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 Japan Europe and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143 a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102 an antisense oligonucleotide for the treatment of Angelman syndrome; UX111 (ABO-102) an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A or MPS IIIA a rare lysosomal storage disease; UX701 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co. Ltd. The company was incorporated in 2010 and is headquartered in Novato California.