Solid Biosciences, a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced positive initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience.
Clinical Data of Duchenne Gene Therapy Candidate SGT-003
- Average microdystrophin expression of 110% (N=3) and significant improvements in multiple additional muscle health biomarkers observed support the potential of SGT-003 as a next-generation, best-in-class Duchenne muscular dystrophy gene therapy candidate.
- Encouraging early signals of potential cardiac benefit observed.
- SGT-003 has been well-tolerated in the 6 participants dosed as of February 11, 2025, with no serious adverse events observed.
- Participant enrollment continues, with the 7th participant dosed on February 17, 2025; Company expects to dose approximately 20 total participants by Q4 2025.
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