Ultragenyx Pharmaceutical Inc. (RARE)
NASDAQ: RARE · Real-Time Price · USD
35.97
-1.85 (-4.89%)
Mar 31, 2025, 9:47 AM EDT - Market open

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.

Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical logo
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
Karah Herdman Parschauer J.D. Chief Legal Officer and Executive Vice President of Corporate Affairs
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
Theodore A. Huizenga Senior Vice President, Corporate Controller and Principal Accounting Officer
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
Dennis Karl Huang Chief Technical Operations Officer and Executive Vice President of Gene Therapy Research and Development

Latest SEC Filings

Date Type Title
Mar 28, 2025 ARS Filing
Mar 28, 2025 DEFA14A Additional definitive proxy soliciting materials and Rule 14(a)(12) material
Mar 28, 2025 DEF 14A Other definitive proxy statements
Feb 28, 2025 144 Filing
Feb 27, 2025 144 Filing
Feb 19, 2025 10-K Annual Report
Feb 13, 2025 8-K Current Report
Feb 12, 2025 SCHEDULE 13G/A Filing
Feb 10, 2025 SCHEDULE 13G/A Filing
Jan 13, 2025 8-K Current Report