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.