Solid Biosciences to Present at 2025 Neuromuscular Study Group Annual Scientific Meeting
CHARLESTOWN, Mass., Sept. 25, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation investigational gene therapy intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 26th Annual Scientific Meeting of Neuromuscular Study Group (NMSG), September 26-28, 2025, in Stresa, Italy. Presentations will include previously reported Day 90 biopsy data in addition to updated safety data as of the August 12, 2025, data cutoff from the INSPIRE DUCHENNE trial.
"Our presentations at the NMSG Annual Meeting highlight the emerging, differentiated profile of SGT-003 – particularly its favorable liver tolerability and encouraging early biomarker signals – with an immunomodulation regimen consisting of only steroids," said Bo Cumbo, President and CEO of Solid Biosciences. "The data presentations also reinforce the potential of AAV-SLB101 as a next-generation capsid for targeted gene therapy in neuromuscular and cardiac indications. To date, we have established license agreements with more than 25 companies, institutions and academic labs for the use of AAV-SLB101 and look forward to further expanding access to additional organizations in the coming months."
The following abstracts will be presented during the poster session on Friday, September 26, from 6 to 8 p.m. CEST:
1. Update on INSPIRE DUCHENNE: A Phase 1/2 Study of SGT-003, an Investigational Next-Generation Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Presenter: Perry Shieh, MD, PhD, Professor, Neurology and Pediatrics, David Geffen School of Medicine at UCLA and Principal Investigator in the INSPIRE DUCHENNE clinical trial
2. SGT-003: Initial Safety Evaluation of a Next-Generation Investigational Gene Therapy for Duchenne Muscular Dystrophy
Presenter: Patrick Gonzalez, PhD, Head of Clinical Science, Solid Biosciences
3. AAV-SLB101 in Duchenne Muscular Dystrophy: Nonclinical Safety, Characterization of Efficacy and Preliminary Clinical Insights
Presenter: Patrick Gonzalez, PhD, Head of Clinical Science, Solid Biosciences
Following completion of the conference, presentations will be available on the Scientific Publications & Presentations page of the Our Science section of the Company website, or by clicking here.
About Duchenne
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.
About SGT-003
SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies. SGT-003's microdystrophin construct uniquely includes the R16/17 domains, which localize nNOS to the muscle. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne.
About INSPIRE DUCHENNE
INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric participants with a genetically confirmed Duchenne diagnosis with a documented dystrophin gene mutation. INSPIRE DUCHENNE is a multinational trial designed to enroll participants in the United States, Canada, the United Kingdom and Italy.
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 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.
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 achieve key clinical milestones; the Company's pipeline of capsid products, including SLB-101, and programs for neuromuscular and cardiac diseases, including its SGT-501, SGT-212 and SGT-003 programs and expectations for CTA filings, site activations, clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the sufficiency of the Company's cash, cash equivalents, and available-for-sale securities to fund its operations; 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 and license AAV-SLB101 and advance SGT-212, SGT-003, SGT-501, SGT-601, SGT-401 and other preclinical programs and capsid libraries 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; compete successfully with other companies that are seeking to develop Duchenne, Friedreich's ataxia 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-212, SGT-003, SGT-501, SGT-601, SGT-401 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]
