Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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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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