Sarepta Therapeutics (SRPT)

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company that focuses on the discovery and development of RNA-based therapeutics targeting rare and infectious diseases. It concentrates on the development of exon-skipping drug candidates targeting Duchenne muscular dystrophy (DMD), a rare genetic disorder affecting children. The company received accelerated approval for Exondys 51, Amondys 45 (casimersen) and Vyondys 53 (golodirsen). Exondys 51 is Sarepta's first PMO-based technology to be granted accelerated approval by the FDA. The company is conducting a confirmatory study on the clinical benefits of these drugs for gaining continued approval. The rest of its exon-skipping DMD pipeline includes a PPMO-based, exon 51 skipping candidate, SRP-5051. Sarepta is also developing gene therapies for treating DMD, Limb-girdle muscular dystrophy and central nervous system disorders including Mucopolysaccharidosis type IIIA and Pompe Disease.