The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Opus Genetics for its investigational gene therapy OPGx-LCA5, which is intended to treat Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene. The decision follows early findings from a phase 1/2 clinical trial showing initial safety and potential efficacy in patients with severe vision loss.

LCA5 is a rare inherited retinal disease that causes early onset vision loss. OPGx-LCA5 uses an adeno-associated virus (AAV8) vector to deliver a functional copy of the LCA5 gene to the retina. The ongoing open-label, dose-escalation study is being conducted at the University of Pennsylvania.

The RMAT designation is based on preliminary clinical evidence and provides Opus with expanded FDA interaction, including the opportunity to participate in the agency’s Chemistry, Manufacturing and Controls Development and Readiness Pilot program. These regulatory pathways are designed to support faster development of therapies for serious conditions. In a press release, the company said it plans to continue collaborating with the FDA as it advances the therapy. RP