Duchenne Gene Therapy SGT-003 Shows Positive Interim Trial Data
United States – October 28, 2026 -- Solid Biosciences has dosed 40 participants with its Duchenne gene therapy SGT-003 and reported positive interim safety and biomarker data from the ongoing Phase 1/2 INSPIRE DUCHENNE trial. The company presented the update at the 2026 MDA Clinical & Scientific Conference.
The therapy delivers a shortened dystrophin protein
SGT-003 delivers microdystrophin, a shortened version of dystrophin designed to help support and protect muscle cells during movement. The data show robust microdystrophin expression. They also show restoration of beta-sarcoglycan and nNOS, proteins that normally work with dystrophin in the dystrophin-associated protein complex (DAPC), the muscle cell support system.