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

Solid Biosciences Inc., which acquired UF startup AavantiBio, announced positive initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy candidate for treating Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data from the first three participants showed an average microdystrophin expression of 110%, as measured by Western blot, along with improvements in multiple biomarkers indicating muscle health and resilience.