Sarepta Therapeutics

About Sarepta Therapeutics

What Sarepta Therapeutics does

Sarepta Therapeutics is a global biotechnology company focused on the development of precision genetic medicines to treat rare neuromuscular and central nervous system diseases including Duchenne Muscular Dystrophy, Limb-girdle Muscular Dystrophy, Facioscapulohumeral muscular dystrophy and Myotonic dystrophy type 1. It advances siRNA, RNA platforms and gene therapy while building large-scale manufacturing capacity and securing FDA-approved treatments. The company emphasizes shortening lab-to-patient timelines and rethinking access models for rare disease therapies.

Key features

• Gene Therapy
• RNA Platform
• siRNA Technology
• Gene Therapy Manufacturing
• Clinical Trials
• FDA-Approved Treatments