NC Award Science RJ Samulski Thomas Shenk

Lewis Thomas Laboratory
Princeton University
Princeton, NJ 08544-1014

April 8, 2021

To Whom It May Concern:

I write with great enthusiasm to support the nomination of Professor Richard Jude Samulski for the 2021 North Carolina Award.

Professor Samulski has pioneered and led the development and utilization of adeno-associated virus (AAV) as a vector for human gene transfer to treat genetic disorders. He was first to clone the AAV genome, and he went on to delineate numerous details of its biology. He was the first to demonstrate long-term expression from an AAV vector in an animal, a feature critical to the utility of a gene delivery vehicle. He has led the field in AAV vector design, optimizing capacity and transduction efficiency and developing vector capsids with enhanced tissue targeting specificities. Following his pioneering designs, he has engineered a system to produce the vectors, enabling their use by many investigators in the clinic. He was instrumental in initiating gene transfer studies in humans for brain (Canavan Disease 2002), muscle (Duchenne Muscular Dystrophy DMD 2006), and bleeding disorders (Hemophilia 2012) – trials that helped to establish and significantly advanced the field of gene therapy. His work continues today as he plays a leading role in the development of AAV-based therapeutics to treat muscular dystrophy, Pompe disease, Parkinson's disease and congestive heart failure – all current clinical stage programs.

It is no exaggeration to say that Professor Samulski has revolutionized the field of gene therapy. His investigations of human gene transfer have driven the gene therapy field forward, motivating young scientists and physicians to enter the field. His discoveries have provided a tool kit for the transfer of therapeutic genes into humans. Samulski is internationally known for mentoring and outreach, and he has held impactful leadership positions, including director of the influential University of North Carolina Gene Therapy Center and president of the American Society for Gene and Cell Therapy.

Sincerely,

Thomas Shenk
Member, U.S. National Academy of Sciences
Member, U.S. National Academy of Medicine