Sarepta Therapeutics,, Inc. Common Stock
Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat Duchenne muscular dystrophy (Duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of Duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of Duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients with Duchenne with a confirmed mutation in the Duchenne gene. The company is also developing SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Arrowhead Pharmaceuticals, Inc.; University of Western Australia; Catalent Maryland, Inc.; Nationwide Children's Hospital; Dyno Therapeutics; Hansa Biopharma; Duke University; and Genethon. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.
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Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing and commercializing therapeutics to treat and prevent human disease. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its portfolio focuses on central nervous system disorders, as well as immunology, infectious disease, and rare disease product candidates. The company's products pipeline include TNX-102 SL (cyclobenzaprine HCl sublingual tablet), for the management of fibromyalgia; and is being developed to prevent and treat acute stress reaction, posttraumatic stress disorder, Alzheimer's disease, alcohol use disorder, and multi-site pain associated with Long COVID. It also develops TNX-1300, a double-mutant cocaine esterase, which is in Phase 2 clinical trials for the treatment of cocaine intoxication; and TNX-1900, an intranasal potentiated oxytocin for the treatment of binge eating disorder, adolescent obesity, and bone health in pediatric autism, as well as for social anxiety disorder. In addition, the company develops TNX-1500, an Fc-modified humanized monoclonal antibody indicated to prevent organ transplant rejection, as well as to treat autoimmune conditions; TNX-1700, a recombinant Trefoil Factor Family 2 fusion protein for the treatment of gastric and colorectal cancers; TNX-801 that is in the pre-IND development stage for protection against smallpox and mpox; and TNX-1800, a live virus vaccine for SARS-CoV-2. Its rare disease portfolio consists of TNX-2900, an intranasal potentiated oxytocin to treat for Prader-Willi syndrome. The company has collaboration agreement with Bilthoven Biologicals to advance TNX-801 and Makana Therapeutics, Inc. to study TNX-1500 for human-compatible organs and cells for the treatment of organ failure. Tonix Pharmaceuticals Holding Corp. is headquartered in Chatham, New Jersey.
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