Sarepta Therapeutics 1 article

From a biotechnology perspective, Sarepta Therapeutics is a genetic-medicine company spanning RNA exon skipping and gene therapy shaped by exon-skipping RNA therapeutics and AAV gene therapy. For Sarepta Therapeutics, micro-dystrophin gene replacement and AAVrh74 vector platform broaden the research base, while neuromuscular genetic medicines adds a specific target, pathway, delivery method or disease context. The corporate tag for Sarepta Therapeutics includes skeletal muscle gene delivery as context, while individual drugs and mechanisms are modeled independently.