Solid Biosciences Provides 2026 Outlook Underscoring Neuromuscular and Cardiac Pipeline Momentum and Expanded Access to Next-Generation Capsid AAV-SLB101

- Duchenne: Dosed 33 participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of January 9, 2026; SGT-003 continues to be generally well tolerated using a steroid-only prophylactic immunomodulation regimen -

- Duchenne: First participant enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected in Q1 2026 -

- FA: First participant dosed in Phase 1b FALCON clinical trial; FDA Orphan Drug designation granted to SGT-212, the only dual route of administration gene therapy in development to treat Friedreich's ataxia (FA) -

- CPVT: Clinical trial sites activated for ARTEMIS, a Phase 1b first-in-human clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT); participant screening is underway -

- Capsids (AAV-SLB101): Executed 50+ agreements, including licenses, with corporations, institutions and academic labs for the use of proprietary, next-generation capsid AAV-SLB101 -

CHARLESTOWN, Mass., Jan. 13, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will provide a corporate update outlining progress in advancing its neuromuscular and cardiac gene therapy programs in a presentation delivered by Bo Cumbo, President and CEO, at the 44^th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 5:15 p.m. PT (8:15 p.m. ET).

"Over the past year, we have executed across our pipeline, building critical momentum as we enter 2026 with four active clinical trials in three devastating neuromuscular and cardiac rare diseases with significant unmet need, including the recently announced dosing of the first participant in our Phase 1b FALCON trial evaluating SGT-212 for the treatment of FA," said Mr. Cumbo. "By rapidly and responsibly advancing innovative science and forging collaborations to enhance gene therapy delivery using our next-generation capsid, we are determined to build a better future for the patient communities we serve while driving value for our shareholders. With multiple regulatory interactions planned in the coming months, we aim to achieve alignment on a potential accelerated approval pathway for SGT-003 and to bring our investigational therapy to market to meet the overwhelming demand from the Duchenne community."

Highlights from the presentation to be given at the J.P. Morgan Healthcare Conference include:

Neuromuscular Pipeline

SGT-003 for Duchenne muscular dystrophy (Duchenne)



U.S.:

• As of a safety cutoff date of January 9, 2026, SGT-003 has been generally well tolerated in the 33 participants dosed in the ongoing INSPIRE DUCHENNE Phase 1/2 clinical trial.
  • SGT-003's safety and tolerability profile continues to support a steroid-only prophylactic immunomodulation regimen.
  • Outpatient dosing has been enabled since September 2025.
  • No drug-induced liver injury (DILI), thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS) or myocarditis observed as of the January 9, 2026, cutoff date.
  • Cardiac safety monitoring has shown reductions in cardiac injury and early signals of cardiac systolic function normalization, as measured by left ventricular ejection fraction (LVEF) (N=14, data cutoff of September 29, 2025).
• Compelling microdystrophin expression levels (N=10, data cutoff of September 29, 2025) and concordant restoration of key components of the dystrophin-associated protein complex (DAPC) have been observed.
• Improvements across a range of biomarkers of muscle integrity have been observed suggesting a coordinated downstream effect (N=11-14, data cutoff of September 29, 2025).
• During the first half of 2026, the Company plans to have multiple interactions with the U.S. Food and Drug Administration (FDA) to align on its Phase 3 confirmatory trial design and on necessary confirmatory evidence required to support potential accelerated approval for SGT-003, with an update expected mid-year 2026.
• The Company expects to report additional data from the Phase 1/2 INSPIRE DUCHENNE trial in mid-2026.
  
  
  
  

Ex-U.S.:

• The first participant has been enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled, ex-U.S. clinical trial, with dosing expected to occur in the first quarter of 2026.
• The IMPACT DUCHENNE trial has two active clinical trial sites, located in Canada and Australia, with planned expansion into additional countries, including in Europe, beginning in mid-year 2026, subject to the receipt of regulatory approvals.
• SGT-003 has been awarded the Innovation Passport Designation under the new U.K. Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market and facilitate patient access to new medicines, positioning SGT-003 to potentially become the first-to-market Duchenne gene therapy in the U.K.
  
  
  
  

SGT-212 for Friedreich's ataxia (FA)

• The first participant has been dosed in FALCON, a Phase 1b first-in-human clinical trial evaluating SGT-212, Solid's investigational gene therapy for the treatment of FA.
  • Intra-procedural MRI-imaging demonstrated promising intradentate nuclei (IDN) targeting and coverage.
  • SGT-212 is the first investigational gene therapy for FA to utilize a dual route of administration and is intended to promote restoration of therapeutic levels of the frataxin protein to address the neurologic, cardiac and systemic clinical manifestations of FA.
  • SGT-212 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA.
• The Company anticipates reporting initial data from the FALCON trial in the second half of 2026, subject to participant enrollment.
  
  
  
  

Cardiac Pipeline

SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT)

• Clinical trial sites have been activated with participant screening underway for ARTEMIS, a first-in-human, open-label, Phase 1b clinical trial to evaluate the safety, tolerability and efficacy of SGT-501, Solid's investigational gene therapy for the treatment of CPVT.
• SGT-501 is a novel, investigational gene therapy intended to achieve augmentation of CASQ2 protein levels to address the underlying ryanodine receptor (RYR2) instability and calcium dysregulation seen in CPVT. There are currently no FDA-approved treatments that address the underlying mechanisms of CPVT.
• The Company anticipates reporting initial safety data from the ARTEMIS trial in the second half of 2026, subject to participant enrollment.
  
  
  
  

Platform Technologies – Capsids

Solid continues to broaden access to its proprietary next-generation capsid, AAV-SLB101, designed with the goal of enhancing skeletal muscle and cardiac tropism with reduced biodistribution to the liver.

• Solid has executed 50+ agreements, including licenses, with corporations, institutions, and academic labs for the use of AAV-SLB101.
• Used in SGT-003, AAV-SLB101 has been generally well tolerated in the 33 participants dosed in the INSPIRE DUCHENNE trial as of January 9, 2026, and has shown compelling levels of vector transduction, protein expression (N=10, data cutoff of September 29, 2025), and reduced liver impact (N=14, data cutoff of September 29, 2025).

44^th Annual J.P. Morgan Healthcare Conference Webcast

Mr. Cumbo will present at the 44^th Annual J.P. Morgan Healthcare Conference today at 5:15 p.m. PT (8:15 p.m. ET). A live webcast of the presentation will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page.

About Solid Biosciences

Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich's ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic neuromuscular and cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid's mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company's goals, priorities and key clinical and preclinical milestones; strategies and expectations for the company's SGT-003, SGT-212 and SGT-501 programs; expectations for additional site activations, planned enrollment, planned data announcements, planned regulatory interactions and the potential approval pathways for SGT-003; plans for data announcements for the clinical trial of SGT-212; timing of planned enrollment and data announcements for the clinical trial SGT-501; and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," "working" and similar expressions. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company's ability to advance SGT-003, SGT-212, SGT-501, SGT-601 and other preclinical programs, capsid libraries and other enabling technologies on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company's product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; enroll patients in ongoing trials; activate clinical trial sites; replicate preliminary or interim data from clinicals trials in the final data of such trials; compete successfully with other companies that are seeking to develop Duchenne, FA, CPVT and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-212, SGT-501, SGT-601 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof and should not be relied upon as representing the company's views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.

Solid Biosciences Investor Contact:

Nicole Anderson

Director, Investor Relations and Corporate Communications

Solid Biosciences Inc.

[email protected]

Media Contact:

Glenn Silver

FINN Partners

[email protected]