Enabling Gene Editing Platforms for Novel In Vivo and Ex Vivo Therapeutics
Friday, May 31, 2024 09:15 AM - 10:15 AM
Room 212-214
Concurrent Session
Chair
Genome editing technologies has enabled modification of DNA sequences with unprecedented precision and efficiency. Together with efficient platforms for ex vivo and in vivo delivery into cell types of interest, it offers an exciting possibility of tailored medicine to effect lasting curative therapies. Recent advances in the technology landscape have paved the way for rapid development of new medicine for any genetic disease. This session brings together pioneers in the field who will share important perspectives on developing and delivering these novel approaches that navigate current manufacturing, clinical and regulatory challenges to achieve affordable therapies.
- Uma Lakshmipathy, PhD, Pharma Services, Thermo Fisher, United States
- Bruce Levine, PhD, University of Pennsylvania, United States, "The Next Chapter in the Fantastic Voyage of CAR T Cells"
- Fyodor Urnov, PhD, University of California Berkeley, United States, "A Platform for CRISPR Cures: a First-In-Class Academia-Industry Partnership to Treat Inborn Errors of Immunity"
- Laura Sepp-Lorenzino, PhD, Intellia Therapeutics, United States, "Clinical Advances in In Vivo CRISPR Editing"
Genome editing technologies has enabled modification of DNA sequences with unprecedented precision and efficiency. Together with efficient platforms for ex vivo and in vivo delivery into cell types of interest, it offers an exciting possibility of tailored medicine to effect lasting curative therapies. Recent advances in the technology landscape have paved the way for rapid development of new medicine for any genetic disease. This session brings together pioneers in the field who will share important perspectives on developing and delivering these novel approaches that navigate current manufacturing, clinical and regulatory challenges to achieve affordable therapies.
Uma Lakshmipathy
Senior Director R&D, Site Head Patheon Translational Services
Thermo Fisher Scientific
Chair
Senior Director R&D, Site Head Patheon Translational Services
Thermo Fisher Scientific
Chair
Bruce Levine
Barbara and Edward Netter Professor in Cancer Gene Therapy
University of Pennsylvania
Speaker
Barbara and Edward Netter Professor in Cancer Gene Therapy
University of Pennsylvania
Speaker
Laura Sepp-Lorenzino
Chief Scientific Officer, Executive Vice President
Intellia Therapeutics
Speaker
Chief Scientific Officer, Executive Vice President
Intellia Therapeutics
Speaker