Emil D. Kakkis, M.D., Ph.D.

Chief Executive Officer and President

Dr. Kakkis is currently Chief Executive Officer, President and Founder of Ultragenyx Pharmaceutical where he leads a team developing and commercializing multiple rare and ultra-rare disease treatments.

Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and working on policy issues affecting rare disease treatment development. He began his work as an assistant professor developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I. After joining BioMarin in 1998, Dr. Kakkis guided the development and approval of two more treatments for rare diseases, MPS VI and PKU and has contributed to the development of approved or development stage products of four other rare diseases (CN2, MPSIVA, PKU, Achondroplasia).

Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing as many rare and ultra-rare disease therapeutics. The company went public in January 2014 (RARE; NASDAQ). Since its founding, Ultragenyx has grown to more than 500 employees developing treatments for seven clinical stage rare and ultra-rare diseases and has now received approvals for two new products for rare diseases, Crysvita® for XLH and Mepsevii® for MPS VII. The company works on rare metabolic, bone, muscle and neurologic diseases with no approved treatments.   

Dr. Kakkis graduated from Pomona College, magna cum laude and received the Vaile prize for his biology research.  He received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program and received the Bogen Prize for his research on graduation. He completed both 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. Dr. Kakkis is board certified in both Pediatrics and Medical Genetics.