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.

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Ultragenyx Highlights New UX111 Data in FDA Resubmission

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In a report released today, Yigal Nochomovitz from Citi maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $50.00. Nochom...

Market Cap	$59.93M	P/E Ratio	N/A
52W High	$46.50	52W Low	$18.41
Div Yield	N/A	Payout Ratio	N/A
EPS Growth 3Y	2.1%	52W Position	22.9%
Overall Score	46	Value Score	40
Quality Score	50	Technical Score	50

EPS Current Est	$-5.75	EPS Next Est	$-4.23
EPS Growth Est	N/A	DPS Growth 3Y	0.0%

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1	Symbol	Price	Change	Volume
2	RARE	$24.83	+0.28%	1.24M
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1	Symbol	Price	Change	Vol
2	RARE	$24.83	+0.3%	1.24M
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RARE

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