Ultragenyx Pharmaceutical Inc (RARE) USD0.001
0.27%- This stock can be held in a
Stocks and Shares ISA
$0.48
(1.63%)$0.48
(1.63%)Company Information
About this company
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.
Key people
Key facts
- EPICRARE
- LocationUnited States
- SectorHealthcare
- IndustryBiotechnology & Drugs
- ISINUS90400D1081
- Market cap$2.79bn
- Employees1,294
- Shares in issue94.70m
- ExchangeNASDAQ
- IndexTR Equity United States Index
- Website
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