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Ultragenyx Pharmaceutical Inc. NasdaqGS:RARE 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.

Location: 60 Leveroni Court, Novato, CA, 94949, United States | Website: https://www.ultragenyx.com | Industry: Biotechnology | Sector: Healthcare

Market Cap

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52 Wk Range

$ - $

Previous Close

$N/A

Open

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Day Range

N/A - N/A

Volume

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Avg Volume

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Enterprise Value

3.708B

Cash

494.4M

Avg Qtr Burn

-97.48M

Short % of Float

6.48%

Insider Ownership

3.47%

Institutional Own.

98.52%

Qtr Updated

03/31/25

Drug Pipeline

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Drug & Indication	Stage & Event	Catalyst Date
UX111 (ABO-102) Details Rare diseases, Sanfilippo Syndrome	PDUFA Approval decision	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
DTX401 Details Glycogen Storage Disease Type Ia	BLA Submission	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
DTX301 Details Ornithine transcarbmylase deficiency	Phase 3 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
GTX-102 Details Angelman Syndrome	Phase 3 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
UX143 (Setrusumab) Details Osteogenesis imperfecta	Phase 2/3 Update	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
UX701 Details Rare diseases, Wilson's disease	Phase 1/2 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
UX053 Details Rare diseases, glycogen storage disease type III	Phase 1/2 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.