AGTC Announces Data Evaluating the Natural History of Patients With X-Linked Retinoschisis at 48th Annual Retina Society Meeting

Applied Genetic Technologies Corporation

today announced that data from a study evaluating the natural history of X-linked retinoschisis (XLRS) will be presented at The Retina Society 48th Annual Scientific Meeting, taking place in Paris, France from October 7-11. "These results provided useful information to inform the design of AGTC's ongoing phase 1/2 clinical trial to evaluate a candidate AAV-based gene therapy product for treating XLRS, a rare inherited retinal disease that can lead to a significant reduction in visual acuity and other serious complications," said Sue Washer, President and CEO of AGTC. "We recently reached an enrollment milestone in our dose escalation study and plan to share initial topline safety data from the first treatment cohorts in the coming months." The presentation, entitled "A Natural History Study of Subjects with X-linked Retinoschisis in Anticipation of a Phase I/II Gene Therapy Trial," included results from 55 participants with XLRS and confirmed mutations in the retinoschisin (RS1) gene. Patients were evaluated on cross sectional and longitudinal measures of visual acuity, visual field sensitivity, microperimetry, cyst