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Jean Bennett; Ocular gene therapy: Clinical successes and lessons learned. Invest. Ophthalmol. Vis. Sci. 201657(12):.
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© ARVO (1962-2015); The Authors (2016-present)
Presentation Description :
Gene therapy has the potential to reverse disease or prevent further deterioration in patients with incurable degenerative diseases. The demonstration of safe and stable recovery of retinal/visual function in children and adults with congenital blindness due to RPE65 mutations in gene therapy trials being carried out at The Children’s Hospital of Philadelphia (CHOP) and at the University of Iowa provide great hope for people with other more common blinding diseases. The CHOP Phase 1-2 study is now >8 years past initiation and the multi-center Phase 3 (pivotal) trial is well underway. The latter study is the first randomized controlled Phase 3 gene therapy trial for genetic disease of any kind. The first set of results from the Phase 3 studies revealed robust improvements in retinal and visual function and in functional vision as well as a high degree of safety, thereby placing the recombinant adeno-associated virus reagent (AAV2-hRPE65v2) as the frontrunner for being the first approved gene therapy drug in the USA. This presentation will describe the challenges presented by the nature of the targeted disease itself, decisions that were made at various junctures of the studies and the modifications that were made in response to clinical findings. The net result is a path that can be adapted for future ocular gene therapy studies.
This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.
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