

The US Food and Drug Administration (FDA) has awarded a regenerative medicine advanced therapy (RMAT) designation to Opus Genetics’ gene therapy OPGx-LCA5 for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene.
The designation was granted based on preliminary clinical evidence from an ongoing dose-escalation, open-label Phase I/II trial, assessing the therapy’s potential efficacy and safety in individuals with severe vision loss caused by confirmed LCA5 gene mutations.
Opus Genetics has received an invitation to take part in the FDA’s initial comprehensive multidisciplinary RMAT meeting, which will support the company’s development and manufacturing strategy.
It is also set to join the agency’s chemistry, manufacturing and controls (CMC) development and readiness pilot (CDRP) programme, providing further guidance for expediting CMC product development under an investigational new drug application.
The designation allows for closer communication with the US regulator, offering guidance on development, manufacturing and potential surrogate endpoints for accelerated approval.
OPGx-LCA5 delivers a functional LCA5 gene to the outer retina using an adeno-associated virus 8 vector, targeting a form of LCA associated with biallelic mutations in the LCA5 gene.

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By GlobalDataIndividuals with LCA5-associated inherited retinal disease experience early-onset severe retinal dystrophy.
Research indicates a disconnect between retinal structure and visual function in this condition, suggesting a window for therapeutic gene augmentation.
The ongoing Phase I/II trial of the therapy, led by Dr Tomas Aleman at the University of Pennsylvania, US, is focused on evaluating the initial efficacy and safety in subjects with inherited retinal degeneration due to LCA5 gene mutations.
Opus Genetics CEO George Magrath said: “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.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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