Recent Updates — IRD
Opus Genetics, Inc. issued a revised data presentation on September 11, 2026, correcting an immaterial error in translating logMAR visual acuity to ETDRS letters for Cohort 1 of its Phase 1/2 trial of OPGx-BEST1 in patients with BEST1-related retinal diseases. The correction adjusted mean best-corrected visual acuity changes from baseline through three months, showing individual participant improvements or losses ranging from a 12-letter gain to an 8-letter loss in treated eyes. High-resolution microperimetry images were added to the appendix. Opus Genetics operates in the biotechnology industry and develops gene therapies for inherited retinal diseases.
Opus Genetics announced positive interim data from Cohort 1 of its Phase 1/2 BIRD-1 clinical trial for OPGx-BEST1, a gene therapy for BEST1-related retinal diseases. All five participants treated at 1.5 x 10⁹ vg/eye demonstrated clinically meaningful improvements in visual function and structural metrics, with no serious adverse events reported. The company advanced to higher-dose Cohort 2 (4.5 x 10⁹ vg/eye), which is over-enrolled with eight participants; dosing completion is expected in Q4 2026. Additionally, Opus Genetics aligned with the FDA on potential pivotal trial endpoints and manufacturing requirements, targeting Phase 3 participant dosing to begin in 2027. The company operates as a clinical-stage biopharmaceutical firm developing gene therapies for inherited retinal diseases.
Opus Genetics reported a net loss of $8.1 million, or ($0.08) per basic share, for the second quarter ended June 30, 2026, compared to a $7.4 million loss in the prior year period. Revenue declined to $0.8 million from $2.9 million as R&D expenses rose to $11.2 million due to higher manufacturing and clinical costs associated with inherited retinal disease programs. The company holds $88.8 million in cash, projecting a runway into 2029. Clinically, the OPGx-LCA5 registrational trial enrollment is complete with dosing planned for Q4 2026, while OPGx-BEST1 Cohort 1 topline data is expected in September 2026. Opus Genetics operates as a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases.