Dr. Samulski received his Ph.D. in medical microbiology and immunology from the University of Florida. His graduate work (1978-82) demonstrated the first use of AAV2 as a viral vector which culminated in the first U.S. FDA approved gene therapy drug for Leber Congenital Amaurosis (LCA). After completing post-doctoral training at Princeton, Dr. Samulski was hired at University of Pittsburgh where his lab demonstrated the first use of AAV for gene delivery to the CNS which eventually led to the initial treatment of AAV for Canavan’s disease. In 1993, he was recruited to University of North Carolina (UNC) to initiate, build and direct the UNC Gene Therapy Center. In this setting, development of self-complementary AAV vectors (currently used in approved SMA gene therapy drug), Chimeric AAV capsids (first example of synthetic vector used in humans - DMD trial), and critical research that enabled large scale production was carried out. Research headed by Dr. Xiao Xiao, Ph.D., demonstrated the first AAV-mediated long-term gene transduction in muscle (J. Virology, 1996). This finding precipitated efforts to develop 1st generation AAV vectors to treat Duchenne muscular dystrophy that is now in Phase 3 clinical trials.
In total, Dr. Samulski has worked with AAV for 40 years, and for 25 years, he was director of the University of North Carolina Gene Therapy Center. He was the scientific founder of Bamboo Therapeutics, Inc. and served as the Chief Scientific Officer and Executive Chairman of the company until its acquisition by Pfizer in 2016. Upon its acquisition, Dr. Samulski joined Pfizer, as VP Gene Therapy, to ensure the successful transition of Bamboo’s Duchenne muscular dystrophy therapeutic. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Cell and Gene Therapy with their inaugural Outstanding Achievement Award for his work and later served as President of the Society. He was also invited by Pope Francis to the Vatican in recognition for his work in the treatment of Canavan’s disease.
Dr. Samulski has advanced therapeutics into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and congestive heart failure, to name a few. He has published more than 450 articles and holds more than 300 patents related to AAV technology. Currently, Dr. Samulski is professor in the Department of Pharmacology at UNC and founder and member of Columbus Children’s Foundation that has focused on developing AAV gene therapies for “ultra-rare” disorders.