Speaker Profile

M.D., Ph.D., President & CEO, Ultragenyx

A medical geneticist by training, Dr. Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO at Ultragenyx, a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases. The company has three approved therapies and holds ex-US rights to a fourth approved product, and a deep pipeline of potential treatments across a variety of therapeutic modalities including gene therapy, mRNA therapy, biologics, and small molecule. Before founding Ultragenyx in 2010, Dr. Kakkis guided and contributed to the development of multiple approved rare disease products as chief medical officer at BioMarin Pharmaceutical. Dr. Kakkis also founded and serves on the Board of EveryLife Foundation for Rare Diseases, a non-profit dedicated to accelerating innovation for rare diseases. Dr. Kakkis has been recognized for his leadership by Biotechnology Innovation Organization (BIO), the National MPS Society, WORLDSymposium, and California Life Sciences.