Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical is a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare genetic diseases. Founded in 2010 by Emil D. Kakkis, the company is headquartered in Novato, California and has established itself as a leader in the field of orphan drug development. Its strategy centers on leveraging advanced modalities, including gene therapy, mRNA technology, and antibody-based approaches, to address serious unmet medical needs. The company's commercial portfolio and robust clinical pipeline target a range of conditions, such as X-linked hypophosphatemia, various glycogen storage diseases, and muscular dystrophy.

History

The company was established in 2010 by Emil D. Kakkis, a physician-scientist previously involved with the BioMarin Pharmaceutical team that developed treatments for mucopolysaccharidosis. Its initial focus was on ultra-rare metabolic disorders, securing early funding from venture capital firms like New Enterprise Associates and Adage Capital Management. A significant early milestone was the 2013 collaboration with Kyowa Kirin to develop burosumab, which later became the approved therapy Crysvita. The company completed its initial public offering on the NASDAQ in 2014, raising capital to advance its clinical programs. Subsequent years saw strategic expansion through acquisitions, such as Dimension Therapeutics in 2017 to bolster its gene therapy capabilities, and the establishment of a commercial infrastructure to launch its first products globally.

Products and pipeline

The commercial portfolio includes Crysvita (burosumab), approved for X-linked hypophosphatemia and tumor-induced osteomalacia, which was developed in partnership with Kyowa Kirin. Another key product is Dojolvi (triheptanoin), a proprietary medium-chain triglyceride oil for treating long-chain fatty acid oxidation disorders. The company also markets Mepsevii (vestronidase alfa-vjbk) for mucopolysaccharidosis VII. Its clinical pipeline features investigational therapies across multiple platforms, including DTX401 (a gene therapy for glycogen storage disease type Ia), UX143 (a monoclonal antibody targeting activin A for fibrodysplasia ossificans progressiva), and GTX-102 (an antisense oligonucleotide for Angelman syndrome). Preclinical work is advancing in areas like congenital adrenal hyperplasia and other muscle diseases utilizing mRNA and adeno-associated virus vector technologies.

Research and development

The company's research strategy is built on identifying validated biological targets in monogenic disorders and applying tailored technological solutions. A core focus is on advancing next-generation gene therapy constructs, often utilizing novel adeno-associated virus capsids to improve targeting and efficiency for diseases affecting the liver and central nervous system. Its mRNA platform, enhanced by the acquisition of ExpandBio, aims to develop therapies for intracellular protein replacement. Research efforts in muscle diseases include programs for Duchenne muscular dystrophy and facioscapulohumeral muscular dystrophy. The R&D team works closely with academic institutions like the University of Florida and patient advocacy groups, such as the Angelman Syndrome Foundation, to guide preclinical and clinical development.

Collaborations and partnerships

Strategic alliances are fundamental to the company's operational model. Its longest-standing partnership is with Kyowa Kirin for the global co-development and commercialization of Crysvita. In gene therapy, key collaborations include work with Dyno Therapeutics to apply artificial intelligence for capsid discovery and with Arcturus Therapeutics on mRNA therapeutics for ornithine transcarbamylase deficiency. The company has also entered into agreements with Bayer for a gene therapy program targeting Wilson disease and with GeneTx Biotherapeutics for the development of GTX-102 for Angelman syndrome. These partnerships often involve shared R&D costs, milestone payments, and profit-sharing arrangements to de-risk and accelerate the development pathway.

Corporate affairs

The company is led by CEO and President Emil D. Kakkis, with Shalini Sharp serving as Chief Financial Officer and Eric Crombez as Chief Commercial Officer. Its corporate headquarters are in Novato, California, with additional major offices in Boston and Amsterdam. As a public company, its stock trades on the NASDAQ under the ticker symbol "RARE". The company engages extensively with the rare disease community, providing programs like UltraCare for patient support and maintaining a strong commitment to regulatory science to navigate the approval processes of agencies like the U.S. Food and Drug Administration and the European Medicines Agency. Its manufacturing strategy relies on a network of contract development and manufacturing organization partners to produce clinical and commercial supplies.

Category:Biotechnology companies of the United States Category:Pharmaceutical companies established in 2010 Category:Companies based in Marin County, California