Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Solid Biosciences Inc., a leading life sciences company specialising in precision genetic medicines for neuromuscular and cardiac diseases, has announced the FDA clearance for its Investigational New Drug (IND) application for SGT-003, a groundbreaking gene therapy for Duchenne Muscular Dystrophy (DMD).

Key Highlights:

• FDA Approval: Solid Biosciences has received FDA clearance for its IND application for SGT-003, a next-generation gene therapy for Duchenne Muscular Dystrophy (DMD).
• Innovative Approach: SGT-003 combines a differentiated microdystrophin transgene with a next-generation muscle-tropic capsid and a transient transfection manufacturing process, addressing unmet needs in the Duchenne community.
• Phase 1/2 Trial: Solid Biosciences plans to initiate the Phase 1/2 open-label, multicenter clinical trial, SGT-003-101, targeting pediatric DMD patients aged 4 to < 6 years. The trial aims to determine safety, tolerability, and efficacy over a 5-year period following treatment.

The company believes that SGT-003 has the potential to make a meaningful impact on the lives of those living with Duchenne, addressing the urgent need for improved therapies for this devastating disease.

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