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,”
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