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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.
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