Opus Genetics Reports Vision Improvements at Low Dose in BEST1 Gene Therapy Trial, Stock Hits 52-Week High

Opus Genetics (Nasdaq: IRD) released three-month topline data from Cohort 1 of its Phase 1/2 BIRD-1 gene therapy trial on September 9, reporting that its OPGx-BEST1 therapy produced vision improvements even at the low starting dose tested. The company said the results showed encouraging signs of efficacy in participants with both dominant and recessive forms of BEST disease, an inherited retinal condition. Shares hit a 52-week high on the news as investors responded to the positive readout, which had been targeted for the second week of September after full enrollment of Cohort 1 was completed earlier in 2026.

Wedbush raised its price target on Opus Genetics stock following the data release, citing alignment with the FDA as an additional positive development alongside the clinical findings. A separate analyst note maintained a Buy rating with a $10 price target, describing the BIRD-1 data as encouraging and pointing to an expanded commercial opportunity for the BEST1 program. The company has cash runway into 2029, which it expects to support multiple clinical milestones including enrollment in its LCA5 registrational trial, with dosing planned for the fourth quarter of 2026.

Opus Genetics had previously telegraphed the September data readout in a July 9 program update, and the positive Cohort 1 results set the stage for dose escalation into Cohort 2 of the BIRD-1 trial. The company plans to present the data at the EURETINA Congress in Vienna, Austria.