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 approaches 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 Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; SRP-9003, a limb-girdle muscular dystrophies gene therapy program; and LYS-SAF 302 to treat mucopolysaccharidosis type IIIA. The company has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; StrideBio; the United States Army Medical Research Institute of Infectious Diseases; the Department of Defense's lead laboratory; the Dyno Therapeutics, Inc; and Personalis, Inc. 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 founded in 1980 and is headquartered in Cambridge, Massachusetts.
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