Sarepta Therapeutics

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. The company offers EXONDYS 51 injection to treat duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping; and VYONDYS 53 for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. The company has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio. It also has a research and option agreement with Codiak BioSciences, Inc. to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing, and RNA technologies for neuromuscular diseases; and research collaboration with Genevant Sciences for lipid nanoparticle-based gene editing therapeutics. Sarepta Therapeutics, Inc. was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.