Gene therapy fails to show clinical activity in X-linked retinoschisis

An investigational AAV-based gene therapy from Applied Genetic Technologies Corporation for X-linked retinoschisis due to mutations in the RS1 gene showed no signs of clinical activity at 6 months in a phase 1/2 trial, the company announced.
The rAAV2tYF-CB-hRS1 intravitreal injection was shown to be safe and well-tolerated in the dose-escalation study, but due to the lack of clinical activity, the company has decided not to move forward with further trials, Sue Washer, CEO of AGTC, said in a conference call regarding the study results.
“We will continue to analyze (Read more...)

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