Sarepta Company profile
Sarepta Therapeutics, Inc. focuses on the discovery and development of RNA-based therapeutics, gene therapy, and other genetic medicine approaches for the treatment of rare neuromuscular diseases. The company offers EXONDYS 51, a disease-modifying therapy for the treatment of duchenne muscular dystrophy (DMD), which is a rare genetic muscle-wasting disease caused by the absence of dystrophin. It also provides Golodirsen, a product candidate that binds to exon 53 of dystrophin pre-mRNA, which results in exclusion or skipping of exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping; and Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene. In addition, the company provides SRP-5051, a peptide conjugated PMO that binds to exon 51 of dystrophin pre-mRNA resulting in exclusion of exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. The company has strategic alliances with Nationwide Children's Hospital for the advancement of micro-dystrophin gene therapy program under the research and license option agreement, as well as Galgt2 gene therapy program under the license agreement; and Genethon for the advancement of micro-dystrophin gene therapy program under a research and exclusive license option agreement. It also has a research and license option agreement with Duke University for the advancement of gene editing CRISPR/Cas9 technology for muscular dystrophy; and a collaboration and license agreement with Summit (Oxford) Ltd. to commercialize products in Summit's utrophin modulator pipeline. The company distributes its products through a network of specialty distributors and specialty pharmacies in the United States, as well as through distributors internationally. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts. 
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