Company
Headquarters: Novato, CA, United States
Founded: 2010
Employees: 1,119
CEO: Dr. Emil D. Kakkis M.D., Ph.D.
$2.28 Billion
USD as of Jan. 1, 2026
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Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; and Dojolvi for treating long-chain fatty acid oxidation disorders. The company is also developing DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
| Last Financial Reports Date | Sept. 30, 2025 |
| Revenue TTM | $630.6 M |
| EBITDA | $-508,700,992 |
| Gross Profit TTM | $-163,816,000 |
| Profit Margin | -91.95% |
| Operating Margin | -106.85% |
| Quarterly Revenue Growth | 14.70% |
Ultragenyx Pharmaceutical Inc. has the following listings and related stock indices.
Stock: NASDAQ: RARE wb_incandescent
Stock: FSX: UP0 wb_incandescent
Research Antibodies, Biotech
Emil Kakkis (president & founder)