Stephanie Cherqui is Professor in the Department of Pediatrics at the University of California San Diego, Director of the UCSD Gene Therapy Initiative, and co-founder of Papillon Therapeutics. Her laboratory primarily centers on the development of hematopoietic stem cell (HSC) gene therapy-based treatments for genetic disorders. Her work led to the first-in-human HSC gene therapy clinical trial for cystinosis. She is applying this strategy to other disorders including the neurodegenerative diseases, Friedreich’s ataxia, Sanfilippo C, and Alzheimer’s disease. Dr. Cherqui is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium, the Chair of the Scientific Review Board of the Cystinosis Research Foundation, and a member of the ASCGT Gene and Cell Therapy of Genetic and Metabolic Diseases committee.