Emil D. Kakkis

Dr. Emil D. Kakkis is best known for his work over the last 18 years to develop novel treatments for rare disorders. He began his work developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I, with minimal funding and support. The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of the Ryan Foundation, a patient organization formed by Mark and Jeanne Dant for their son Ryan. Aldurazyme development was later supported by BioMarin Pharmaceutical and eventually their partner Genzyme leading to FDA approval in 2003. During his tenure at BioMarin, Dr. Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU, and has contributed to the initiation of 7 other treatment programs for rare disorders, three of which are now in clinical development. After 11 years at BioMarin, Dr. Kakkis left industry to initiate an effort to improve the regulatory and clinical development process for rare diseases. In early 2009, Dr. Kakkis launched and funded the Kakkis EveryLife Foundation to accelerate biotech innovation for rare diseases. The Foundation initiated a campaign to improve the regulatory and clinical development process for rare diseases. In just over a year, 160 patient organizations and physician society partners have endorsed the Campaign. Dr. Kakkis has founded Ultragenyx™ to return to development of drugs for rare diseases. For many rare diseases, reasonable science exists that needs to get translated to patients. He will build on his previous experiences and will assemble an experienced team to efficiently develop treatments for rare diseases.