Ultragenyx Pharmaceutical, Inc. is a biopharmaceutical company, which 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 1,294 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. Its 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, UX701, UX143, UX111, and GTX-102. UX143 for the treatment of Osteogenesis Imperfecta.
