Ultragenyx Pharmaceutical Inc.

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, Europe, and internationally. 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 treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include 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; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, 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.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

Ultragenyx Pharmaceutical Inc. trades as RARE on NASDAQ. The company is classified in Healthcare / Biotechnology and reports in USD.

The current profile places the business in Biotechnology. This section is intended to summarize the operating segments, products, geographies, and main revenue lines from official filings.

Latest available fiscal data shows $673.00M of revenue and -$575.00M of net income.

Use this area for management strategy, capital allocation priorities, target markets, and measurable goals from the latest annual report or investor presentation.

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Ultragenyx Pharmaceutical Inc. can be compared against peers such as Ascentage Pharma Group International, Apogee Therapeutics, Inc., Celcuity Inc., CG Oncology, Inc. Common stock, Centessa Pharmaceuticals plc, Dyne Therapeutics, Inc..

A complete thesis should compare growth, margins, balance-sheet risk, valuation multiples, and market position against direct competitors.

Current signals to investigate include market capitalization of $2.16B, beta of 0.38, and return on equity of +718.8%.

This section should be validated with evidence such as durable margins, brand strength, regulation, switching costs, cost advantage, distribution, or technology.

Key risks should include financial leverage, cyclicality, customer concentration, regulatory exposure, currency risk, and execution risk.

RARE currently shows total debt of $1.28B and beta of 0.38. Missing data should be treated as a research gap, not as low risk.

Production-capacity detail is not available as structured data yet. For industrial, defense, semiconductor, or real-estate companies, this should be reviewed from annual reports and investor presentations.

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Recent filings to review: 4 (2026-06-01 00:00:00), 4 (2026-05-18 00:00:00), 4 (2026-05-18 00:00:00), 4 (2026-05-18 00:00:00).

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Company website: https://www.ultragenyx.com

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