A return to rational capsid design? Predicting the future of AAV vector R&DPublished: May 15, 2020
Trends and advances in gene therapy delivery and gene editing
R. Jude Samulski
R JUDE SAMULSKI received his PhD in Medical Microbiology and Immunology from the University of Florida. His graduate work (1978–82) involved the cloning of the adeno-associated virus (AAV) genome and the demonstration of AAV as a viral vector, including the first US patent involving non-AAV genes inserted into AAV. During his post-doctoral training at Princeton, he developed the AAV 2 ITR vector backbone, commonly used by most labs today as well as the initial establishment of an AAV production system. At the University of Pittsburgh Department of Biology, he was the first to demonstrate AAV transduction in rodent brain and muscle that culminated in the first clinical trials in the brain (Canavan) and muscle (DMD). In 1993, he was hired at the University of North Carolina (UNC) to establish a Gene Therapy Center. For over 25 years, Dr Samulski, as a Professor of Pharmacology and the Director of the Gene Therapy Center at UNC, has led a team of multiple Principal Investigators developing novel viral vectors and clinical gene therapy programs. He was recognized in 2008 by the American Society of Gene & Cell Therapy (ASGCT) as the first recipient of the Outstanding Achievement Award, was awarded the National Hemophilia Foundation’s Investigator of the Year in 1999 and was the first non-MD to be placed on the University of Florida’s Wall of Fame. He has served as past President of ASGCT and was invited to China to meet with the Chinese Minister of Health and soon after was recognized as one of China’s Thousand Points of Light, a recognition bestowed on individuals whose contributions are benefiting mankind. In addition to being the lead inventor on over 300 patents in the field of AAV vectors and gene therapy, he is a scientific founder of ASGCT, Merlin, Asklepios BioPharmaceutical, NanoCor Therapeutics, Chatham Therapeutics, Bamboo Therapeutics, Viralgen, and other entities that continue to advance the field of human gene therapy and was selected as a seminal speaker at the Royal Society of Science in London in the Isaac Newton Lecture room on ‘Delivering novel therapeutic in the 21st century’ (October 24, 2018).
Citation: Cell & Gene Therapy Insights 2020; 6(4), 533–541