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 ( ($RARE) ) has issued an update. On April 2, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug Admin...

Ultragenyx BLA for UX111 Accepted for FDA Review

The latest update is out from Ultragenyx Pharmaceutical ( ($RARE) ). On March 30, 2026, Ultragenyx Pharmaceutical announced that the U.S. Food and ...

Ultragenyx Wins FDA IND Clearance for UX016 Therapy

Ultragenyx Pharmaceutical, Inc. ($RARE) announced an update on their ongoing clinical study. The phase 3 study “Adeno-Associated Virus Serotype 8-M...

Ultragenyx Nears Key Inflection Point With Completed Phase 3 Gene Therapy Study in GSDIa

A class action lawsuit was filed against Ultragenyx Pharmaceutical ($RARE) on February 4, 2026. The federal securities class action alleges that pl...

Ultragenyx Facing Shareholder Lawsuit over Brittle Bone Disease Drug Claims

Analysts have been eager to weigh in on the Healthcare sector with new ratings on Immutep (IMMP – Research Report) and Ultragenyx Pharmaceutical (R...

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