James M. Wilson, MD, PhD is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has been at the nexus of the field of gene therapy from its birth. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. He created the first and largest academic-based program in gene therapy after being recruited to Penn in 1993, initially focusing on the clinical translation of existing gene transfer technologies but soon redirecting his efforts to the development of second and third generation gene transfer platforms, the first of which was licensed to a biotechnology company he founded that resulted in the first, and only, commercially approved gene therapy in the western hemisphere.
More recently, his laboratory discovered a family of viruses from primates that could be engineered to be very effective gene transfer vehicles. Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of several biotechnology companies. He is currently leading a national dialogue on the challenges of commercializing these potentially lifesaving treatments due to the disruptive nature they will have on traditional business models. Throughout his career, the focus of Dr. Wilson's research has been rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of metabolic disorders. He is the founder of a 501(c)3 called Health Through Fitness in Orphan Diseases and Director of a bicycle team called Rare Disease Cycling whose participants compete at a national level and help raise money for rare disease research.
Dr. Wilson has published over 550 papers, reviews, commentaries and editorials in peer-reviewed literature and is an inventor on over 117 patents. He was the second President of the American Society of Gene Therapy, the 2014 recipient of the William Osler Patient Oriented Research Award of the University of Pennsylvania and 2015 recipient of the Scientific Achievement Award from Pennsylvania Bio. He was cited by the journal Nature Biotechnology as the "second most productive bio-entrepreneur in life sciences."