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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Sanofi engages in the research, development, manufacture, and marketing of therapeutic solutions. It provides immunology and inflammation, rare diseases, neurology, oncology, and other medicines and vaccines. It also offers poliomyelitis, pertussis, and haemophilus influenzae type b (Hib) pediatric vaccines; respiratory syncytial virus protection and hexavalent combination vaccines protecting against diphtheria, tetanus, pertussis, polio, Hib, and hepatitis B; influenza, booster, meningitis, and travel and endemic vaccines, that includes hepatitis A, typhoid, yellow fever, and rabies vaccines. It has a collaboration and license agreement with Exscientia to develop up to 15 novel small-molecule for oncology and immunology; ABL Bio, Inc. to develop ABL301, a treatment for alpha-synucleinopathies; Innate Pharma SA for a natural killer cell engager program targeting B7-H3; and Blackstone Life Sciences to develop pivotal studies and clinical development program. Further, it has a collaboration agreement with IGM Biosciences, Inc. to develop, manufacture, and commercialize IgM antibody; Kymera Therapeutics, Inc. to develop and commercialize protein degrader therapies targeting IRAK4 in patients with immune-inflammatory diseases; Nurix Therapeutics, Inc. to develop novel targeted protein degradation therapies; Denali Therapeutics Inc. to treat a range of systemic inflammatory diseases, such as ulcerative colitis; and Adagene Inc. for the discovery and development of antibody-based therapies. Additionally, it has a collaboration with Scribe Therapeutics Inc. to develop genome editing technologies; and co-promotion service agreement with Provention Bio, Inc. for the commercialization of teplizumab, as well as a strategic alliance with the Department of Health " Abu Dhabi for the development of global vaccines. The company was formerly known as Sanofi-Aventis and changed its name to Sanofi in May 2011. Sanofi was incorporated in 1994 and is headquartered in Paris, France.
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