The 49th annual Carl Moore Memorial Lecture was held on November 8, 2023 with invited speaker Katherine High, M.D., discussing her pioneering work developing gene therapy approaches for genetic diseases. The lectureship is in honor of Dr. Carl Moore, a renowned physician-scientist who established the Division of Hematology and later served as Chair of the Department of Medicine. The lectureship was established after his death to honor his belief in the important role of physician-scientists in biomedical research. Dr. High is currently visiting Professor, Rockefeller University, and William Bennett Professor Emerita, Perelman School of Medicine of the University of Pennsylvania, Founding Director, Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia and Founder, Spark Therapeutics. She led groundbreaking work developing adeno-associated viral-mediated gene therapy approaches for hemophilia and other diseases, and her efforts have facilitated rapid translation of preclinical discoveries into clinical application.