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Tuesday, June 26 • 4:15pm - 5:30pm
#285: Gene Therapy: Advances in Translating Technology

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-613-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

Identify recent advances in the field of gene therapy; Discuss the existing regulatory framework.


Peter W. Marks


How Has Adeno-Associated Virus (AAV) Evolved?
Terry Flotte, MD

Update on the Development of LentiGlobin for Beta-Hemoglobinopathies
David Davidson, MD

Advancing Genome Editing Into the Clinic
Derek Jantz, PhD

Michael Havert, PhD

avatar for David Davidson

David Davidson

Chief Medical Officer, bluebird bio, Inc.
Dave Davidson, M.D. has served as CMO at bluebird bio since 2012. Previously, he conducted clinical research at Genzyme and GelTex. He completed clinical and research fellowships in infectious diseases at the Harvard Longwood Combined Infectious Diseases Program, and completed an... Read More →

Terry Flotte

Dean, Provost and Executive Deputy Chancellor, University of Massachusetts Medical School
Provost & Executive Deputy Chancellor, Celia & Isaac Haidak Professor of Medical Education & Dean of the UMass Medical School. Internationally known pioneer in human gene therapy, He is currently investigating the use of gene therapy for genetic diseases, including alpha-1 antitrypsin... Read More →
avatar for Michael Havert

Michael Havert

Biologist/CMC Reviewer, Gene Therapy Branch, OTAT, CBER, FDA
Mike has been a CMC Reviewer in the Gene Therapy Branch since 2004. Gene Therapies are a diverse group of new technologies that include genetically modified viruses and cells. Mike’s recent accomplishments include serving as BLA Chair for a genetically modified autologous T-cell... Read More →

Derek Jantz

Chief Scientific Officer, Co-Founder, Precision Biosciences
Derek Jantz, PhD co-founded genome editing company Precision BioSciences in 2006 and serves as Chief Scientific Officer. He has a BA in biology from the University of Colorado, a PhD in Biophysics from the Johns Hopkins University School of Medicine, and completed a postdoctoral fellowship... Read More →
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA, United States
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →

Tuesday June 26, 2018 4:15pm - 5:30pm EDT
Room 156ABC Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  06: PreClin Dev-EarlyPhaseCR, Session