On April 8, 2025, Opus Genetics, Inc. announced one-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label trial for LCA5-related inherited retinal disease. The data showed an encouraging early safety profile and meaningful improvement in visual function.
The first pediatric participant, aged 16 at the time of consent, experienced significant brightening of objects and improved ability to distinguish letters and navigate independently after one month. No drug-related adverse events have been reported to date.
A second pediatric patient was recently dosed, and the pediatric cohort is expected to complete enrollment in the second quarter of 2025, with initial data from all three patients anticipated in the third quarter of 2025. An FDA Type D meeting provided clarity on the design for a potential registrational trial, with initiation possible in 2026.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.