A medical geneticist by training, Dr. Emil Kakkis is a pioneer in the development and commercialization of treatments for rare diseases. He is founder and CEO of 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, World Orphan Drug Congress and California Life Sciences.
In his first book, Saving Ryan, (published June 21,2022) Dr. Kakkis captures the passionate drive and impossible odds faced in order to develop a treatment to save a young boy’s life diagnosed with a disease so rare that no one would ever consider working on it, even though the science existed to treat it.