Ultragenyx Ventures into Gene Therapy for Wilson Disease

Ultragenyx Pharmaceutical is committing resources to study the potential of gene therapy in Wilson disease. Laboratory research on animals suggests its gene therapy can reduce liver disease. The next is to begin human trials to test the safety of its gene therapy and determine if it works. This investigational trial will being in 2021.
Emil D. Kakkis, M.D., Ph.D.
CEO, President and Founder, Ultragenyx Pharmaceutical Inc.
Novato, CA
Dr. Kakkis is Chief Executive Officer, President and Founder of Ultragenyx Pharmaceutical where he leads a team developing and commercializing multiple rare and ultra-rare disease treatments. Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and for advocating on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases. 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, Naglazyme for MPS VI and Kuvan for PKU and has contributed to the development for four other treatments for rare diseases (Vimizim for MPS IVA, Brineura for CLN2, Palenziq for PKU and VOSORITIDE for achondroplasia). Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing more therapeutics for rare and ultra-rare diseases. Since its founding, Ultragenyx has worked on developing treatments for twenty different genetic diseases and has now received four approvals for Crysvita® for XLH and for TIO, Mepsevii® for MPS VII, and Dojolvi for LC-FAOD. Dr. Kakkis graduated from Pomona College, magna cum laude in 1982 with the Vaile Prize for his research and he received his combined M.D. and Ph.D. degrees om 1989 from the UCLA (MSTP) with the Emil Bogen Prize for his research. He completed both a Pediatrics residency and Medical Genetics Training Fellowship at Harbor-UCLA Medical Center where he began work on MPS I. In 2016, Dr. Kakkis received the Roscoe O. Brady Award for Innovation and Accomplishment from the WORLD lysosomal organization and in 2019, he received the Henri A. Termeer Biotechnology Visionary Award from BIO, the biotechnology industry organization.