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About Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical, Inc. engages in the identification, acquisition, development, and commercialization of novel products for the treatment of genetic diseases. The company is headquartered in Novato, California and currently employs 1,276 full-time employees. The company went IPO on 2014-01-31. The firm is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. Its therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The Company’s four approved product candidates include Crysvita (burosumab) for the treatment of X-linked hypophosphatemia (XLH), and tumor-induced osteomalacia (TIO), Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII (MPSVII) or Sly Syndrome, Dojolvi (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its clinical product candidates include DTX401, DTX301, and UX701. DTX401 for the treatment of glycogen storage disease type la.
Industry: Pharmaceutical Preparations Peers: ACADIA PHARMACEUTICALS INC AMICUS THERAPEUTICS, INC. ARROWHEAD PHARMACEUTICALS, INC. Beam Therapeutics Inc. Denali Therapeutics Inc. INSMED Inc PTC THERAPEUTICS, INC. Relay Therapeutics, Inc. Vir Biotechnology, Inc.
