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FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation To Opus Genetics’ OPGx-LCA5 Gene Therapy For Inherited Retinal Disease

Opus Genetics' gene therapy OPGx-LCA5 receives RMAT designation from the FDA for treating LCA5-related vision loss.

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  • May 07, 2025

  • Simantini Singh Deo

FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation To Opus Genetics’ OPGx-LCA5 Gene Therapy For Inherited Retinal Disease

Opus Genetics, Inc., a clinical-stage biotechnology company focused on developing gene therapies for inherited retinal diseases (IRDs) and other eye disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational gene therapy, OPGx-LCA5. This therapy is being developed for the treatment of Leber Congenital Amaurosis (LCA) caused by mutations in the LCA5 gene—a rare and severe inherited eye condition that leads to significant vision loss from an early age.


The RMAT designation was granted based on encouraging early clinical data from Opus’s ongoing Phase 1/2 open-label, dose-escalation study. This trial is evaluating the safety and potential effectiveness of OPGx-LCA5 in patients with severe vision impairment due to confirmed LCA5 gene mutations. The FDA’s decision reflects recognition of both the strength of the initial trial results and the urgent medical need within this patient population.


George Magrath, M.D., Chief Executive Officer, Opus Genetics, mentioned, “The FDA’s decision to grant RMAT designation to OPGx-LCA5 is a major milestone for the LCA5 patient community and a strong validation of our early clinical data. We’re encouraged by the potential of OPGx-LCA5 to meaningfully impact patients living with this ultra-rare and debilitating form of inherited blindness, and we look forward to continued collaboration with the FDA to accelerate its development.”


Alongside the RMAT designation, Opus Genetics has been invited to participate in two important FDA initiatives aimed at accelerating the development of advanced therapies. The company will take part in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting, which is designed to support the alignment of development and manufacturing strategies. Additionally, Opus has been selected for the FDA’s Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program, which provides enhanced guidance for the efficient preparation of CMC data required for regulatory review.


The RMAT designation is part of a special FDA program intended to speed up the development and review of promising regenerative medicine products that target serious or life-threatening conditions. It offers benefits such as early and frequent interactions with the FDA, support for streamlined development and manufacturing processes, and the opportunity to discuss the use of surrogate endpoints for potential accelerated approval. This recognition marks a significant step forward for Opus Genetics in its mission to bring transformative treatments to patients living with inherited retinal diseases.

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