Dr. Kakkis is currently Ultragenyx’s President and Chief Executive Officer. He is also President of the non-profit EveryLife Foundation for Rare Diseases, an organization he founded in 2009 to work on regulatory policy issues. Over the last 22 years Dr. Kakkis is best known for his work developing novel treatments for rare disorders. He began his work developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I, with minimal funding and support. The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of the Ryan Foundation, a patient organization formed by Mark and Jeanne Dant for their son Ryan. Aldurazyme development was later supported by BioMarin and Genzyme leading to FDA approval in 2003. After joining BioMarin in 1998, Dr. Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU, and has contributed to the initiation of 7 other treatment programs for rare disorders, three of which are now in clinical development or approved.
After 11 years at BioMarin, Dr. Kakkis funded and launched the EveryLife Foundation for Rare Diseases, a non-profit foundation dedicated to the acceleration of biotech innovation for rare diseases through practical and scientifically sound improvements to development strategies, regulatory policy and law. The Foundation successfully advocated for improvements to the accelerated approval pathway for rare disease treatments that was passed by Congress in FDASIA in 2012.
Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing as many rare and ultra-rare disease therapeutics as possible. Since its founding, Ultragenyx has grown to more than 100 employees developing treatments for five rare and ultra-rare diseases in Phase 2 and Phase 3 clinical development. The company went public in January 2014 (RARE NASDAQ).
Dr. Kakkis is board certified in both Pediatrics and Medical Genetics. He graduated from Pomona College, magna cum laude and received the Vaile prize for his research. He received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program and received the Bogen prize for his research. He completed a Pediatrics residency and Medical Genetics Training Fellowship at Harbor- UCLA Medical Center from 1989-1993 and from 1993 to 1998, was an assistant professor of Pediatrics at Harbor-UCLA Medical Center where he initiated the enzyme therapy program for MPS I.