James M. Wilson, MD, PhD to receive the 2025 Roscoe O. Brady Award
Dr. Wilson is President and CEO of GEMMA Biotherapeutics (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.
About the WORLDSymposium™ Roscoe O. Brady Award
The Roscoe O. Brady Award is given annually by WORLDSymposium to recognize one individual for outstanding innovation and accomplishment in the field of lysosomal disease research and therapy. Dr. Brady was the third recipient of the “Award for Innovation and Accomplishment.” After decades of research on the biology and biochemistry of lysosomal diseases, Dr. Brady undertook experimental administration of exogenous proteins to replace the patients’ genetic enzyme deficiencies. His ongoing work led to many discoveries in the field, mentoring and inspiring numerous other investigators.
In the process of treating three patients with Gaucher disease, biomarkers were discovered and validated. His work ultimately resulted in FDA approval (1991) and commercialization of the first “enzyme replacement therapy” (ERT). This was an intravenous biologic medication alglucerase for Gaucher disease, the human enzyme purified from massive quantities of normal human placenta. This landmark accomplishment was followed by approval of imiglucerase made using recombinant DNA technology. Later in his career, Dr. Brady investigated substrate depletion treatment, molecular chaperone therapy, and gene therapy for patients with lysosomal disorders.
After a lifetime (October 11, 1923 – June 13, 2016) with decades of work in the field, and with the approval from his family, the WORLDSymposium award for innovation and accomplishment was renamed in his honor at the WORLDSymposium 2018 meeting.
Nominations are solicited annually, from which one individual is selected by the Awards Committee. The awardee is announced in the February “Lysosomes Issue” of Molecular Genetics and Metabolism and is invited to accept the award and address the audience with a featured presentation at the annual WORLDSymposium meeting.
Award Nominations Deadline: September 3, 2024.
Past Award Recipients:
2004 William Krivit, MD, PhD
2006 Henri A. Termeer
2008 Roscoe O. Brady, MD
2009 Elizabeth F. Neufeld, PhD
2010 William S. Sly, MD
2011 Steven U. Walkley, PhD
2012 John J. Hopwood, PhD
2013 Robert J. Desnick, PhD, MD
2014 Gregory A. Grabowski, MD
2015 Stephen C. Groft, PharmD
2016 Emil D. Kakkis, MD, PhD
2017 Konrad Sandhoff, PhD
2018 Mark E. Haskins, VMD, PhD
2019 Danilo A. Tagle, PhD, MS
2020 John F. Crowley
2021 Ellen Sidransky, MD
2022 Stuart A. Kornfeld, MD
2023 William A. Gahl, MD, PhD
2024 Elsa Shapiro, PhD