Solid Biosciences to Present at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
CHARLESTOWN, Mass., March 06, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced presentations on SGT-003, its next-generation investigational gene therapy for Duchenne muscular dystrophy, and SGT-212, its dual-route investigational gene therapy for Friedreich’s ataxia (FA), at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Orlando, Florida, March 8-11, 2026.
Oral Presentation
Title: Update on the INSPIRE DUCHENNE Phase 1/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy
Poster Number: 441 O
Session: Clinical Trial Updates
Room: Florida 4
Date/Time: March 11, 2026, at 1:00 p.m. ET
Presenter: Aravindhan Veerapandiyan, MD, Director of the Comprehensive Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children 's Hospital, and principal investigator in the INSPIRE DUCHENNE clinical trial
Poster Presentations
All posters from Solid Biosciences will be exhibited on Tuesday, March 10.
Title:Novel Pharmacokinetic and Pharmacodynamic Effects of AAV-SLB101, Solid Biosciences’ Rationally Designed, Next-Generation Capsid
Poster Number: 274 T
Presenter: Matthew Harmelink, MD, Medical Director and Therapeutic Head, Neuromuscular, Solid Biosciences
Title: Positive Preliminary Safety and Liver Toxicity Profile Using SGT-003, Solid Biosciences’ Next-Generation Investigational Gene Therapy for Duchenne Muscular Dystrophy
Poster Number: 279 T
Presenter: Patrick Gonzalez, PhD, Vice President, Head of Clinical Science at Solid Biosciences
Title: SGT-003 Gene Therapy Stabilizes the DAPC and Improves Muscle Integrity in Duchenne Muscular Dystrophy
Poster Number: 266 T
Presenter: Patrick Gonzalez, PhD, Vice President, Head of Clinical Science at Solid Biosciences
Title: SGT-003 Demonstrates High Cardiac Tropism and Positive Preliminary Clinical Findings Using the AAV-SLB101 Next-Generation Muscle-Tropic Capsid
Poster Number: 278 T
Presenter: Patrick Gonzalez, PhD, Vice President, Head of Clinical Science at Solid Biosciences
Title: Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia
Poster Number: 299 T
Presenter: Jun Lee, PhD, Director, Clinical Science at 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 data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and in nonclinical studies. SGT-003’s microdystrophin construct uniquely includes the R16/17 binding domain, which localizes nNOS to the muscle membrane. 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. INSPIRE DUCHENNE is a multinational trial designed to enroll participants in the United States, Canada, the United Kingdom and Italy.
About Friedreich’s Ataxia (FA)
FA is an inherited, life-threatening, degenerative multisystem disease caused by variants in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes, including energy production. FA is known to cause progressive nervous system damage, movement problems, and cardiac dysfunction, with cardiac complications identified as the primary cause of death. FA impacts approximately 5,000 people in the United States and 15,000 in Europe. There are currently no treatments that provide a cure or halt disease progression.
About SGT-212
SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (FXN) via a dual route of administration: intradentate nucleus (IDN) infusion, using an FDA-approved neurosurgical device in a stereotactic, precision MRI-guided technique, followed by an intravenous (IV) infusion, with the intent to increase therapeutic FXN levels in the cerebellar dentate nuclei, cardiomyocytes and other systemic tissues. Targeted delivery to the dentate nuclei will be confirmed in real time via MRI. Restoration of FXN levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address neurologic, cardiac and systemic manifestations of the disease.
About the FALCON Clinical Trial
FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who have been diagnosed with FA and cardiac hypertrophy. FALCON is being conducted in the United States.
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 and other clinical and pre-clinical programs; expectations for site activations, planned enrollment, planned data announcements, planned regulatory interactions and the potential approval pathways for SGT-003; 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 and other clinical and 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 and its 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
Senior Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
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