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.

Ultragenyx Pharmaceutical ( ($RARE) ) has shared an update. On February 3, 2026, Ultragenyx Pharmaceutical reported new long-term clinical data for...

Ultragenyx Highlights New UX111 Data in FDA Resubmission

There’s a lot to be optimistic about in the Healthcare sector as 3 analysts just weighed in on High Tide (HITI – Research Report), Ultragenyx Pharm...

Analysts Offer Insights on Healthcare Companies: High Tide (HITI), Ultragenyx Pharmaceutical (RARE) and Kymera Therapeutics (KYMR)

An announcement from Tactical Resources Corp. ( ($TSE:RARE) ) is now available. Tactical Resources Corp. plans to raise up to C$2 million through a...

Tactical Resources Launches $2 Million Private Placement to Fund Rare Earth Initiatives

There’s a lot to be optimistic about in the Healthcare sector as 3 analysts just weighed in on Ultragenyx Pharmaceutical (RARE – Research Report), ...

Analysts Offer Insights on Healthcare Companies: Ultragenyx Pharmaceutical (RARE), Dexcom (DXCM) and Health Catalyst (HCAT)

Companies in the Healthcare sector have received a lot of coverage today as analysts weigh in on Inogen (INGN – Research Report), Exelixis (EXEL – ...

Analysts Offer Insights on Healthcare Companies: Inogen (INGN), Exelixis (EXEL) and Ultragenyx Pharmaceutical (RARE)

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

	A	B	C	D
1	Symbol	Price	Change	Volume
2	RARE	$24.76	+2.87%	3.44M
3
4
5

	A	B	C	D
1	Symbol	Price	Change	Vol
2	RARE	$24.76	+2.9%	3.44M
3
4
5
6

RARE

RARE

Get Ultragenyx Pharmaceutical Inc. Data in Excel

Get Ultragenyx Pharmaceutical Inc. Data in Excel