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.
종목 코드 RARE
회사 이름Ultragenyx Pharmaceutical Inc
상장일Jan 31, 2014
설립일2011
CEODr. Emil D. Kakkis, M.D., Ph.D.
직원 수1294
유형Ordinary Share
회계 연도 종료Jan 31
주소60 Leveroni Ct
도시NOVATO
증권 거래소NASDAQ Global Select Consolidated
국가United States of America
우편 번호94949
전화14154838800
웹사이트https://www.ultragenyx.com/
종목 코드 RARE
상장일Jan 31, 2014
설립일2011
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