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.

There’s a lot to be optimistic about in the Healthcare sector as 2 analysts just weighed in on Ultragenyx Pharmaceutical (RARE) and Danaher (DHR) w...

Analysts Offer Insights on Healthcare Companies: Ultragenyx Pharmaceutical (RARE) and Danaher (DHR)

In a report released today, Tazeen Ahmad from Bank of America Securities reiterated a Buy rating on Ultragenyx Pharmaceutical, with a price target ...

Bank of America Securities Remains a Buy on Ultragenyx Pharmaceutical (RARE)

In a report released today, Laura Chico from Wedbush assigned a Hold rating to Ultragenyx Pharmaceutical, with a price target of $30.00. According ...

Wedbush Releases a Hold Rating on Ultragenyx Pharmaceutical (RARE)

Insiders have been trading these 5 stocks: (($ASX)), (($PRQR)), (($ABSI)), (($RARE)) and (($YPF)). Here is a breakdown of their recent trades and t...

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2	RARE	$25.12	-3.18%	518.47K
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2	RARE	$25.12	-3.2%	518.47K
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