Ultragenyx Pharmaceutical Inc. (RARE)

About Ultragenyx Pharmaceutical Inc.

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, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

Company profile, industry and headcount are as reported by our data provider and can lag a company's own filings by a reporting period. The annual report is the authority.

Super investor ownership

7 of the funds we track reported a position in their latest SEC 13F filing. Largest first:

• Cliff Asness, AQR CAPITAL MANAGEMENT LLC$214.14M · 0.1% of book
• Two Sigma, TWO SIGMA INVESTMENTS, LP$120.91M · 0.1% of book
• D. E. Shaw, D. E. Shaw & Co., Inc.$57.77M · 0.0% of book
• Israel Englander, MILLENNIUM MANAGEMENT LLC$25.40M · 0.0% of book
• Renaissance Technologies, RENAISSANCE TECHNOLOGIES LLC$13.81M · 0.0% of book

A word of warning on reading these figures: a 13F reports the market value of a holding, so a fund that traded nothing at all still appears to have sold when the price fell. We found 102 companies where the standard reading gives the opposite answer. Only the share count is honest.

Politician trades in RARE

Trades in RARE disclosed by members of Congress and executive-branch officials, latest first:

• Gilbert Cisneros · HouseBuy · $1,001 - $15,000 · traded 3 Mar 2026 · disclosed 7 Apr 2026
• Gilbert Cisneros · HouseBuy · $1,001 - $15,000 · traded 18 Feb 2026 · disclosed 9 Mar 2026
• Gilbert Cisneros · HouseBuy · $1,001 - $15,000 · traded 8 Aug 2025 · disclosed 12 Sept 2025
• Gilbert Cisneros · HouseBuy · $1,001 - $15,000 · traded 15 Jul 2025 · disclosed 14 Aug 2025

A disclosure reports a value band, not an amount, and is due up to 45 days after the trade; some arrive later. Every filer we track, each with their own page, is on the Congress trading tracker.

Common questions