Speaker Profile
PMWC PIONEER AWARD
Groundbreaking work in viral vector-based gene therapies, significantly impacting the treatment of rare genetic diseases
M.D., Ph.D., President and CEO, Gemma Biotherapeutics
Biography
James M. Wilson, MD, PhD, is the President and CEO of Gemma Biotherapeutics (GEMMABio) and Chair of Franklin Biolabs. Previously, he served as 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. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published 600 papers and is named on more than 1200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. More recently Dr. Wilson's laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.
Session Abstract – PMWC 2025 Silicon Valley
Track Co-Chairs:
Yael Weiss, Mahzi Tx
Morten Sogaard, Astellas Gene Therapies
- PMWC 2025 Award Ceremony
Pioneer Honoree: James M. Wilson, Gemma Biotherapeutics
Luminary Honoree: Carolyn Bertozzi, Stanford
- Keynote: Bioorthogonal Chemistry, from Basic Science to Clinical Translation
- Carolyn Bertozzi, Stanford
- Keynote: The Promise of Genetic Medicines Unless You're Too Poor or Too Rare
- James M. Wilson, Gemma Biotherapeutics
- Barriers in Gene Therapy
Chair: Timothy Hunt, Alliance for Regenerative Medicine
- Tay Salimullah, 4BIO Capital
- James M. Wilson, Gemma Biotherapeutics
- Courtney Silverthorn, Bespoke Gene Therapy Consortium (BGTC) - Next-Generation Delivery Systems for Gene Therapies (PANEL)
Chair: James M. Wilson, Gemma Biotherapeutics
- Mathieu Nonnenmacher, Voyager Therapeutics
- Kevin Friedman, Kelonia Therapeutics
- Kunwoo Lee, GenEdit
- Haig Aghajanian, Capstan Therapeutics
- Beyond the Genome: Harnessing Epigenetics in Rare Disease Treatment (PANEL)
Chair: Charles Gersbach, Duke
- Prashant Mali, UCSD
- Nadav Ahituv, UCSF
- Blythe Sather, Tune Therapeutics
- Fyodor D Urnov, Berkely University
- Navigating the Regulatory Landscape for Gene and Cell Therapies (PANEL)
Chair: Yael Weiss, Mahzi Tx
- Rob Califf, FDA
- Courtney Silverthorn, Bespoke Gene Therapy Consortium (BGTC)
- Ben Dewees, Kyverna Therapeutics
- Paul Kruszka, GeneDx
- AI-driven Improvements in Gene and Cell Therapy (PANEL)
Chair: John Androsavich, Ginkgo Bioworks
- Francois Vigneault, Shape Therapeutics
- Eeshit Dhaval Vaishnav, Sequome
- Sam Sinai, Dyno Therapeutics
- Kira Radinsky, mana.bio
