“...two of the obstacles patients with rare disease encounter first are the high costs associated with manufacturing and with getting into first-in-human trials.diseases...”
Abi Pinchbeck, Editor, Cell & Gene Therapy Insights, speaks with Terence Flotte, Elisabeth Chair for the Dean of Medicine and Executive Deputy Chancellor of UMass Chan Medical School, past President of the American Society of Gene and Cell Therapy, about the origins of AAV-based gene therapy, the evolution of the field over three decades, the remaining safety and dose challenges, expanding beyond rare disease, and emerging initiatives to improve global patient access.
