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Phase 3 study of SGT-003 gene therapy in ambulant boys with Duchenne muscular dystrophy
2025-522949-22-00Phase 3Authorised
- Sponsor
- Solid Biosciences
- Start
- Oct 2026
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 9
01
Overview
Phase 3, randomised, double-blind trial in Duchenne muscular dystrophy (DMD) comparing SGT-003 with Placebo (2 arms). Primary endpoint: Change from baseline in time to rise velocity at Day 540. Planned start Oct 2026; status: authorised.
02
Drugs
SGT-003
03
Conditions
Duchenne muscular dystrophy (DMD)
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