On May 5, 2025, Opus Genetics, Inc. announced one-year results from adult patients treated in the ongoing Phase 1/2 study of its lead gene therapy candidate, OPGx-LCA5. These results were presented at the 2025 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO).
The data showed durable efficacy, with subjective and objective signs of visual improvement observed at six months persisting for a full year in patients with severe vision impairment due to LCA5 gene mutations. Patients experienced gains in cone-mediated vision, improving reading ability and object recognition.
The administration of OPGx-LCA5 by subretinal injection was well tolerated by study participants. These findings support the continued development of OPGx-LCA5, with discussions ongoing with the FDA regarding a registrational trial design for potential initiation in 2026.
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