Sarepta Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery and development of RNA-based therapeutics 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 develops SRP-4045 and SRP-4053, which are exon skipping clinical product candidates for the treatment of DMD. The company has a strategic alliance with Nationwide Children's Hospital for the advancement of microdystrophin gene therapy program under the research and option agreement, as well as Galgt2 gene therapy program under the license agreement; Catabasis Pharmaceuticals, Inc to explore a combination drug treatment approach for DMD under the research collaboration agreement; and Charley's Fund, Inc. to support the development of product candidates using its proprietary exon-skipping technologies under the research agreement. It also has a license agreement with the University of Western Australia for treatment of DMD by inducing the skipping of certain exons; and collaboration and license agreement with Summit (Oxford) Ltd. for the development of ezutromid, an utrophin modulator which is in phase II clinical trials for the treatment of DMD. The company distributes its products through a network of specialty distributors and specialty pharmacies in the United States. Sarepta Therapeutics, Inc. was founded in 1980 and is headquartered in Cambridge, Massachusetts.
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