Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003

Core Insights - Solid Biosciences Inc. announced positive initial data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a gene therapy candidate for Duchenne muscular dystrophy, showing an average microdystrophin expression of 110% and improvements in muscle health biomarkers [2][3][4] Group 1: Clinical Trial Data - The interim 90-day biopsy data from the first three participants indicated significant microdystrophin expression and improvements in muscle health indicators [2][4] - SGT-003 demonstrated a favorable safety profile, with no serious adverse events reported among the six participants dosed as of February 11, 2025 [3][4][16] - The trial is ongoing, with plans to dose approximately 20 participants by Q4 2025 [5][6] Group 2: Safety and Tolerability - Adverse events observed were typical of AAV gene therapy, including nausea and transient declines in platelets, with no serious adverse events or unexpected serious adverse reactions [3][16] - All treatment-related adverse events resolved without sequelae, and no additional immunomodulatory agents were required [16][12] Group 3: Cardiac Benefits - Early signals of potential cardiac benefits were observed, including a reduction in hs-troponin I levels and improvements in cardiac function as measured by echocardiography [4][11][16] - At Day 180, mean cardiac function increased by 8% from baseline in two participants [11] Group 4: Future Plans - The company plans to request a meeting with the FDA in mid-2025 to discuss the potential for an accelerated approval pathway for SGT-003 [3][5] - Enrollment in the INSPIRE DUCHENNE trial is ongoing, with additional clinical sites expected to be activated by the end of 2025 [7][6]