AGTC Announces Completion of Enrollment of Phase 1 / 2 Clinical Study of Investigational Gene Therapy

Applied Genetic Technologies (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced the completion of enrollment in a clinical study of the company’s gene therapy product candidate, in collaboration with Biogen, for the treatment of x-linked retinoschisis (XLRS). This multicenter study is designed to evaluate the collaboration’s AAV vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with XLRS caused by mutations in the RS1 gene. Topline data are anticipated by Q4 2018 with the final analysis at the twelve-month time point.

As quoted in the press release:

The Phase 1/2 trial is an open-label, dose escalation study designed to assess the safety and efficacy of intravitreal administration of the AAV-based gene therapy in approximately 27 patients diagnosed with XLRS caused by mutations in the RS1 gene. Trial participants were enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 received a low, middle or high dose of the investigational study agent. Patients in Group 4 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. 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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