James M. Wilson, MD, PhD received the 2025 Roscoe O. Brady Award

Dr. Wilson is President and CEO of GEMMABio. 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 focused on 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 ten 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, approximately 20 active programs currently in development, 95 patents on gene therapy-related technologies, and first-in-patient studies in 15 different diseases. He is Founder and Advisor for Scout Bio, Passage Bio, and iECURE, and Executive Chair at Franklin Biolabs.

Congratulations to Dr. Wilson from the entire WORLDSymposium audience! The 2025 Roscoe O. Brady Award was presented on Tuesday, February 4, at 08:00 PST, followed by a scientific presentation by Dr. Wilson: Lysosomal Diseases: A paradigm for personalized medicine.