Home Challenging Results TrialWatch: Gene Therapy Overall Fails in X-Linked Retinoschisis

TrialWatch: Gene Therapy Overall Fails in X-Linked Retinoschisis

175
0

Applied Genetic Technologies Corporation’s investigational AAV-based gene therapy for X-linked retinoschisis due to mutations in the RS1 gene showed no signs of clinical activity at 6 months in a Phase I/2 trial. 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.

LEAVE A REPLY

Please enter your comment!
Please enter your name here

This site uses Akismet to reduce spam. Learn how your comment data is processed.