Opus reports sustained vision gains as LCA5 gene therapy moves toward Phase 3 dosing

Opus Genetics reported sustained visual improvements in six participants receiving its experimental OPGx-LCA5 gene therapy, with follow-up through 24 months in three adults and six months in three adolescents. Presenting at Retina in Rio, the company said its Phase 3 natural history and treatment cohorts were fully enrolled, with dosing expected in the fourth quarter of 2026. The therapy delivers a functional LCA5 gene to retinal light-sensitive cells to restore production of lebercilin, a protein needed for normal retinal function. Opus advanced the low dose into Phase 3 without further dose escalation. The findings remain interim, and the company cautioned that initial results may not predict final trial outcomes.