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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Vistagen Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development and commercialization of therapies for neuropsychiatric and neurological disorders. The company's product pipeline includes Fasedienol, an investigational pherine nasal spray that is in Phase III clinical trial for the treatment of anxiety in adults with social anxiety disorder; and Itruvone, an odorless synthetic investigational neuroactive pherine nasal spray, which is in Phase II clinical trial to treat major depressive disorder. It also develops PH80, an odorless and tasteless synthetic investigational pherine nasal spray that is in Phase II clinical trial for the treatment of vasomotor symptoms, including hot flashes due to menopause, premenstrual dysphoric disorder, and other women's health disorders; PH15, an investigational pherine nasal spray, which is in Phase II clinical trial for the treatment to improve cognitive impairment caused by mental fatigue and potentially other disorders; PH284, an odorless and tasteless synthetic investigational pherine nasal spray that is in Phase II clinical trial to treat loss of appetite associated with chronic disorders, such as cancer or heart disease, as well as cachexia; and AV-101, an oral prodrug that targets N-methyl-D-aspartate receptor in the brain for treating depression and neurological disorders. In addition, the company's clinical-stage pipeline consists of intranasal product candidates comprising pherines. It has a license and collaboration agreement with AffaMed Therapeutics, Inc. to develop, manufacture, and commercialize fasedienol for multiple anxiety-related disorders. Vistagen Therapeutics, Inc. was founded in 1998 and is headquartered in South San Francisco, California.
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