Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company. The Company 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.
公司代码RARE
公司名称Ultragenyx Pharmaceutical Inc
上市日期Jan 31, 2014
CEOKakkis (Emil D)
员工数量1294
证券类型Ordinary Share
年结日Jan 31
公司地址60 Leveroni Ct
城市NOVATO
上市交易所NASDAQ OMX - NASDAQ BASIC
国家United States of America
邮编94949
电话14154838800
网址https://www.ultragenyx.com/
公司代码RARE
上市日期Jan 31, 2014
CEOKakkis (Emil D)