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Breakthroughs in Genetic Medicine

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Folkman Auditorium

Enders Building, Boston Children’s Hospital

320 Longwood Avenue

Boston, MA 02115

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James Wilson, M.D. Ph.D., Rose H. Weiss Orphan Disease Center, University of Pennsylvania
Delivery Challenges of In Vivo Gene Therapy and Genome Editing

James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. 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.

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Folkman Auditorium

Enders Building, Boston Children’s Hospital

320 Longwood Avenue

Boston, MA 02115

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