Opus Genetics has secured a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration for its investigational gene therapy candidate OPGx-LCA5.
Currently, OPGx-LCA5 is in early-stage clinical evaluation for Leber congenital amaurosis caused by LCA5 gene mutations.
The RMAT designation was granted to OPGx-LCA5 based on initial data from an ongoing Phase 1/2 dose-escalation study evaluating its safety and early signs of efficacy.
The open-label clinical trial is being led by Dr Tomas Aleman at the University of Pennsylvania and focuses on patients affected by inherited retinal degeneration due to confirmed biallelic mutations in the LCA5 gene.
In addition to the designation, the FDA has invited Opus Genetics to participate in its Initial Comprehensive Multidisciplinary RMAT Meeting. The company will also join the agency’s Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) programme, which offers additional regulatory support to streamline the development of products submitted under investigational new drug applications.
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.”
The RMAT pathway, created under the 21st Century Cures Act, facilitates earlier and more frequent engagement with the FDA for sponsors of regenerative therapies. It enables discussions on clinical trial design, development strategy, and surrogate endpoints that may support accelerated approval.
To qualify, candidates must target serious or life-threatening conditions and present preliminary evidence indicating potential benefit.
OPGx-LCA5 is engineered to deliver a functional copy of the LCA5 gene using an adeno-associated virus 8 (AAV8) vector. The treatment aims to restore expression of the lebercilin protein, which is deficient in individuals with LCA5-related disease.
This inherited retinal disorder typically presents in early childhood and is characterised by severe vision loss. Research has indicated a dissociation between preserved retinal structure and impaired visual function in affected patients, suggesting a therapeutic window where gene replacement could be beneficial.
The company has indicated that the RMAT designation will support its efforts to advance OPGx-LCA5 through clinical development with regulatory alignment on manufacturing and safety requirements. The participation in the CDRP initiative is expected to help address chemistry, manufacturing and control-related milestones required for future stages of development.
Opus Genetics is a clinical-stage biotechnology company listed on Nasdaq under the ticker symbol IRD. The firm is focused on developing gene therapies to treat inherited retinal diseases and other ocular conditions.
It previously announced the initiation of its Phase 1/2 trial in 2023 and is advancing additional candidates in its pipeline targeting other forms of inherited retinal dystrophy.
