. In November 2025, we entered into a lease agreement for 15,000 square feet of laboratory space in Morrisville, North Carolina pursuant to a lease agreement that expires five years after the lease commencement date. We have the option to extend the term of the lease for a period of three years.

We believe that our facilities are suitable and adequate to meet our needs. We intend to add new facilities or expand existing facilities as we add employees, and we believe that suitable additional or substitute space will be available as needed to accommodate any such expansion of our operations.

Item 2 of the 10-K filed Mar 19, 2026.3

Leadership

Who has led Taysha Gene Therapies, Inc., and when. Pick a name to see everywhere that person has worked.

Chief executive

  1. R.a. Session Ii.2020–2022
  2. Sean P. Nolan2023–now

President

  1. R.a. Session Ii.2020–2022
  2. Sukumar Nagendran2023–now
2025
Board members and other officers (10)

Named in SEC Form D filings

People the company listed in its private-offering notices.

  • Executive OfficerKamran P. Alam named Nov 7, 2022 to Aug 25, 202324
  • Executive Officer, DirectorRA Session II named Nov 7, 202225

Timeline

  1. Aug 11, 20268-K Results of operations26
  2. Jun 25, 20268-K Entered a material agreement; Other events27
  3. Jun 22, 20268-K Regulation FD disclosure; Other events28
  4. Jun 2, 20268-K Shareholder vote29
  5. May 6, 20268-K Results of operations30
  6. Mar 19, 20268-K Results of operations31
  7. Nov 4, 20258-K Entered a material agreement; Results of operations32
  8. Oct 16, 20258-K Other events33
  9. Oct 9, 20258-K Other events34
  10. Oct 2, 20258-K Other events35
  11. Aug 12, 20258-K Results of operations36
  12. Jun 3, 20258-K Bylaws or fiscal year changed; Shareholder vote37
  13. Jan 4, 2023New chief executive Sean P. Nolan becomes CEO12
  14. Sep 23, 2020New chief executive R.a. Session Ii. becomes CEO13

Calendar

Coming up

  1. Nov 3, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.09) a share, on Nov 4, 2025.38

When it reported results

  1. Aug 11, 2026Reported quarterly or annual results26
  2. May 6, 2026Reported quarterly or annual results30
  3. Mar 19, 2026Reported quarterly or annual results31
  4. Nov 4, 2025Reported quarterly or annual results32
  5. Aug 12, 2025Reported quarterly or annual results36
  6. May 15, 2025Reported quarterly or annual results39
  7. Feb 26, 2025Reported quarterly or annual results40
  8. Nov 13, 2024Reported quarterly or annual results41

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

Filings

307 filings to the SEC since October 2021: 6 annual reports, 15 quarterly reports, 65 major events, 85 insider forms, 46 large holders, 2 private raises, 88 other. The latest 20:

  1. Aug 14, 2026SCHEDULE 13G/A EDGAR
  2. Aug 13, 2026SCHEDULE 13G/A EDGAR
  3. Aug 11, 202610-Q EDGAR
  4. Aug 11, 20268-K EDGAR
  5. Jul 10, 2026144 EDGAR
  6. Jul 10, 20264 EDGAR
  7. Jun 25, 20268-K EDGAR
  8. Jun 25, 2026424B5 EDGAR
  9. Jun 24, 2026424B5 EDGAR
  10. Jun 22, 20268-K EDGAR
  11. Jun 2, 20264 EDGAR
  12. Jun 2, 20264 EDGAR
  13. Jun 2, 20264 EDGAR
  14. Jun 2, 20264 EDGAR
  15. Jun 2, 20268-K EDGAR
  16. May 15, 2026SCHEDULE 13G/A EDGAR
  17. May 15, 2026SCHEDULE 13G/A EDGAR
  18. May 6, 202610-Q EDGAR
  19. May 6, 20268-K EDGAR
  20. Apr 22, 2026ARS EDGAR

The 307 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

47 open-market trades by officers, directors and large owners since October 2021: 16 buys and 31 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Jul 10, 2026Nagendran SukumarPresident and Head of R&DSale200,000$6.24$1.2MForm 4
Apr 10, 2026Nagendran SukumarPresident and Head of R&DSale200,000$4.46$892.0KForm 4
Feb 4, 2026Alam KamranCHIEF FINANCIAL OFFICERSale1,655$4.52$7.5KForm 4
Jan 26, 2026Alam KamranCHIEF FINANCIAL OFFICERSale23,849$4.61$109.9KForm 4
Jan 26, 2026Nagendran SukumarPresident and Head of R&DSale26,918$4.61$124.1KForm 4
Jan 26, 2026Nolan Sean P.Chief Executive OfficerSale41,312$4.61$190.4KForm 4
Jan 23, 2026Alam KamranCHIEF FINANCIAL OFFICERSale78,968$4.75$375.1KForm 4
Jan 23, 2026Nagendran SukumarPresident and Head of R&DSale89,132$4.75$423.4KForm 4
Jan 23, 2026Nolan Sean P.Chief Executive OfficerSale136,789$4.75$649.7KForm 4
Jan 12, 2026Nagendran SukumarPresident and Head of R&DSale200,000$4.71$942.0KForm 4
Dec 1, 2025Nagendran SukumarPresident and Head of R&DSale260,047$4.51$1.2MForm 4
Nov 28, 2025Nagendran SukumarPresident and Head of R&DSale110,125$4.75$523.1KForm 4
Sep 10, 2025Nagendran SukumarPresident and Head of R&DSale200,000$3.23$646.0KForm 4
Aug 21, 2025Nagendran SukumarPresident and Head of R&DSale57,054$2.85$162.6KForm 4
Aug 21, 2025Alam KamranCHIEF FINANCIAL OFFICERSale54,491$2.85$155.3KForm 4
May 30, 2025Manning Paul B10% ownerBuy750,000$2.75$2.1MForm 4
Jun 27, 2024Manning Paul B10% ownerBuy1,333,333$2.25$3.0MForm 4
Nov 17, 2023Manning Paul B10% ownerBuy100,000$1.63$163.0KForm 4
Aug 24, 2023Alam KamranCHIEF FINANCIAL OFFICERSale33,000$2.33$76.9KForm 4
Aug 16, 2023Manning Paul B10% ownerBuy16,466,667$0.90$14.8MForm 4

All 47 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
2025Sean NolanChief Executive Officer and Director58$645.8K$4.1M56
2025Kamran AlamChief Financial Officer48$473.3K$2.1M56
2025Sukumar Nagendran, M.D.President and Head of Research and Development and Director60$608.2K$2.1M56
2024Sean NolanChief Executive Officer and Director58$621.0K$4.7M56
2024Sukumar Nagendran, M.D.President and Head of Research and Development and Director60$584.8K$3.6M56
2024Kamran AlamChief Financial Officer48$455.1K$2.5M56

Money raised

Private offerings the company reported to the SEC on Form D.

  1. Aug 16, 2023Raised $150 million in a private offering of equity Form D: $150 million offered in all Form D
  2. Oct 21, 2022Raised $30 million in a private offering of equity 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
Avoro Capital Advisors LLC23,888,888$162.7M1.5%+2,188,888
Ra Capital Management, L.P.23,555,648$160.4M1.4%No change
Goldman Sachs Group Inc22,713,118$154.7M0.0%+343,473
BlackRock, Inc.20,691,927$140.9M0.0%+2,326,319
Vestal Point Capital, LP19,415,000$132.2M3.1%+1,415,000
Morgan Stanley16,487,479$112.3M0.0%−1,477,892
Rtw Investments, LP13,560,258$92.3M0.8%−119,990
Fmr LLC13,336,413$90.8M0.0%−984,842
Vanguard Capital Management LLC12,478,595$85.0M0.0%+2,247,093
State Street Corp11,421,920$77.8M0.0%+212,842
Siren, L.L.C.9,846,579$67.1M1.4%+1,016,666
Octagon Capital Advisors LP9,693,244$66.0M6.1%+833,333
Geode Capital Management, LLC6,560,394$44.7M0.0%+1,028,439
Polar Capital Holdings Plc5,117,133$34.8M0.1%−537,207
Mpm Bioimpact LLC4,500,000$30.6M1.9%New
D. E. Shaw & Co., Inc.4,398,652$30.0M0.0%−28,649
Baker Bros. Advisors LP4,004,756$27.3M0.1%No change
Wellington Management Group Llp3,975,605$27.1M0.0%+3,349,244
Healthcare Of Ontario Pension Plan Trust Fund3,885,501$26.5M0.0%+1,002,200
GordonMD Global Investments LP723,185$24.5M11.9%−14,365
Casdin Capital, LLC3,250,000$22.1M1.1%No change
Vanguard Portfolio Management LLC3,140,009$21.4M0.0%+1,117,511
ADAR1 Capital Management, LLC3,040,466$20.7M1.0%+61,037
Pictet Asset Management Holding SA2,770,701$18.9M0.0%+2,751,383
Aberdeen Group plc2,327,152$15.8M0.0%+1,306,089

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

SEC filer profile

How Taysha Gene Therapies, Inc. is registered with the Securities and Exchange Commission, from its EDGAR record and the cover page of its latest annual report.

  • Name on EDGARTaysha Gene Therapies, Inc.1
  • CIK0001806310
  • Employer ID (EIN)84-31995123
  • Filer statusNon-accelerated filer not an emerging growth company; a smaller reporting company; not a shell company3
  • Incorporated inDelaware3
  • Fiscal year endsDecember 311
  • Industry code (SIC)2836 Biological Products, (No Diagnostic Substances)1
  • SEC reviewing officeOffice of Life Sciences, Division of Corporation Finance1
  • Business address3000 Pegasus Park Drive, Suite 1430, Dallas, TX 752471
  • Phone(214) 612-00001
  • Shares outstanding325,833,545 as of Aug 11, 202610
  • Public float$544.8M as of Jun 30, 20253
  • AuditorDeloitte & Touche LLP, Chicago, IL PCAOB ID 34; no auditor attestation of internal control over financial reporting3
  • On EDGAR sinceMay 4, 20201
  • Filings401 in all: 4 (106), 8-K (85), 144 (32), 10-Q (18), SCHEDULE 13G/A (17), SC 13G/A (13), 3 (12), SC 13G (12)1
    All 40 form types

    424B5 (10), CORRESP (9), S-8 (8), DEF 14A (7), 10-K (6), UPLOAD (6), 8-K/A (5), DEFA14A (5), EFFECT (5), ARS (4), D (4), S-3 (4), SCHEDULE 13G (4), PRE 14A (3), S-1/A (3), SC 13D (3), SC 13D/A (3), 424B3 (2), SEC STAFF LETTER (2), 10-K/A (1), 144/A (1), 4/A (1), 424B4 (1), 5 (1), 8-A12B (1), CERT (1), DEFR14A (1), DRS (1), S-1 (1), S-1MEF (1), S-3ASR (1), SCHEDULE 13D/A (1)

  • Latest filingsAnnual report (10-K) Mar 19, 2026
    Quarterly report (10-Q) Aug 11, 2026
    Proxy statement (DEF 14A) Apr 22, 2026
    Latest 8-K (8-K) Aug 11, 2026

Securities registered for trading

ClassSymbolExchange
Common stock, par value $0.00001 per shareTSHAThe Nasdaq Stock Market LLC

From the cover page of the 10-K filed Mar 19, 2026 (Section 12(b) of the Exchange Act).3

Risk factors and legal proceedings

The risks Taysha Gene Therapies, Inc. names in Item 1A of its 10-K filed Mar 19, 2026: 70 risks under these headings, as written.3

  • Selected Risks Affecting Our Business
  • Risks Related to our Financial Position and Capital Needs
  • We have incurred significant losses since our inception. We expect to incur losses over the next several years and may never achieve or maintain profitability.
  • We have a limited operating history and no history of commercializing products, which may make it difficult for an investor to evaluate the success of our business to date and to assess our future viability.
  • We will need substantial additional funding to meet our financial obligations and to pursue our business objectives. If we are unable to raise capital when needed, we could be forced to curtail our planned operations and the pursuit of our growth strategy.
  • Our existing indebtedness contains restrictions that potentially limit our flexibility in operating our business. In addition, we may be required to make a prepayment or repay our outstanding indebtedness earlier than we expect under our 2025 Trinity Term Loan Agreement if we are unable to satisfy certain conditions.
  • Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
  • Unfavorable global economic conditions could adversely affect our business, financial condition or results of operations.
  • Risks Related to the Development of our Product Candidates
  • TSHA-102 is currently our lead product candidate and there is no guarantee that we will be able to successfully develop and commercialize TSHA-102.
  • All of our product candidates are in clinical or preclinical development. If we are unable to successfully develop, receive regulatory approval for and commercialize our product candidates for these or any other indications, or successfully develop any other product candidates, or experience significant delays in doing so, our business will be harmed.
  • We intend to identify and develop novel gene therapy product candidates, which makes it difficult to predict the time, cost and potential success of product candidate development.
The other 61 risks
  • Because gene therapy is novel and the regulatory landscape that governs any product candidates we may develop is rigorous, complex, uncertain and subject to change, we cannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for any product candidates we may develop.
  • Preclinical studies and clinical trials are expensive, time-consuming, difficult to design and implement and involve an uncertain outcome. Further, we may encounter substantial delays in completing the development of our product candidates.
  • The regulatory approval processes of the FDA, European Commission and comparable foreign authorities are lengthy, time consuming and inherently unpredictable. If we are not able to obtain required regulatory approval for our product candidates, our business will be substantially harmed.
  • Interim “top-line” and preliminary results from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.
  • We are substantially dependent on the success of our lead product candidate, TSHA-102. There is no guarantee that we will be able to successfully develop and commercialize TSHA-102. If we are unable to complete development of, obtain regulatory approval for and commercialize TSHA-102 in one or more indications and in a timely manner, our business, financial condition, results of operations and prospects will be significantly harmed.
  • We have not yet completed testing of any product candidate in clinical trials. Success in preclinical studies or earlier clinical trials, including our Phase 1/2 REVEAL trials of TSHA-102, may not be indicative of results in future clinical trials, or provide support for the submission of a marketing application.
  • Our preclinical studies and clinical trials may fail to demonstrate the safety and efficacy of our product candidates, or serious adverse or unacceptable side effects may be identified during the development of our product candidates, which could prevent or delay regulatory approval and commercialization, increase our costs or necessitate the abandonment or limitation of the development of some of our product candidates.
  • As an organization, we have not previously conducted pivotal clinical trials such as REVEAL Part B.
  • The disorders we seek to treat have low prevalence and it may be difficult to identify and enroll patients with these disorders. If we experience delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
  • We may seek orphan drug designation for some of our product candidates and we may be unsuccessful, or may be unable to maintain the benefits associated with orphan drug designation, including the potential for market exclusivity, for product candidates for which we obtain orphan drug designation.
  • We have received rare pediatric disease designation for TSHA-102 for the treatment of Rett syndrome. However, a marketing application for TSHA-102, if approved, may not meet the eligibility criteria for a PRV or the rare pediatric disease designation program may sunset before FDA is able to consider us for a voucher.
  • We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
  • We are currently conducting our TSHA-102 REVEAL trials in Canada and plan to conduct in the future additional clinical trials for our product candidates outside the United States, and the FDA and similar foreign regulatory authorities may not accept data from such trials.
  • We may not be successful in our efforts to build a pipeline of additional product candidates.
  • If we do not achieve our projected development goals in the time frames we announce and expect, the commercialization of our products may be delayed.
  • The United Kingdom’s withdrawal from the European Union may adversely impact our ability to obtain regulatory approvals of our product candidates in the European Union, result in restrictions or imposition of taxes and duties for importing our product candidates into the European Union and require us to incur additional expenses in order to develop, manufacture and commercialize our product candidates in the European Union.
  • Risks Related to the Manufacturing of our Product Candidates
  • Gene therapies 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.
  • We and our contract manufacturers for AAV9 are subject to significant regulation with respect to manufacturing our products. The third-party manufacturing facilities on which we rely may have limited capacity or fail to meet the applicable stringent regulatory requirements.
  • We depend on third-party suppliers for materials used in the manufacture of our product candidates, and the loss of these third-party suppliers or their inability to supply us with adequate materials could harm our business.
  • Any contamination or interruption in our manufacturing process, shortages of raw materials or failure of our suppliers of plasmids and viruses to deliver necessary components could result in delays in our clinical development or marketing schedules.
  • Changes in methods of product candidate manufacturing or formulation may result in additional costs or delay.
  • Risks Related to the Commercialization of our Product Candidates
  • Even if any of our product candidates receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, third-party payors and others in the medical community necessary for commercial success.
  • Negative public opinion of gene therapy and increased regulatory scrutiny of gene therapy and genetic research may adversely impact the development or commercial success of our current and future product candidates.
  • If we are unable to establish sales, marketing and distribution capabilities for TSHA-102 or any other product candidate that may receive regulatory approval, we may not be successful in commercializing those product candidates if and when they are approved.
  • The affected populations for our other product candidates may be smaller than we or third parties currently project, which may affect the addressable markets for our product candidates.
  • We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
  • Biologic products may face competition sooner than anticipated.
  • The success of our product candidates will depend significantly on coverage and adequate reimbursement or the willingness of patients to pay for these procedures.
  • Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we may develop.
  • Risks Related to Our Dependence on Third Parties
  • We currently rely on our collaboration with UT Southwestern for our preclinical research and development programs, including for discovering, preclinically developing and conducting all IND-enabling studies for our lead product candidates and our near-term future pipeline. Failure or delay of UT Southwestern to fulfill all or part of its obligations to us under the agreement, a breakdown in collaboration between the parties or a complete or partial loss of this relationship would materially harm our business.
  • We intend to rely on third parties to conduct a significant portion of our existing clinical trials and potential future clinical trials for product candidates, and those third parties may not perform satisfactorily, including failing to meet deadlines for the completion of such trials.
  • We may seek collaborations with third parties for the development or commercialization of our product candidates. If those collaborations are not successful, we may not be able to capitalize on the market potential of these product candidates.
  • Risks Related to our Intellectual Property
  • If we are unable to obtain or protect intellectual property rights related to any of our product candidates, we may not be able to compete effectively in our market.
  • Patent terms may be inadequate to protect our competitive position on our products for an adequate amount of time, and if we do not obtain protection under the Hatch-Waxman Amendments and similar non-U.S. legislation for extending the term of patents covering each of our product candidates, our business may be materially harmed.
  • If we fail to comply with our obligations in our current and future intellectual property licenses with third parties, we could lose rights that are important to our business.
  • Patent reform legislation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our future patents.
  • We may be involved in lawsuits to protect or enforce our patents, which could be expensive, time-consuming and unsuccessful.
  • We may be unsuccessful in licensing or acquiring intellectual property from third parties that may be required to develop and commercialize our product candidates.
  • Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain.
  • We may be subject to claims that our employees, consultants, or independent contractors have wrongfully used or disclosed confidential information of third parties.
  • We may be subject to claims challenging the inventorship or ownership of our future patents and other intellectual property.
  • Reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
  • We may enjoy only limited geographical protection with respect to certain patents and we may not be able to protect our intellectual property rights throughout the world.
  • 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.
  • Any trademarks we have obtained or may obtain may be infringed or otherwise violated, or successfully challenged, resulting in harm to our business.
  • Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
  • Risks Related to Legal and Regulatory Compliance Matters
  • Our relationships with customers, healthcare providers, including physicians, and third-party payors are subject, directly or indirectly, to federal, state and foreign healthcare fraud and abuse laws, false claims laws, health information privacy and security laws, and other healthcare laws and regulations. If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties.
  • Even if we obtain regulatory approval for TSHA-102 or any future product candidates, they will remain subject to ongoing regulatory oversight.
  • Even if we obtain FDA or European Commission approval any of our product candidates in the United States or European Union, we may never obtain approval for or commercialize any of them in any other jurisdiction, which would limit our ability to realize their full market potential.
  • Healthcare legislative or regulatory reform measures may have a negative impact on our business and results of operations.
  • Disruptions at the FDA, the SEC and other government agencies and regulatory authorities caused by funding shortages or global health concerns could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
  • We are subject to legal proceedings and claims from time to time that may seek material damages or otherwise may have a material adverse effect on our business. The costs we incur in defending ourselves or associated with settling any of these proceedings, as well as a material final judgment or decree against us, could materially adversely affect our financial condition.
  • Risks Related to Employee Matters and Managing our Growth
  • Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel, and recent changes to our team might harm future operating results.
  • Our employees, independent contractors, consultants, collaborators, principal investigators, CROs, suppliers and vendors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements.
  • Risks Related to Ownership of our Common Stock
  • The trading price of the shares of our common stock may be volatile, and purchasers of our common stock could incur substantial losses.
  • If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline.
  • Sales of a substantial number of shares of our common stock in the public market could cause the market price of our common stock to drop significantly, even if our business is doing well.
  • An active trading market for our common stock may not be sustained.
  • Provisions in our corporate charter documents and under Delaware law may prevent or frustrate attempts by our stockholders to change our management and hinder efforts to acquire a controlling interest in us, and the market price of our common stock may be lower as a result.
  • Concentration of ownership of our common stock among our principal stockholders may prevent new investors from influencing significant corporate decisions.
  • Because we are no longer an "emerging growth company" as defined in the JOBS Act, we may incur additional expenses and devote increased management time to compliance with additional disclosures that are applicable to companies that are not emerging growth companies.

Legal proceedings

In January 2024 and April 2024, the Company was named a nominal defendant in two putative stockholder derivative actions filed by stockholders of the Company in the Court of Chancery of the State of Delaware. The lawsuits have since been consolidated and a lead plaintiff has been appointed. In October 2024, the lead plaintiff filed an amended complaint asserting claims relating to the Company’s August 2023 Private Placement against (i) certain of the Company’s current and former directors and officers for breach of fiduciary duty and unjust enrichment; and (ii) certain participants in the Company’s August 2023 Private Placement for aiding and abetting breach of fiduciary duty and unjust enrichment. The complaints seek an unspecified award of damages in the Company’s favor, plus pre-judgment and post-judgment interest, and an award to the plaintiffs for the costs and disbursement of the action, including fees for their attorneys and experts. The board of directors of the Company has formed a special litigation committee to investigate the claims and allegations in the amended complaint. On March 3, 2026, the special litigation committee moved to terminate the action, stating its conclusion that dismissal is in the best interest of the company and its stockholders. The Company has not recorded a liability related to these lawsuits because, at this time, the Company is unable to reasonably estimate possible losses or gains or determine whether an unfavorable outcome is either probable or remote.

The opening of Item 3, Legal Proceedings.3

Sources

The numbers on this page point here. Each one links to the record it came from.

  1. 1
  2. 2
  3. 3
  4. 4
  5. 5
  6. 6
  7. 7
  8. 8
  9. 9
  10. 10
  11. 11
  12. 12
  13. 13
  14. 14
  15. 15
  16. 16
  17. 17
  18. 18
  19. 19
  20. 20
  21. 21
  22. 22
  23. 23
  24. 24
  25. 25
  26. 26
  27. 27
  28. 28
  29. 29
  30. 30
  31. 31
  32. 32
  33. 33
    SEC EDGAROther events
  34. 34
    SEC EDGAROther events
  35. 35
    SEC EDGAROther events
  36. 36
  37. 37
  38. 38
  39. 39
  40. 40
  41. 41
  42. 42
  43. 43
  44. 44
  45. 45
  46. 46
  47. 47
  48. 48
  49. 49
  50. 50
  51. 51
  52. 52
  53. 53
  54. 54
  55. 55
  56. 56