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The FDA granted regenerative medicine advanced therapy designation to OPGx-LCA5 for the treatment of Leber congenital amaurosis due to genetic variations in the LCA5 gene, according to a press release from Opus Genetics.
The gene therapy is designed to deliver a functional LCA5 gene to the outer retina using an adeno-associated virus 8 vector. Early clinical evidence from an ongoing phase 1/2 open-label dose-escalation trial investigating the safety and potential efficacy of OPGx-LCA5 served as the basis