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.

In a report released yesterday, Anupam Rama from J.P. Morgan maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $72.00. T...

Ultragenyx Pharmaceutical (RARE) Receives a Buy from J.P. Morgan

Ultragenyx Pharmaceutical, Inc. (($RARE)) has held its Q1 earnings call. Read on for the main highlights of the call. Ultragenyx Pharmaceutical’s l...

Ultragenyx Earnings Call Balances Growth and Cash Burn

Ultragenyx Pharmaceutical ( ($RARE) ) has issued an announcement. At its Annual Meeting of Stockholders held on May 14, 2026, Ultragenyx Pharmaceut...

Ultragenyx Shareholders Approve Incentive Plan, Elect Directors

There’s a lot to be optimistic about in the Healthcare sector as 3 analysts just weighed in on Kodiak Sciences (KOD), IN8bio (INAB) and Ultragenyx ...

Analysts Offer Insights on Healthcare Companies: Kodiak Sciences (KOD), IN8bio (INAB) and Ultragenyx Pharmaceutical (RARE)

In a report released today, Eliana Merle from Barclays maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $43.00. Merle c...

Barclays Keeps Their Buy Rating on Ultragenyx Pharmaceutical (RARE)

There’s a lot to be optimistic about in the Healthcare sector as 2 analysts just weighed in on Alphatec Holdings (ATEC) and Ultragenyx Pharmaceutic...

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