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Gene Therapy: Advances in Translating Technology
Session Chair(s)
Peter W. Marks, MD, PhD
Director, Center for Biologics Evaluation and Research
FDA, United States
Gene therapy is now a reality in the clinic with additional products working toward regulatory approval. Advances in gene editing are expanding the boundaries of what was previously thought possible for gene therapy on somatic cells. However, challenges remain in translating scientific advances into clinical progress. This session will discuss/review recent advances in the field, discuss the existing regulatory framework, and provide perspectives from several different sponsors developing products.
Learning Objective : Identify recent advances in the field of gene therapy; Discuss the existing regulatory framework.
Speaker(s)
How Has Adeno-Associated Virus (AAV) Evolved?
Terry Flotte, MD
University of Massachusetts Medical School, United States
Dean, Provost and Executive Deputy Chancellor
Update on the Development of LentiGlobin for Beta-Hemoglobinopathies
David Davidson, MD
bluebird bio, Inc., United States
Chief Medical Officer
Advancing Genome Editing Into the Clinic
Derek Jantz, PhD
Precision Biosciences, United States
Chief Scientific Officer, Co-Founder
Panelist
Michael Havert, PhD
StrideBio, United States
Vice President, Regulatory Affairs
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