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SLDB on Nasdaq

Solid Biosciences Inc.

Solid Biosciences Inc. is an American biological products company based in Charlestown.1 Its shares trade on Nasdaq under the symbol SLDB.1

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Last updated Oct 21, 2026.

Overview

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  • TickerSLDB on Nasdaq1
  • IndustryBiological Products, (No Diagnostic Substances)1
  • HeadquartersCharlestown, Massachusetts1
  • Incorporated inDE2
  • Business address500 Rutherford Avenue, 3Rd Floor, Charlestown, Ma 021292
  • Filer categoryNon-accelerated filer, Smaller reporting company2
  • Legal formCorporation

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We are a life sciences company focused on advancing a portfolio of current and future gene therapy candidates, which we refer to collectively as our Candidates, including SGT-003 for the treatment of Duchenne muscular dystrophy (“Duchenne”), SGT-212 for the treatment of Friedreich’s ataxia (“FA”), SGT-501 for the treatment of Catecholaminergic polymorphic ventricular tachycardia (“CPVT”), SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy (“TNNT2 DCM”), and additional assets for the treatment of genetic cardiac and neuromuscular diseases, at different stages of development, with varying levels of investment. We are advancing our diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management and care. Patient-focused and founded by those directly impacted by Duchenne, our mission is to improve the daily…4 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 2022

-1315.2%Operating margin5
-1062.3%Net margin5
-40.6%Revenue growth, 20226
-40.6%Return on equity7
-$101.0MFree cash flow5

Where the money goes

Where each dollar of revenue went, fiscal 2022

  • Research and development100¢$78.4M

Out of $8.1M in revenue.65 The company lost money this year.

Financial statements

Revenue86$8.1M in 2022
0$5.0M$10.0M$15.0M2019: $0$020192020: $0$020202021: $13.6M$13.6M20212022: $8.1M$8.1M2022−41%
Operating income865-$106.5M in 2022−11% since 2019
-$150.0M-$100.0M-$50.0M02019: -$119.3M-$119.3M20192020: -$88.4M-$88.4M2020−26%2021: -$72.3M-$72.3M2021−18%2022: -$106.5M-$106.5M2022+47%
Net income965-$86.0M in 2022−27% since 2019
-$150.0M-$100.0M-$50.0M02019: -$117.2M-$117.2M20192020: -$88.3M-$88.3M2020−25%2021: -$72.2M-$72.2M2021−18%2022: -$86.0M-$86.0M2022+19%

Every line of the income statement as filed: the last 2 fiscal years, latest first.12

Period ending2025-12-312024-12-31
Research and development$140.3M$96.4M
General and administrative$38.9M$33.3M
Total operating expenses$179.2M$129.7M
Loss from operations-$179.2M-$129.7M
Interest income$9.9M$9.5M
Interest expense$203.0K-$340.0K
Change in fair value of derivative liabilities-$6.0M-$4.8M
Other income, net$824.0K$652.0K
Total other income, net$4.9M$5.0M
Net loss-$174.3M-$124.7M
Net loss per share, basic-$1.99-$3.06
Net loss per share, diluted-$1.99-$3.06
Weighted average shares of common stock outstanding used to compute basic net loss per share87.5M shares40.8M shares
Weighted average shares of common stock outstanding, used to compute diluted net loss per share87.5M shares40.8M shares

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Corporate structure

Solid Biosciences Inc. sits at the top of its group. 3 entities, 1 country on record. Switch between the flow chart and the network, and select any entity to see its details.

Major holders of Solid Biosciences Inc.

  • PERCEPTIVE ADVISORS LLC Filed SCHEDULE 13D/A on Solid Biosciences Inc., latest Aug 14, 2026 (8 filings since Sep 2, 2022)
  • Vestal Point Capital, LP Filed SCHEDULE 13G/A on Solid Biosciences Inc., latest Aug 14, 2026 (4 filings since Nov 14, 2024)
  • FMR LLC Filed SCHEDULE 13G on Solid Biosciences Inc., latest Aug 6, 2026
  • BlackRock Filed SCHEDULE 13G on Solid Biosciences Inc., latest Jul 30, 2026 (5 filings since Feb 4, 2022)
  • RA CAPITAL MANAGEMENT, L.P. Filed SCHEDULE 13D/A on Solid Biosciences Inc., latest Jun 24, 2026 (10 filings since Sep 6, 2022)
  • Invus Global Management, LLC Filed SCHEDULE 13G/A on Solid Biosciences Inc., latest May 5, 2026 (5 filings since Aug 2, 2024)
  • BCLS SB Investco, LP Filed SCHEDULE 13D/A on Solid Biosciences Inc., latest Mar 10, 2026 (8 filings since Aug 18, 2022)
  • Adage Capital Management, L.P. Filed SCHEDULE 13G/A on Solid Biosciences Inc., latest Feb 12, 2026 (4 filings since Nov 12, 2024)
  • Commodore Capital LP Filed SCHEDULE 13G/A on Solid Biosciences Inc., latest Aug 14, 2025 (2 filings since Feb 25, 2025)
  • Camber Capital Management LP Filed SCHEDULE 13G/A on Solid Biosciences Inc., latest Feb 14, 2025 (2 filings since Dec 12, 2022)
  • James E. Flynn Filed SC 13G/A on Solid Biosciences Inc., latest Nov 14, 2024 (2 filings since Jan 12, 2024)
  • MILLENNIUM MANAGEMENT LLC Filed SC 13G/A on Solid Biosciences Inc., latest Nov 1, 2024 (2 filings since Jun 5, 2024)
  • Solid Biosciences Inc. Charlestown, Massachusetts
    • AavantiBio, Inc. Subsidiary · Delaware, United States
    • Solid Biosciences 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 Solid Biosciences 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$18.4M12 Dec 31, 2025$17.8M12 Dec 31, 2024+3.3%
Accumulated depreciation$14.2M12 Dec 31, 2025$13.0M12 Dec 31, 2024+8.9%
Property and equipment, net$4.2M12 Dec 31, 2025$4.7M12 Dec 31, 2024−12.2%
Operating-lease assets (leased space)$21.9M12 Dec 31, 2025$24.3M12 Dec 31, 2024−9.8%

Investments

ItemLatestA year beforeChange
Cash and cash equivalents$59.9M12 Dec 31, 2025$80.2M12 Dec 31, 2024−25.3%
Marketable securities, current$128.0M12 Dec 31, 2025$68.7M12 Dec 31, 2024+86.3%
Debt securities available for sale$128.0M12 Dec 31, 2025$68.7M12 Dec 31, 2024+86.3%

Properties, in its own words

We lease our corporate headquarters, which consists of approximately 49,869 square feet of office, laboratory, research and development and manufacturing space in Charlestown, Massachusetts. The lease for our corporate headquarters has an initial term of approximately ten years that expires in 2032 with an option to extend the lease for an additional five years. In addition, we lease smaller laboratory and office space in North Carolina.

Item 2 of the 10-K filed Mar 19, 2026.4

Leadership

Who has led Solid Biosciences Inc., and when. Pick a name to see everywhere that person has worked.

Chief executive

  1. Ilan Ganot2018–2022
  2. Alexander Cumbo2022–now

President

  1. Gilad David Hayeem2018
  2. Ilan Ganot2018–2022
  3. Alexander Cumbo2022–now

Chief financial officer

  1. Jennifer Lynn Ziolkowski2019–2021
  2. Stephen J. Dipalma2021
  3. Kevin Tan2023–now

Chief operating officer

  1. Pedro Alvaro Amorrortu2018–2019
  2. Joel Solomon Zev Schneider2021–2022
  3. David T. Howton2023–now
20202025
Board members and other officers (29)

Named in SEC Form D filings

People the company listed in its private-offering notices.

  • Executive OfficerPaul Tyronne Herizch named Dec 9, 202250

Subsidiaries and ownership

Subsidiaries (Exhibit 21)

  • AavantiBio, Inc. Delaware3
  • Solid Biosciences Securities Corporation Massachusetts3

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

Former names

  • Solid Biosciences, LLCAug 4, 2017 to Jan 25, 20182

Timeline

  1. Aug 6, 20268-K Other events51
  2. Jun 10, 20268-K Shareholder vote52
  3. Apr 10, 20268-K Executive or director change53
  4. Mar 12, 20268-K Regulation FD disclosure; Other events54
  5. Mar 6, 20268-K Entered a material agreement; Results of operations; Unregistered sale of shares55
  6. Nov 3, 20258-K Results of operations; Regulation FD disclosure; Other events56
  7. Jun 13, 20258-K Executive or director change; Shareholder vote57
  8. May 15, 20258-K Other events58
  9. Feb 18, 20258-K Results of operations; Other events59
  10. Feb 18, 20258-K Other events60
  11. Feb 18, 20258-K Entered a material agreement; Other events61
  12. Jun 11, 20248-K Executive or director change; Shareholder vote62
  13. Dec 5, 2022New chief executive Alexander Cumbo becomes CEO14
  14. Jan 25, 2018New chief executive Ilan Ganot becomes CEO15

Calendar

Coming up

  1. Nov 2, 2026Reports results for the quarter ending Sep/2026 A year earlier it reported ($0.48) a share, on Nov 3, 2025.63

When it reported results

  1. Mar 6, 2026Reported quarterly or annual results55
  2. Nov 3, 2025Reported quarterly or annual results56
  3. Feb 18, 2025Reported quarterly or annual results59
  4. Jan 8, 2024Reported quarterly or annual results64
  5. Jan 9, 2023Reported quarterly or annual results65
  6. Aug 11, 2022Reported quarterly or annual results66
  7. Apr 27, 2022Reported quarterly or annual results67
  8. Jan 10, 2022Reported quarterly or annual results68

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

Filings

428 filings to the SEC since October 2021: 5 annual reports, 15 quarterly reports, 30 major events, 213 insider forms, 62 large holders, 1 private raises, 102 other. The latest 20:

  1. Oct 6, 20264 EDGAR
  2. Aug 14, 2026SCHEDULE 13D/A EDGAR
  3. Aug 14, 2026SCHEDULE 13G/A EDGAR
  4. Aug 6, 20268-K EDGAR
  5. Aug 6, 2026424B5 EDGAR
  6. Aug 6, 2026S-3ASR EDGAR
  7. Aug 6, 202610-Q EDGAR
  8. Aug 6, 2026SCHEDULE 13G EDGAR
  9. Jul 30, 2026SCHEDULE 13G EDGAR
  10. Jul 7, 20264 EDGAR
  11. Jun 24, 2026SCHEDULE 13D/A EDGAR
  12. Jun 24, 20263 EDGAR
  13. Jun 11, 20264 EDGAR
  14. Jun 11, 20264 EDGAR
  15. Jun 11, 20264 EDGAR
  16. Jun 11, 20264 EDGAR
  17. Jun 11, 20264 EDGAR
  18. Jun 11, 20264 EDGAR
  19. Jun 11, 20264 EDGAR
  20. Jun 11, 20264 EDGAR

The 428 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

66 open-market trades by officers, directors and large owners since October 2021: 7 buys and 59 sales. Grants, option exercises and gifts are left out.

DateWhoTradeSharesPriceValueFiling
Feb 18, 2026Cumbo AlexanderPresident and CEOSale16,644$5.82$96.9KForm 4
Feb 18, 2026Hanrahan JessieChief Regulatory OfficerSale4,134$5.82$24.1KForm 4
Feb 18, 2026Ganot IlanDirectorSale2,658$5.82$15.5KForm 4
Feb 18, 2026Herzich PaulChief Technology OfficerSale3,616$5.82$21.0KForm 4
Feb 18, 2026Howton David TChief Operating OfficerSale7,469$5.82$43.5KForm 4
Feb 18, 2026Brooks GabrielChief Medical OfficerSale0Form 4
Feb 18, 2026Tan KevinCFO & TreasurerSale5,404$5.82$31.5KForm 4
Feb 4, 2026Howton David TChief Operating OfficerSale18,894$6.44$121.7KForm 4
Feb 4, 2026Cumbo AlexanderPresident and CEOSale48,913$6.44$315.0KForm 4
Feb 4, 2026Ganot IlanDirectorSale7,205$6.44$46.4KForm 4
Feb 4, 2026Tan KevinCFO & TreasurerSale14,783$6.44$95.2KForm 4
Feb 4, 2026Brooks GabrielChief Medical OfficerSale12,616$6.44$81.2KForm 4
Feb 4, 2026Hanrahan JessieChief Regulatory OfficerSale12,348$6.44$79.5KForm 4
Feb 4, 2026Herzich PaulChief Technology OfficerSale10,905$6.44$70.2KForm 4
Feb 2, 2026Tan KevinCFO & TreasurerSale26,837$6.44$172.8KForm 4
Feb 2, 2026Cumbo AlexanderPresident and CEOSale80,258$6.44$516.9KForm 4
Feb 2, 2026Hanrahan JessieChief Regulatory OfficerSale26,535$6.44$170.9KForm 4
Feb 2, 2026Brooks GabrielChief Medical OfficerSale28,335$6.44$182.5KForm 4
Feb 2, 2026Howton David TChief Operating OfficerSale37,771$6.44$243.2KForm 4
Feb 2, 2026Herzich PaulChief Technology OfficerSale26,250$6.44$169.1KForm 4

All 66 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
2025Alexander Cumbo,55$651.0K$5.2M87
2025David Tyronne Howton,54$513.4K$2.6M87
2025Gabriel BrooksM.D.,49$502.2K$2.3M87
2024President and Chief Executive Officer$608.4K$4.9M87
2024Chief Operating Officer and Secretary$473.2K$2.6M87
2024Chief Medical Officer$468.0K$2.1M87

Money raised

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

  1. Dec 2, 2022Raised $9.2 million in a private offering of equity Form D: 13 investors 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
Ra Capital Management, L.P.12,036,126$120.2M1.0%+2,696,148
Perceptive Advisors LLC11,906,646$118.9M1.9%No change
Bain Capital Life Sciences Investors, LLC9,808,819$98.0M5.8%No change
Siren, L.L.C.7,690,405$76.8M1.6%No change
BlackRock, Inc.6,934,248$69.3M0.0%+1,975,894
Fmr LLC6,875,957$68.7M0.0%+3,269,553
Armistice Capital, LLC4,278,000$42.7M1.2%+1,078,000
State Street Corp4,175,365$41.7M0.0%+3,049,878
Jupiter Topco LLC4,043,605$40.4M0.0%New
Vanguard Capital Management LLC4,009,922$40.1M0.0%+1,335,298
Rtw Investments, LP3,892,926$38.9M0.3%New
Deep Track Capital, LP3,565,062$35.6M0.5%No change
Franklin Resources Inc3,440,708$34.4M0.0%+1,247,771
Citadel Advisors LLC2,815,122$28.1M0.0%+218,322
Alyeska Investment Group, L.P.2,280,484$22.8M0.1%+359
Ameriprise Financial Inc2,225,783$22.2M0.0%−213,066
Vestal Point Capital, LP2,160,000$21.6M0.5%−2,840,000
Redmile Group, LLC1,948,695$19.5M1.3%−63,200
Geode Capital Management, LLC1,940,824$19.4M0.0%+644,840
Goldman Sachs Group Inc1,602,917$16.0M0.0%+667,382
Adage Capital Partners Gp, L.l.c.1,506,506$15.0M0.0%−250,000
Dimensional Fund Advisors LP1,266,290$12.6M0.0%+310,155
Blair William & Co/il756,277$7.6M0.0%+105,061
Vanguard Portfolio Management LLC748,589$7.5M0.0%+409,959
Woodline Partners LP674,076$6.7M0.0%+179,000

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

SEC filer profile

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

  • Name on EDGARSolid Biosciences Inc.1
  • CIK0001707502
  • Employer ID (EIN)90-09434024
  • Filer statusNon-accelerated filer not an emerging growth company; a smaller reporting company; not a shell company4
  • Incorporated inDelaware4
  • 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 address500 Rutherford Avenue, 3Rd Floor, Charlestown, MA 021291
  • Phone617-337-46801
  • Shares outstanding107,225,238 as of Aug 4, 202611
  • Public float$317.7M as of Jun 30, 20254
  • AuditorPricewaterhouseCoopers LLP, Boston, Massachusetts PCAOB ID 238; no auditor attestation of internal control over financial reporting4
  • On EDGAR sinceAug 4, 20171
  • Filings734 in all: 4 (335), 8-K (66), 3 (39), 144 (29), 10-Q (26), SC 13D/A (24), SC 13G/A (20), CORRESP (17)1
    All 47 form types

    SC 13G (17), EFFECT (13), S-8 (13), UPLOAD (13), S-3 (12), DEFA14A (11), SCHEDULE 13D/A (10), SCHEDULE 13G/A (10), 10-K (9), DEF 14A (8), 4/A (6), 424B5 (6), SCHEDULE 13G (6), ARS (4), PRE 14A (4), SC 13D (4), D (3), S-1/A (3), 8-K/A (2), DRSLTR (2), FWP (2), S-3/A (2), SEC STAFF LETTER (2), 10-K/A (1), 144/A (1), 3/A (1), 424B3 (1), 424B4 (1), 424B7 (1), 8-A12B (1), CERT (1), CT ORDER (1), DEFM14A (1), DRS (1), DRS/A (1), PREM14A (1), S-1 (1), S-1MEF (1), S-3ASR (1)

  • Latest filingsAnnual report (10-K) Mar 19, 2026
    Quarterly report (10-Q) Aug 6, 2026
    Proxy statement (DEF 14A) Apr 23, 2026
    Latest 8-K (8-K) Aug 6, 2026

Securities registered for trading

ClassSymbolExchange
Common Stock $0.001 par value per shareSLDBThe Nasdaq Global Select Market

From the cover page of the 10-K filed Mar 19, 2026 (Section 12(b) of the Exchange Act).4

Risk factors and legal proceedings

The risks Solid Biosciences Inc. names in Item 1A of its 10-K filed Mar 19, 2026: 75 risks under these headings, as written.4

  • Risks Related to our Financial Position and Need for Capital Requirements
  • We have incurred significant net losses since inception and anticipate that we will continue to incur net losses for the foreseeable future and may never achieve or maintain profitability.
  • We will need 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 our product development efforts or other operations.
  • Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights to our technologies or Candidates.
  • We have never generated revenue from product sales and do not expect to do so for the foreseeable future, if ever.
  • Our limited operating history may make it difficult for our stockholders to evaluate the success of our business to date and to assess our future viability.
  • Unfavorable global economic conditions could harm our business, financial condition or results of operations.
  • We hold a portion of our cash and cash equivalents that we use to meet our working capital and operating expense needs in deposit accounts that could be adversely affected if the financial institutions holding such funds fail.
  • Risks Related to the Development of our Candidates
  • Our pipeline of gene transfer Candidates utilize novel technology, which makes it difficult to predict the time and cost of development and of subsequently obtaining regulatory approval. To our knowledge, only a limited number of gene transfer products have been approved for commercialization in the United States and the European Union.
  • Our Candidates may cause undesirable side effects or have other properties that could delay or prevent their clinical development, regulatory approval, limit their commercial potential or result in significant negative consequences following any potential marketing approval.
  • One of our prior clinical trials had been placed on clinical hold by the FDA in the past, and we cannot guarantee that similar events will not happen in ongoing, planned and future clinical trials for our Candidates.
The other 65 risks
  • We have never completed a clinical trial and may be unable to do so for any Candidate, including SGT-003, SGT-212, SGT-501 and other Candidates.
  • Success in preclinical studies or early clinical trials may not be indicative of results obtained in later trials.
  • Preliminary or interim data that we announce or publish from time to time may change as more data becomes available and are subject to audit and verification procedures that could result in material changes in the final data.
  • We may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
  • If our third-party clinical trial vendors fail to comply with strict regulations, any clinical trials for our Candidates may be delayed or unsuccessful.
  • We may find it difficult to enroll participants in our clinical trials, which could delay or prevent us from proceeding with clinical trials of SGT-003, SGT-212, SGT-501 or our other Candidates.
  • We plan to conduct clinical trials at sites outside the United States. The FDA may not accept data from trials conducted in such locations, and the conduct of trials outside the United States could subject us to additional delays and expense.
  • Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize our Candidates and the approval may be for a narrower indication than we seek.
  • Even if we obtain regulatory approval for a Candidate, our Candidates will remain subject to regulatory oversight.
  • Any regulatory approval to market our products will be limited by indication. If we fail to comply or are found to be in violation of FDA regulations restricting the promotion of our products for unapproved uses, we could be subject to criminal penalties, substantial fines or other sanctions and damage awards.
  • Even if we obtain and maintain approval of one or more of our Candidates from the FDA, we may never obtain approval for our Candidates outside of the United States, which would limit our market opportunities and adversely affect our business.
  • Changes in and uncertainty surrounding U.S. and international trade policies, particularly with respect to China, may adversely impact our business and operating results.
  • Regulatory requirements governing gene therapy products are periodically updated and may continue to change in the future.
  • We may not be able to obtain orphan drug exclusivity for one or more of our Candidates, and even if we do, that exclusivity may not prevent the FDA or the EMA from approving other competing products.
  • We may seek a breakthrough therapy designation for one or more of our Candidates, but we might not receive such designation, and even if we do, such designation may not lead to a faster development or regulatory review or approval process.
  • Accelerated approval by the FDA, even if granted for one or more of our Candidates, may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that our Candidates will receive marketing approval.
  • A potential regenerative medicine advanced therapy designation by the FDA for our Candidates may not lead to a faster development or regulatory review or approval process, and it does not increase the likelihood that our Candidates will receive marketing approval.
  • We may seek PRIME Designation in the EU for one or more of our Candidates, but we might not receive such designations and, even if we do, such designations may not lead to a faster development or regulatory review or approval process.
  • We may seek a Rare Pediatric Disease Designation for our Candidates. However, a BLA for such Candidates may not meet the eligibility criteria for a priority review voucher upon approval.
  • We may seek a fast track designation for one or more of our Candidates. However, such designation may not actually lead to a faster development or regulatory review or approval process. We might not receive such designation for one or more of our Candidates.
  • We may seek priority review designation for one or more of our Candidates, but we might not receive such designation, and even if we do, such designation may not lead to a faster development or regulatory review or approval process.
  • Disruptions and delays 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 program and develop and secure approval of our Candidates in a timely manner, which would negatively impact our business.
  • We face significant competition and our competitors may achieve regulatory approval before us or develop therapies that are more advanced or effective than ours, which may adversely affect our ability to develop, successfully market or commercialize our Candidates. Changes within the competitive landscape could lead us to alter our regulatory and/or clinical trial strategy, baseline eligibility criteria or make other modifications to clinical trial designs.
  • We may fail to capitalize on other potential Candidates that may represent a greater commercial opportunity or for which there is a greater likelihood of success.
  • Risks Related to the Manufacturing and Commercialization of our Candidates
  • We have entered into, and may in the future enter into, collaborations with third parties for the development or commercialization of our Candidates. If our collaborations are not successful, we may not be able to capitalize on the market potential of these Candidates and our business could be adversely affected.
  • We may not be successful in finding strategic collaborators for continuing development of our Candidates or platform technologies, or for successfully commercializing or competing in the market for certain indications.
  • We have limited gene therapy manufacturing experience and could experience production problems and delays in obtaining regulatory approval of our manufacturing processes, which could result in delays in the development or commercialization of SGT-003, SGT-212, SGT-501, or our other Candidates. In addition, changes to manufacturing sites or processes, or formulations for our Candidates may result in additional cost or delay.
  • We expect to utilize third parties to conduct our product manufacturing for the foreseeable future. Therefore, we are subject to the risk that these third parties may not perform satisfactorily or meet regulatory requirements.
  • If we are unable to establish sales, distribution and marketing capabilities or enter into agreements with third parties to market and sell our Candidates, we will be unable to generate any product revenue.
  • If we are unable to establish medical affairs capabilities, we will be unable to establish an educated market of physicians to administer any future products.
  • If the market opportunities for any of our future products are smaller than we believe they are, our revenue prospects may be adversely affected and our business may suffer.
  • Certain patients’ immune systems might prohibit the successful delivery of certain gene therapy products, thereby potentially limiting the population of patients amenable to gene transfer.
  • The commercial success of any of our Candidates, if approved, will depend upon market acceptance by physicians, patients, third-party payors and others in the medical community.
  • Our gene transfer approach utilizes capsids derived from a virus, which may be perceived as unsafe or may result in unforeseen adverse events. Negative public opinion and increased regulatory scrutiny of gene therapy may damage public perception of the safety of gene transfer Candidates and adversely affect our ability to conduct our business or obtain regulatory approvals for our Candidates.
  • Any contamination in our manufacturing process, shortages of materials or failure of any of our key suppliers to deliver necessary components could result in interruption in the supply of our Candidates and delays in our clinical development or commercialization schedules.
  • The insurance coverage and reimbursement status of newly approved products is uncertain. Failure to obtain or maintain adequate coverage and reimbursement for our Candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue.
  • Governments outside the United States tend to impose strict price controls, which may adversely affect our revenue, if any.
  • If we obtain approval to commercialize our future products outside of the United States, in particular in the European Union, a variety of risks associated with international operations could materially adversely affect our business.
  • If we engage in future acquisitions or strategic collaborations, this may increase our capital requirements, dilute our stockholders, cause us to incur debt or assume contingent liabilities and subject us to other risks.
  • Risks Related to our Business Operations
  • Our future success depends on our ability to retain key employees, consultants and advisors and to attract, retain and motivate qualified personnel.
  • If we are unable to manage growth in the scale and complexity of our operations, our performance may suffer.
  • Our employees may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements, which could cause significant liability for us and harm our reputation.
  • Enacted and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our Candidates and may affect the prices we may set.
  • Changes in tax laws or in their implementation or interpretation could adversely affect our business and financial condition.
  • Current and future legislative efforts may limit the prices for our products, if and when they are licensed for marketing and that could materially impact our ability to generate revenues.
  • Our relationships with customers, physicians and third-party payors will be subject, directly or indirectly, to federal and state health care fraud and abuse laws, false claims laws, health information privacy and security laws, and other health care laws and regulations. If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties.
  • 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, contractual obligations and 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.
  • We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws and other laws governing our operations. If we fail to comply with these laws, we could be subject to civil or criminal penalties, other remedial measures and legal expenses, be precluded from developing manufacturing and selling certain products outside the United States or be required to develop and implement costly compliance programs, which could adversely affect our business, results of operations and financial condition.
  • Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any Candidate that we may develop.
  • If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
  • Our internal computer systems, or those of our collaborators, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development.
  • Risks Related to our Intellectual Property
  • We heavily rely on certain in-licensed patents and other intellectual property rights in connection with our development of our Candidates and may be required to acquire or license additional patents or other intellectual property rights to continue to develop and commercialize our Candidates.
  • If we are unable to obtain and maintain patent protection for our Candidates, or if the scope of the patent protection obtained is not sufficiently broad, our competitors could develop and commercialize products similar or identical to ours, and our ability to successfully commercialize our Candidates may be adversely affected.
  • We may not be able to file, prosecute, maintain, enforce, defend or license all patents that are necessary to our business.
  • Our licensed patents, and any patents we may own in the future, may be challenged, narrowed, invalidated or held unenforceable.
  • Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors.
  • If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
  • Third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business.
  • Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
  • 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.
  • Some intellectual property that we have in-licensed 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. manufacturing. Compliance with such regulations may limit our exclusive rights, and limit our ability to contract with non-U.S. manufacturers.
  • We may not be able to protect our intellectual property and proprietary rights throughout the world.
  • If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
  • We may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets 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 Candidates.

Sources

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