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About
Ultragenyx Pharmaceutical, Inc. is a clinical-stage biotechnology company that is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. It intends to build therapies to address these clinically underserved diseases. The company was founded by Emil D. Kakkis on April 22, 2010 and is headquartered in Novato, CA. | |||
Description
Ultragenyx Pharmaceutical Inc., a clinical-stage biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of various products for the treatment of rare and ultra-rare genetic diseases in the United States. The company is developing various biologics product candidates, including KRN23, a human monoclonal antibody that in Phase III adult study to bind and reduce the biological activity of fibroblast growth factor 23 to enhance abnormally low phosphate levels in patients with X-linked hypophosphatemia, as well as for the treatment of tumor-induced osteomalacia. Its biologics product candidates also comprise recombinant human beta -glucuronidase (rhGUS), an enzyme replacement therapy, which completed the Phase 3 clinical study for the treatment of mucopolysaccharidosis 7; and recombinant human protective protein cathepsin-A (rhPPCA), an enzyme replacement therapy, which is in preclinical development for galactosialidosis. The company is also developing range of small-molecule product candidates, such as UX007, a substrate replacement therapy that is in Phase II study for patients with fatty acid oxidation disorders, as well as for patients with glucose transporter type-1 deficiency syndrome; and aceneuramic acid extended-release (Ace-ER), an oral formulation of sialic acid, which is in Phase III extension study to treat GNE myopathy. It has collaboration and license agreements with Arcturus Therapeutics, Inc.; Takeda Pharmaceutical Company Limited; Kyowa Hakko Kirin Co. Ltd.; Saint Louis University; Baylor Research Institute; Nobelpharma Co., Ltd.; Alcami Corporation; HIBM Research Group; and St. Jude Children's Research Hospital. The company was founded in 2010 and is headquartered in Novato, California. | |||
