Cell & Gene Therapy of Genetic and Metabolic Diseases

This committee is broadly focused on identifying and disseminating the most contemporary therapeutic strategies to treat genetic and metabolic disorders. Inborn errors of metabolism including PKU, urea cycle defects, lysosomal storage disorders, organic acidemias, glycogen storage disorders, diabetes, and hemophilia are some of the conditions that have traditionally been under our purview. Because genetic and metabolic disorders serve as important proof of concept targets for traditional and novel treatment approaches, the committee has remained agnostic of methodologies and focused on assessing submitted work based on results and impact, fully embracing viral and non-viral platforms, genome editing, and stem cell therapies.