Kathy High, MD, a pioneer in gene therapy and former president of Spark Therapeutics, shares her inspiring journey from working with hemophilia to advancing retinal disease treatments. Joined by Hans-Peter Kiem, MD, PhD, they delve into the challenges of developing gene therapies, including the complexities of AAV vectors and the hurdles of clinical trials for children. They also discuss recent innovations like synthetic AAVs, the importance of collaboration in biotech research, and offer wisdom for early-career hematologists pursuing their passions.

Terry Flotte, MD, a leading figure in gene therapy and Dean at UMass Chan Medical School, shares his journey from childhood inspiration to groundbreaking research. He discusses the pioneering work in AAV gene therapy for cystic fibrosis, emphasizing safety and technological evolution. Alongside ASGCT President Hans-Peter Kiem, MD, PhD, they explore the future of gene therapy, touching on exciting advancements aimed at treating genetic disorders. Flotte's passion for pediatrics and personal interests, including baseball and obscure films, add a delightful touch.