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Jean Bennett, M.D., Ph.D. - "From DNA to FDA: Experiences in Translational Research for Inherited Blindness"

University of Pennsylvania, Philadelphia, USA
When Oct 23, 2017
from 12:00 PM to 01:30 PM
Where Tigem Auditorium "Vesuvius"
Contact Name
Contact Phone 081-19230659
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Abstract
Gene therapy has the potential to reverse disease or prevent further deterioration in patients with incurable degenerative diseases. Studies more than 2 decades in the making, including Phase 1 studies done in collaboration with investigators at TIGEM and Second University of Naples, now nearly 10 years post initiation, showed safe recovery of retinal/visual function in children and adults with congenital blindness due to RPE65 mutations. In a Phase 3 gene therapy trial carried out at The Children’s Hospital of Philadelphia (CHOP) and at the University of Iowa, a total of 29 participants received bilateral injection of the test reagent with nine participants having been randomly assigned to a control group receiving intervention one year later. Eighteen of 20 of the individuals assigned to the early intervention group improved on the primary outcome measure, a multi-luminance mobility maze as shown by read-out of year one measures and 13 passed the test at the lowest light level. None in the untreated comparison group of nine patients improved. Individuals in the control group then received the intervention. They also showed improvement (again with read-out at the year one timepoint). The improvements persisted through the 3 year timepoint (with follow-up ongoing) and there were also improvements in secondary measures. The results answer many questions about the potential benefits and risks of subretinal gene therapy and pave the way for development of additional retinal gene therapy studies. The presentation will include experiences and observations from the Phase 3 studies and speculation about development of retinal gene therapy in the future.

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