AGTC Completes Enrollment for Phase I/II Study of Gene Therapy for X-linked Retinoschisis

April 13, 2018: By Jon Swedien

Applied Genetic Technologies Corporation, in collaboration with Biogen, has completed enrollment for a Phase I/II trial of its gene therapy candidate for x-linked retinoschisis (XLRS), the Massachusetts-based company announced April 10.

The open-label, dose-escalation study is designed to assess the safety and efficacy of intravitreal administration of the AAV-based gene therapy. About 27 patients diagnosed with XLRS caused by mutations in the RS1 gene will participate in the study, AGTC said.

The company said it expects topline data by Q4-2018, with the final analysis to come at the 12-month time point.

Currently, there no FDA-approved treatment options for XLRS, a leading cause of macular degeneration in young men, AGTC said.

Trial participants were enrolled sequentially in groups. Participants in one group received a low-dose, in another group participants received a middle dose, and participants in a third group received a high dose. In a fourth group, participants received the maximum tolerated dose as determined by the first three groups. In addition, a group of pediatric patients were enrolled at the middle dose.

AGTC said that although the primary endpoint of this study is designed to evaluate safety, efficacy will also be measured by an evaluation of changes in visual structure, function, and quality of life.

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