Company Description
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 North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific.
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; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia.
The company’s products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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 for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome.
It also develops UX701 that is in Phase 2 clinical trial 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.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
| Country | United States |
| Founded | 2010 |
| IPO Date | Jan 31, 2014 |
| Industry | Biotechnology |
| Sector | Healthcare |
| Employees | 1,294 |
| CEO | Emil Kakkis |
Contact Details
Address: 60 Leveroni Court Novato, California 94949 United States | |
| Phone | 415 483 8800 |
| Website | ultragenyx.com |
Stock Details
| Ticker Symbol | RARE |
| Exchange | NASDAQ |
| Stock Type | Common Stock |
| Fiscal Year | January - December |
| Reporting Currency | USD |
| CIK Code | 0001515673 |
| CUSIP Number | 90400D108 |
| ISIN Number | US90400D1081 |
| Employer ID | 27-2546083 |
| SIC Code | 2834 |
Key Executives
| Name | Position |
|---|---|
| Dr. Emil D. Kakkis M.D., Ph.D. | Founder, President, Chief Executive Officer and Director |
| Howard Horn | Executive Vice President of Corporate Strategy and Chief Financial Officer |
| John Richard Pinion II | Chief Quality Operations Officer and Executive Vice President of Translational Sciences |
| Erik Harris M.B.A. | Executive Vice President and Chief Commercial Officer |
| Dr. Eric Crombez M.D. | Chief Medical Officer and Executive Vice President |
| Theodore A. Huizenga | Senior Vice President, Corporate Controller and Principal Accounting Officer |
| Karah Herdman Parschauer J.D. | Chief Legal Officer and Executive Vice President of Corporate Affairs |
| Ernie W. Meyer | Chief Human Resources Officer and Executive Vice President |
| Thomas R. Kassberg | Chief Business Officer and Executive Vice President |
| Vimal Srivastava | Senior Vice President of Business Development and Alliance Management |
Latest SEC Filings
| Date | Type | Title |
|---|---|---|
| Jul 18, 2025 | S-8 | Securities to be offered to employees in employee benefit plans |
| Jul 11, 2025 | 8-K | Current Report |
| Jul 9, 2025 | 8-K | Current Report |
| Jun 27, 2025 | 8-K | Current Report |
| Jun 20, 2025 | 144 | Filing |
| May 19, 2025 | 8-K | Current Report |
| May 7, 2025 | 10-Q | Quarterly Report |
| May 6, 2025 | 8-K | Current Report |
| Apr 24, 2025 | SCHEDULE 13G/A | Filing |
| Mar 28, 2025 | ARS | Filing |