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Ultragenyx Pharmaceutical ($RARE)

RARE Overview

Ultragenyx Pharmaceutical, 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 the United States.Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia; and Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII.The company is also developing small-molecule pipeline comprising UX007, a synthetic triglyceride for the treatment of long chain fatty-acid oxidation disorders, which is a set of rare metabolic diseases that prevents the conversion of fat into energy; and gene therapy pipeline consisting of DTX301, an adeno-associated virus 8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase, as well as DTX401, an AAV8 gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia.In addition, the company is developing UX068, which is in preclinical development for the treatment of creatine transporter deficiency (CTD); and UX053 for the treatment of glycogen storage disease type III.Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Arcturus Therapeutics Holdings Inc. to develop additional nucleic acid therapies for rare diseases.Ultragenyx Pharmaceutical Inc. was founded in 2010 and is headquartered in Novato, California. (From: StockAnalysis.com)

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