Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company headquartered in Novato, California, focused on developing novel therapies for rare and ultra-rare diseases. The company was founded in 2010 by Dr. Emil D. Kakkis with a mission to bring life-changing therapies to patients who have no treatment options.
Overview
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Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company headquartered in Novato, California, focused on developing novel therapies for rare and ultra-rare diseases. The company was founded in 2010 by Dr. Emil D. Kakkis with a mission to bring life-changing therapies to patients who have no treatment options.
Overview
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Ultragenyx specializes in developing treatments for rare genetic diseases, many of which affect the nervous system. The company uses multiple technology platforms including gene therapy, mRNA therapy, and small molecule approaches to address these unmet medical needs["@ultragenyxa"].
Funding
IPO: 2014 (NASDAQ: RARE)
Market Cap: ~$6B (2026)
Corporate Highlights
Founded: 2010
Headquarters: Novato, California
NASDAQ: RARE
Employees: Approximately 1,000
History and Development
Founding and Early Growth (2010-2015)
Ultragenyx was founded in 2010 by Dr. Emil D. Kakkis, a pediatrician and pharmaceutical executive with deep expertise in rare disease drug development. The company initially focused on building a pipeline of therapies for ultra-rare metabolic and neurological disorders.
Clinical Development (2015-2020)
2015: Advanced first programs into clinical trials
2017: FDA approval of Crysvita (burosumab) for X-linked hypophosphatemia
2018: FDA approval of Mepsevii (vestronidase alfa) for mucopolysaccharidosis VII
2019: FDA approval of Dojapviz (givosiran) for acute hepatic porphyria
Recent Advances (2021-Present)
Multiple gene therapy programs in late-stage development
Expansion of mRNA platform
Strategic partnerships for manufacturing
Approved Products
Crysvita (Burosumab)
Indication: X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia
Mechanism: FGF23 antibody
Approval: 2017 (FDA), 2018 (EU)
First disease: First therapy directly addressing FGF23 excess in XLH[@crysvita]
Mepsevii (Vestronidase Alfa)
Indication: Mucopolysaccharidosis VII (Sly syndrome)
Mechanism: Recombinant beta-glucuronidase
Approval: 2017
Significance: First approved therapy for this rare metabolic disorder[@mepsevii]
Dojapviz (Givosiran)
Indication: Acute hepatic porphyria
Mechanism: siRNA targeting ALAS1
Approval: 2019
Significance: First RNA interference therapy for this condition[@dojapviz]