On May 6, 2025, Opus Genetics, Inc. announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5. This investigational gene therapy targets Leber Congenital Amaurosis (LCA) caused by genetic variations in the LCA5 gene.
The RMAT designation is based on early clinical evidence from Opus's ongoing Phase 1/2 open-label, dose-escalation trial, which is evaluating the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss. This decision validates the strength of the initial data and the significant unmet need in this patient population.
The RMAT program offers potential for expedited development and review, early interactions with the FDA, guidance on efficient development and manufacturing, and the opportunity to discuss surrogate endpoints for accelerated approval. Opus has also been invited to participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting and the Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program.
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