Emil D. Kakkis

Dr. Kakkis is currently Chief Executive Officer, President and Founder of Ultragenyx Pharmaceutical. Ultragenyx was founded in 2010 to focus on developing treatments for rare and ultra-rare genetic diseases. The company went public in January 2014 (RARE; NASDAQ) and has grown to more than 700 employees. Ultragenyx now has two approved therapies for rare diseases, Crysvita® for XLH and Mepsevii® for MPS VII.

Throughout his career, 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). He has worked to advance the cause of rare disease treatment through advocacy on policy issues by founding and supporting 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.

In 2019, Dr. Kakkis joined the board of directors of Odylia Therapeutics, a nonprofit biotech with the goal of accelerating the development of gene therapies for people with rare inherited diseases, changing the way treatments are brought from the lab to the clinic. In the same year, Dr. Kakkis received BIO’s Henri Termeer Visionary Leadership award for this transformative work in rare diseases and a Lifetime Achievement Award from the National MPS Society.

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.