James M. Wilson, MD, PhD

Having started his work in gene therapy nearly 40 years ago, Dr. Wilson was recruited to the University of Pennsylvania in 1993 and created the first and largest academic-based program in gene therapy. His laboratory discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These "vectors" have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.

Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of eight biotechnology companies. His research has been focused on rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of neurologic disorders and liver metabolic diseases, and on addressing these unmet needs for patients in marginalized populations. To date, his team’s accomplishments include 3 FDA-approved AAV-based gene therapies, 40+ active programs in development, 95 patents on gene therapy-related technologies, and first-in-patient studies in 15 different diseases.