Ultragenyx

Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel products for the treatment of rare and ultra-rare diseases. The company is based in Novato, California, and was founded in 2010 by Emil D. Kakkis, a physician and scientist known for his work in developing treatments for rare diseases.

History

Ultragenyx was founded by Emil D. Kakkis in 2010. Kakkis previously served as the Chief Medical Officer of BioMarin Pharmaceutical, where he led the development of several treatments for rare diseases. Ultragenyx was established with the goal of developing effective therapies for rare and ultra-rare diseases, a group of conditions that collectively affect millions of people worldwide but each of which individually affects fewer than 200,000 people in the United States.

Products and Research

Ultragenyx has a diverse portfolio of product candidates, including therapies for Mucopolysaccharidosis VII (MPS VII), X-linked hypophosphatemia (XLH), and long-chain fatty acid oxidation disorders (LC-FAOD). The company's first approved product, vestronidase alfa (Mepsevii), is a treatment for MPS VII. Ultragenyx's second approved product, burosumab (Crysvita), is a treatment for XLH.

Ultragenyx's research approach is based on understanding the underlying pathophysiology of rare diseases and developing targeted therapies that can address these mechanisms. The company's research and development efforts are focused on several therapeutic areas, including enzyme replacement therapy, gene therapy, and small molecule therapy.

Financials

As a publicly traded company, Ultragenyx's financial performance is closely watched by investors and analysts. The company's revenue is primarily derived from the sales of its approved products, as well as from collaborations with other biopharmaceutical companies.

See Also

• Emil D. Kakkis
• BioMarin Pharmaceutical
• Rare diseases
• Mucopolysaccharidosis VII
• X-linked hypophosphatemia
• Long-chain fatty acid oxidation disorders