Dr. James Wilson, Director of Penn's Orphan Disease Center


James M. Wilson, MD, PhD, is the Director of the Orphan Disease Center at the University of Pennsylvania. 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. He initially focused on the clinical translation of existing gene transfer technologies but soon redirected 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.  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. Dr. Wilson was noted by the journal Nature Biotechnology to be the “second most productive bio-entrepreneur in life sciences.”