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3-year follow up data in 15 patients receiving RPE-65 gene therapy treatment for LCA

A clinical report on the 3-year follow up data for a gene therapy treatment in patients with Leber’s Congenital Amaurosis (LCA) has concluded the therapy to be “sufficiently safe and substantially efficacious”. The study, led by research groups from the Scheie Eye Institute, University of Pennyslvania and the Center for Gene Therapy, University of Florida, reported on the main outcome measures from an open label dose escalation phase I study of 15 patients. Initial study results were reported contemporaneously with two independent LCA trials in 2008 while the current study provides relatively long-term follow up with clinical data that supports the continued development of gene therapy treatments for retinal degenerations.