On Wednesday, December 15, the NIH Wednesday Afternoon Lecture Series will feature Dr. Jean Bennett, Professor of Ophthalmology and Cell and Developmental Biology at the University of Pennsylvania. The tile of her presentation will be “Seeing is Believing: A Gene Therapy Success.” The lecture will be held at 3:00 p.m. in NIH Clinical Center (Building 10), Masur Auditorium.
Dr. Bennett will explain how gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. She is currently leading a gene therapy trial on Leber congenital amaurosis (LCA) due to RPE65 mutations at The Children’s Hospital of Philadelphia (CHOP). This Phase 1-2 study is now more than 3 years past initiation. The demonstration of safe and stable recovery of retinal/visual function in 12 children and adults in the CHOP trial and encouraging results from four other clinical trials for LCA-RPE65 provide great hope for people with other more common blinding diseases.
Her presentation will describe the animal studies that led to the clinical trials and the latest safety and efficacy results in the LCA-RPE65 clinical trial being held at CHOP. In addition, Dr. Bennett will convey some of the challenges presented by the nature of the targeted disease itself, hurdles that have been navigated in order to conduct the study, and issues that impact the eventual approval of gene augmentation as a therapy for LCA and other blinding diseases.