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About Us

Scientific Advisors

Michel Vellard, Ph.D.

Dr. Michel Vellard, Ph.D., has served as a scientific advisor to Gain since November 2018. Dr. Vellard has more than 25 years of experience in molecular and cellular biology. He currently serves as chief scientific officer for Home Biosciences. He has also developed his career as co-founder and chief scientific officer at Audacity Therapeutics, vice president of research at Ultragenyx Pharmaceuticals and head of lysosomal biology, previously director of research and development, principal scientist at BioMarin where 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 enzyme replacement therapy (ERT) for Morquio syndrome, the latest (VIMIZIM) has been approved in 2014. As a postdoctoral fellow, Dr. Vellard worked on rare diseases specifically researching the gene responsible for lysosomal disease cystinosis. Dr. Vellard studied extensively with Emil Kakkis at Harbor, specifically on lysosomal diseases and ERT. Dr. Vellard holds multiples patents and has authored and co-authored many research publications. Dr. Vellard received his B.S. in Natural and Life Sciences and M.S. in Molecular and Cellular Genetics from the University of Lyon I. He received his Ph.D. in Virology Pasteur from University of Paris VI, VII, XI.

 

 
 
 

Research and Development Collaborations

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Grants

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Patient Associations

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Centers of Research, Translational Science & Clinical Excellence

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