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Emil Kakkis

Founder, Chief Executive Officer and President, Ultragenyx Pharmaceutical Inc.
,United States
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Emil Kakkis's Overview

Total Experience61 years
CompanyUltragenyx Pharmaceutical Inc.
CountryUnited States

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.

Professional Summary

Emil Kakkis's work history includes a series of influential roles in various companies. Here is a detailed list of his professional journey:

Ultragenyx Pharmaceutical Inc. - Founder, Chief Executive Officer and President (2010 to Present)
Actio Biosciences, Inc. - Board Member (2024 to Present)
EveryLife Foundation for Rare Diseases - Board Member (2009 to 2023)
National Organization for Rare Disorders - Board Member (2009 to 2011)
BioMarin - Development Consultant (2009 to 2010)
BioMarin Pharmaceutical Inc. - Chief Medical Officer (1998 to 2009)
Harbor-UCLA Research and Education Institute - Assistant Professor (1993 to 1998)
Harbor UCLA Med Center, Research and Education Institute - Assistant Professor (1993 to 1998)

Notable Achievements

Founder and CEO of Ultragenyx

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.

Pioneer in Enzyme Replacement Therapy (ERT)

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.

Development of Crysvita® (burosumab)

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.

Advocate for Rare Disease Legislation

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.

Educational Background

MD/PhD, biological chemistry

UCLA - Year 1982

Bachelor of Arts (B.A.), Biology

Pomona College - Year 1978

Wilson High School of Long Beach CA - Year 1975

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Notable news
Hiring actively
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Company Overview

Ultragenyx Pharmaceutical Inc.
Total employees1489
HeadquartersNovato
Founded2010

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.

Ultragenyx Pharmaceutical Inc. Stock Information
Ultragenyx Pharmaceutical Inc., Inc. is listed on the NASDAQ under the ticker symbol RARE. The company went public on January 31, 2014

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