Solid Biosciences to Present at 2025 Neuromuscular Study Group Annual Scientific Meeting

Core Insights - Solid Biosciences Inc. is presenting data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a gene therapy for Duchenne muscular dystrophy, at the NMSG Annual Meeting in Italy [1][2] - The company emphasizes the favorable profile of SGT-003, particularly its liver tolerability and early biomarker signals, supported by a steroid-only immunomodulation regimen [2] - Solid Biosciences has established over 25 licensing agreements for the use of its next-generation capsid, AAV-SLB101, which is designed for targeted gene therapy [2] Company Overview - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy [6][7] - The company aims to improve the lives of patients affected by devastating rare diseases, with a mission rooted in personal experiences with Duchenne [7] Product Details - SGT-003 is an investigational gene therapy featuring a differentiated microdystrophin construct and the proprietary AAV-SLB101 capsid, designed to enhance muscle transduction while minimizing liver targeting [4] - The microdystrophin construct includes R16/17 domains, which may improve blood flow to muscles, potentially reducing muscle breakdown and fatigue [4] Clinical Trial Information - INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial assessing the safety, tolerability, and efficacy of SGT-003 in pediatric patients with Duchenne [5] - The trial is multinational, enrolling participants from the United States, Canada, the United Kingdom, and Italy [5]