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AGTC completes enrollment in trial of X-linked retinoschisis gene therapy

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Enrollment has been completed in a phase 1/2 clinical study of a gene therapy product candidate for the treatment of X-linked retinoschisis, Applied Genetic Technologies Corporation announced in a press release.

The open-label, dose escalation trial will assess the safety and efficacy of the adeno-associated virus-based gene therapy through intravitreal administration in 27 patients with X-linked retinoschisis (XLRS) caused by mutations in the RS1 gene, the release said.

“There are currently no FDA-approved treatment options for XLRS, a leading cause of macular degeneration in young men,” Sue Washer, AGTC president and CEO, said in the release. “The completion of enrollment in this AAV trial represents another significant achievement in our gene therapy clinical development program — a milestone that may improve the lives of individuals affected by XLRS.”

Patients were enrolled in four groups, with the first three groups receiving a low, medium or high dose of the treatment and the fourth group receiving the maximum tolerated dose by the first three groups, according to the release. A pediatric group was also enrolled at the middle dose.

Topline data are expected in the fourth quarter.