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Breakthroughs in Genetic Medicine

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Folkman Auditorium

Enders Building, Boston Children’s Hospital

320 Longwood Avenue

Boston, MA 02115

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Jean Bennett, M.D. Ph.D., Center for Advanced Retinal and Ocular Therapeutics (CAROT), University of Pennsylvania

From DNA through FDA: Experiences in Translational Research for Inherited Blindness

Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology, Professor of Cell and Developmental Biology, and Co-Director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania Perelman School of Medicine, is a physician-scientist with experience/expertise in molecular biology, vector development and gene therapy translational studies. She has developed gene transfer approaches to test treatment strategies for retinal degenerative and ocular neovascular diseases, to elucidate retinal differentiation pathways and to identify pathogenetic mechanisms that lead to blindness. Her research, conducted at UPenn over the past 26 years, has established the scientific underpinnings which made it possible to test the first definitive retinal gene therapy treatment for patients with blinding retinal degenerations. Her lab has established a true “from bench to bedside” program, and thus she is familiar with steps necessary to go from proof-of-concept all the way to those necessary for testing of safety and efficacy in humans with blinding disease, including obtaining the appropriate molecular diagnoses.

Dr. Bennett was the scientific leader of a team that translated reversal of blindness in animal models to demonstration of efficacy and safety in children and adults. Her team was the first to enroll pediatric subjects with a non-lethal disease as gene therapy participants. The team completed both a Follow-on (re-administration) study and a Phase 3 registration gene therapy trial for congenital blindness, the first randomized, controlled, multi-center gene therapy Phase 3 gene therapy trial targeting a genetic disease. This work led to the first approved gene therapy drug for retinal disease worldwide and the first US FDA-approved recombinant virus-based gene therapy product to be delivered directly into a person.

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Folkman Auditorium

Enders Building, Boston Children’s Hospital

320 Longwood Avenue

Boston, MA 02115

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