Solid Biosciences to Present at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Core Viewpoint - Solid Biosciences Inc. is advancing its investigational gene therapies SGT-003 for Duchenne muscular dystrophy and SGT-212 for Friedreich's ataxia, with presentations scheduled at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference [1] Group 1: SGT-003 for Duchenne Muscular Dystrophy - SGT-003 is a next-generation gene therapy designed to treat Duchenne muscular dystrophy, featuring a differentiated microdystrophin construct and a proprietary capsid, AAV-SLB101, which enhances cardiac and skeletal muscle transduction [7][8] - The INSPIRE DUCHENNE Phase 1/2 clinical trial is evaluating the safety, tolerability, and efficacy of SGT-003 in pediatric participants with a confirmed Duchenne diagnosis [9] - Preliminary findings indicate that SGT-003 stabilizes the DAPC and improves muscle integrity, with a positive safety profile and reduced liver toxicity [3][4] Group 2: SGT-212 for Friedreich's Ataxia - SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich's ataxia, designed to deliver full-length human frataxin via a dual route of administration [11] - The FALCON Phase 1b clinical trial is assessing the safety and tolerability of SGT-212 in participants aged 18-40 diagnosed with FA and cardiac hypertrophy [12] - Friedreich's ataxia affects approximately 5,000 people in the U.S. and 15,000 in Europe, with no current treatments available to halt disease progression [10] Group 3: Company Overview - Solid Biosciences focuses on developing gene therapy candidates for rare neuromuscular and cardiac diseases, including SGT-003 and SGT-212, and aims to improve the lives of patients affected by these conditions [13]