Speaker Profile
Ph.D., Vice President, Strategic Alliances and Innovation, FNIH
Biography
Dr. Courtney Silverthorn is a distinguished professional with a robust background in science policy and technology transfer. Currently, she oversees business development opportunities for the Foundation for the NIH. Dr. Silverthorn has also played a pivotal role in the Bespoke Gene Therapy Consortium, a collaborative project focused on accelerating the development of gene therapies for diseases that currently have no commercial interest. Her contributions to this consortium underscore her commitment to advancing treatments for rare diseases.In addition to her work at FNIH and prior roles at several federal agencies, Dr. Silverthorn has shared her insights on platforms such as the Rare Disease Advisor Podcast. In an interview with Larry Luxner, she discussed the consortium's impact on rare diseases, highlighting her expertise in scientific partnerships and innovation. Dr. Silverthorns dedication to enhancing healthcare through scientific research and collaboration continues to make her a significant figure in the field.
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, University of Pennsylvania
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, University of Pennsylvania
- Reimbursement for Genetic Medicines in Rare Diseases
Chair:
- Erin Satterwhite, Optum Specialty Pharmacy
- Next-Generation Delivery Systems for Gene Therapies (PANEL)
Chair: Morten Sogaard, Astellas
- Mathieu Nonnenmacher, Voyager Therapeutics
- Kevin Friedman, Kelonia Therapeutics
- Kunwoo Lee, GenEdit
- Haig Aghajanian, Capstan Therapeutics
- James M. Wilson, University of Pennsylvania
- 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
- Courtney Silverthorn, Bespoke Gene Therapy Consortium (BGTC)
- Peter Marks, FDA
- Ben Dewees, Kyverna Therapeutics
- AI-driven Improvements in Gene and Cell Therapy (PANEL)
Chair: Morten Sogaard, Astellas
- Francois Vigneault, Shape Therapeutics
- John Androsavich, Ginkgo Bioworks
- Yogev Debbi, mana.bio
