As a postdoctoral fellow, Dr. Michel Vellard started to work on rare diseases. In the pediatric department at UCLA Harbor Medical Center he researched the gene responsible for lysosomal disease cystinosis. Dr. Vellard studied extensively with Emil Kakkis at Harbor, specifically on lysosomal diseases and enzyme replacement therapy (ERT). For 14 years, Dr. Vellard worked at BioMarin in increasing scientific management capacities. During his tenure, he was involved with most of the products developed there, and managed several programs including TFEB (collaboration with TIGEM/A. Ballabio), Duchenne Muscular Dystrophy (Phase I) and particularly an ERT for Morquio syndrome, the latest (VIMIZIM) has been approved in 2014. Since 2014 he is the VP of Research at Ultragenyx, a company exclusively centered on rare genetic diseases.
Michel received his B.S. in Natural and Life Sciences and M.S. in Molecular and Cellular Genetics from the University of Lyon I, France. He obtained his Ph.D. in Virology, Pasteur, University of Paris VI, VII, XI, France. Michel holds multiples patents and has authored and co-authored many research publications.