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.

Analysts have been eager to weigh in on the Healthcare sector with new ratings on Helus Pharma (HELP), Ultragenyx Pharmaceutical (RARE) and Novavax...

Analysts Offer Insights on Healthcare Companies: Helus Pharma (HELP), Ultragenyx Pharmaceutical (RARE) and Novavax (NVAX)

Analysts have been eager to weigh in on the Healthcare sector with new ratings on Merck & Company (MRK), Envista Holdings (NVST) and Ultragenyx Pha...

Analysts Offer Insights on Healthcare Companies: Merck & Company (MRK), Envista Holdings (NVST) and Ultragenyx Pharmaceutical (RARE)

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Goldman Sachs analyst Salveen Richter maintained a Hold rating on Ultragenyx Pharmaceutical today and set a price target of $19.00. Richter covers ...

Goldman Sachs Reaffirms Their Hold Rating on Ultragenyx Pharmaceutical (RARE)

Ultragenyx Pharmaceutical, Inc. ($RARE) announced an update on their ongoing clinical study. The Phase 1/2 study A Phase 1/2, First-in-human, Doubl...

Ultragenyx Expands Rare Muscle Pipeline With First-in-Human UX016 Trial in GNE Myopathy

Analysts have been eager to weigh in on the Healthcare sector with new ratings on Ultragenyx Pharmaceutical (RARE) and Lifecore Biomedical (LFCR). ...

Analysts Offer Insights on Healthcare Companies: Ultragenyx Pharmaceutical (RARE) and Lifecore Biomedical (LFCR)

Canaccord Genuity analyst Whitney Ijem maintained a Buy rating on Ultragenyx Pharmaceutical today and set a price target of $37.00. Ijem covers the...

Canaccord Genuity Sticks to Its Buy Rating for Ultragenyx Pharmaceutical (RARE)

Ultragenyx Pharmaceutical received a Hold rating and price target from Evercore ISI analyst Gavin Clark-Gartner yesterday. According to TipRanks, C...

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2	RARE	$14.50	-1.83%	4.31M
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2	RARE	$14.50	-1.8%	4.31M
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RARE

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