Case study: developing an ex vivo gene-modified hematopoietic stem cell treatment for a rare monogenic disease in a novel mouse model 

Rafael Andrés Badell-Grau graduated in Biochemistry from Cardiff University in the United Kingdom. He also obtained both a Master’s and a PhD in Biosciences from the same institution, specializing in the development of drugs for the treatment of neurodegenerative diseases (with the works: “The role of the lysosome in Huntington’s disease pathogenesis: development of a new clinical therapy", and "Brug screening for lysosomal storage diseases with a focus on CLN8 disease", respectively). In 2016, he was awarded the Best Student Award in his Master's program for outstanding performance in his cohort.

Dr Badell-Grau then worked at OxVent, a med-tech start-up spun out of the University of Oxford and King’s College London, which focused on producing low-cost mechanical ventilators in response to the COVID-19 pandemic. He is currently a postdoctoral fellow at the University of California, San Diego, sponsored by the Cure Sanfilippo Foundation and the National MPS Society. His research at UC San Diego is focused on the development of cell and gene therapies for lysosomal storage diseases, specifically MPS IIIC (Sanfilippo Syndrome Type C). During his postdoctoral work, he has received two Outstanding Poster Awards and one Travel Award from the American Society of Gene and Cell Therapy.