PTC Therapeutics, Inc.
PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.
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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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