Dr. Emil D. Kakkis is a distinguished physician-scientist and a leading figure in the development of treatments for rare and ultra-rare diseases. He is the founder, Chief Executive Officer, and President of Ultragenyx Pharmaceutical, a company he established in 2010 with a mission to deliver novel therapies to patients with debilitating genetic conditions. With a combined M.D. and Ph.D. from the UCLA School of Medicine, his career is defined by a deep commitment to translating scientific breakthroughs into life-changing medicines. Prior to founding Ultragenyx, he was a key leader at BioMarin Pharmaceutical, where he pioneered the development of enzyme replacement therapies, including the first approved treatment for MPS I. Dr. Kakkis is also a prominent advocate for policy reform to support and accelerate the drug development process for rare diseases.
Emil Kakkis's work history includes a series of influential roles in various companies. Here is a detailed list of his professional journey:
Established Ultragenyx in 2010, building it into a leading biopharmaceutical company focused exclusively on developing and commercializing therapies for patients with rare and ultra-rare genetic diseases.
Led the successful clinical development of Aldurazyme® (laronidase) for Mucopolysaccharidosis I (MPS I) during his tenure at BioMarin, creating a new standard of care for this severe metabolic disorder.
Oversaw the development and approval of Crysvita®, a breakthrough treatment for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO), which fundamentally changed the treatment paradigm for these conditions.
Actively influences U.S. policy to accelerate the development of therapies for rare diseases, contributing expert testimony and insights that helped shape key legislation like the 21st Century Cures Act.
UCLA - Year 1982
Pomona College - Year 1978
Wilson High School of Long Beach CA - Year 1975
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Ultragenyx is a commercial-stage biopharmaceutical company committed to developing and delivering novel therapies for patients with serious rare and ultra-rare genetic diseases. The company has a diverse portfolio of approved medicines and product candidates aimed at addressing diseases with high unmet medical needs, for which the biology and symptomatology are well-characterized.
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