Sarepta Therapeutics Inc

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. It is developing therapeutic candidates for a range of diseases and disorders, including Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. Its commercial products, EXONDYS 51 (eteplirsen) Injection, VYONDYS 53 (golodirsen) Injection and AMONDYS 45 (casimersen) Injection is for the treatment of DMD in patients, who have a confirmed mutation of the DMD gene that is amenable to exon 51, exon 53 and exon 45 skipping. It is also developing gene therapy programs for various forms of LGMDs. Its LGMD product candidate, SRP-9003, is designed to transfer a gene that codes for and restores beta-sarcoglycan protein.