September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
Ocular gene therapy: Clinical successes and lessons learned
Author Affiliations & Notes
  • Jean Bennett
    Center for Advanced Retinal and Ocular Therapeutics, University of Pennsylvania Scheie Eye Institute, Philadelphia, Pennsylvania, United States
    Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
  • Footnotes
    Commercial Relationships   Jean Bennett, Spark Therapeutics (F)
  • Footnotes
    Support  This presentation is made on behalf of the numerous investigators on the CHOP-UPenn-UIowa-Spark RPE65 study team, Dr. M. Ashtari at CHOP/UPenn, and European collaborators involved in the Phase 1 studies; We are grateful for funding from: The Children’s Hospital of Philadelphia, Spark Therapeutics, FFB, NEI/NIH grants R21EY020662, R24EY019861, and 8DP1EY023177, Clinical Translational Science Award NIH/NCRR UL1-RR-024134, RPB, the Mackall Foundation Trust, the Scheie Eye Institute, CAROT, the F.M. Kirby Foundation, the grants from the National Center for Research Resources, and the Howard Hughes Medical Institute
Investigative Ophthalmology & Visual Science September 2016, Vol.57, No Pagination Specified. doi:
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      Jean Bennett; Ocular gene therapy: Clinical successes and lessons learned. Invest. Ophthalmol. Vis. Sci. 2016;57(12):No Pagination Specified.

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      © ARVO (1962-2015); The Authors (2016-present)

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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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