Samulski, a professor in the UNC School of Medicine’s pharmacology department and member of the UNC Lineberger Comprehensive Cancer Center, received the award recently at the society’s annual meeting in Boston. He was selected from a pool of 13 nominees from across the country.
The award recognizes an active member of the society who has achieved a pioneering research success, a specific high-impact accomplishment or a lifetime of significant scientific contributions to the field of gene therapy.
Samulski’s research focuses on the study of the human non-pathogenic parvovirus adeno-associated virus (AAV). He has long pioneered methodologies for using viruses to deliver genes effectively and safely to various targets in the body, including the brain, lungs, heart and muscle. As a graduate student at the University of Florida in the early 1980s, his thesis project was developing AAV as a vector for therapeutic genes.
This work eventually led to development of AAV type-2 as a viral vector, which has been used for gene therapy trials in cystic fibrosis, hemophilia, Parkinson’s disease, retinal disorders and in several other settings, including the first clinical trial of gene therapy for muscular dystrophy in the United States. That trial is currently under way, led by a university biotech start-up that Samulski co-founded with Xiao Xiao, Ph.D., who is now Fred Eshelman Distinguished Professor of Gene Therapy in the UNC School of Pharmacy molecular pharmaceutics division.
Samulski was recruited to UNC as director of the Gene Therapy Center in 1993. In addition to his work at UNC, Dr. Samulski has founded the Chapel Hill Project, a non-profit organization focused on facilitating gene therapy clinical trials for orphan diseases. Orphan diseases are life-threatening or chronically debilitating diseases affecting relatively few people. As a result, pharmaceutical companies do not generally invest significant funds toward their research and development and most sufferers lack sufficient treatments.