Dr. Kakkis is Chief Executive Officer, President and a Director of Ultragenyx. He founded Ultragenyx in 2010 to create a company that is uniquely built around a deep and meaningful engagement with patients and their caregivers to fully understand their needs. This includes designing a novel development model that fundamentally changed the established paradigms in clinical protocols, endpoints and analyses, as well as challenging traditional beliefs around commercialization and access to therapies for patients with rare and ultra-rare genetic diseases.
Dr. Kakkis began at the Harbor-UCLA Medical center where he was developing an enzyme replacement therapy for the rare disorder MPS I. He joined Biomarin in 1998 where he guided the development and approval of three treatments for rare diseases (MPS I, MPS VI and PKU) and contributed to the development of approved or development stage products for four other rare diseases (CLN2, 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.
Dr. Kakkis received the Life Science Leadership Pantheon award from California Life Sciences, a Lifetime Achievement Award from the National MPS Society, the Roscoe O. Brady Award for Innovation and Accomplishment from the WORLDSymposium, and BIO’s Henri Termeer Visionary Leadership award for this transformative work in rare diseases.
Dr. Kakkis serves on the Board of the EveryLife Foundation.
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 upon graduation. He completed a pediatrics residency and a Medical Genetics Training Fellowship at Harbor-UCLA Medical Center, where he was an assistant professor of pediatrics and initiated the enzyme therapy program for MPS I. Dr. Kakkis was board certified in pediatrics and medical genetics.
“I’ve had the opportunity to support families touched by rare diseases for more than two decades. I have been present when the first patients were successfully treated with new medications for conditions that previously had no therapies, and those moments have deeply impacted me and are part of the reason I founded Ultragenyx. At Ultragenyx, our goal is to create those moments for more patients with rare diseases.”