Ultragenyx Pharmaceutical Inc (RARE) USD0.001

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Business summary
Ultragenyx Pharmaceutical Inc. is a clinical-stage biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. Its clinical-stage pipeline consists of two product categories: biologics (including a monoclonal antibody and an enzyme replacement therapy), and small-molecule substrate replacement therapies. Its product candidates under biologics category include KRN23 (UX023) and recombinant human beta-glucuronidase (rhGUS) (UX003). Its product candidates under small-molecule category include UX007 and aceneuramic acid extended-release (Ace-ER) (UX001). It is also developing recombinant human protective protein cathepsin-A (rhPPCA). KRN23 is a fully human monoclonal antibody. rhGUS is an intravenous (IV) enzyme replacement therapy. UX007 is a substrate replacement therapy. It is developing Ace-ER for the treatment of GNE myopathy. rhPPCA is in preclinical development.
Contact details
Important dates
General stock information
- Short code:
- RARE
- ISIN:
- US90400D1081
- Market cap:
- $8.88 billion
- Shares in issue:
- 65.93 million
- Sector:
- Biotechnology
- Exchange:
- NASDAQ
- Country:
- United States
- Currency:
- US dollar
- Indices:
- NASDAQ Comb. Composite
Key personnel

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