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.

Cantor Fitzgerald analyst Kristen Kluska maintained a Buy rating on Ultragenyx Pharmaceutical yesterday and set a price target of $105.00. The comp...

Cantor Fitzgerald Reaffirms Their Buy Rating on Ultragenyx Pharmaceutical (RARE)

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In a report released today, Luca Issi from RBC Capital maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $72.00. Issi co...

RBC Capital Reaffirms Their Buy Rating on Ultragenyx Pharmaceutical (RARE)

In a report released today, Yaron Werber from TD Cowen maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $75.00. The com...

TD Cowen Sticks to Their Buy Rating for Ultragenyx Pharmaceutical (RARE)

There’s a lot to be optimistic about in the Healthcare sector as 2 analysts just weighed in on Ultragenyx Pharmaceutical (RARE – Research Report) a...

Analysts Offer Insights on Healthcare Companies: Ultragenyx Pharmaceutical (RARE) and Exelixis (EXEL)

Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On October 30, 2025, Ultragenyx Pharmaceutical announced the dosing of the first pat...

Ultragenyx Begins Dosing in Aurora Study for Angelman Syndrome

Ultragenyx Pharmaceutical, Inc. (($RARE)) announced an update on their ongoing clinical study. Ultragenyx Pharmaceutical Inc. is conducting a follo...

Ultragenyx’s Ongoing Study on UX111: A Potential Game-Changer for MPS IIIA Treatment

Ultragenyx Pharmaceutical, Inc. (($RARE)) announced an update on their ongoing clinical study. Study Overview: Ultragenyx Pharmaceutical Inc. is co...

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