Opus Genetics to unveil OPGx-BEST1 Phase 1/2 data on 9 September
Opus Genetics has announced it will present topline clinical data from Cohort 1 of its Phase 1/2 BIRD-1 study of OPGx-BEST1 during an investor webcast on 9 September, with fuller results to follow at three international ophthalmology meetings through October.
OPGx-BEST1 is an investigational adeno-associated virus (AAV) gene therapy designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The programme targets BEST1-related inherited retinal diseases, specifically Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB), two rare conditions caused by mutations in the BEST1 gene that progressively impair central vision. The BIRD-1 study is an adaptive, open-label Phase 1/2 trial evaluating safety and efficacy of single-eye subretinal administration in adults.
The data readout schedule
The 9 September investor call, scheduled for 8:00 a.m. ET, will carry topline Cohort 1 findings. A more detailed presentation of the full clinical dataset is then slated for three conferences. At Euretina 2026 on 4 October, Mark Pennesi of the Casey Eye Institute will present initial Cohort 1 results. Retina in Rio on 14 October will include a structure-function correlation analysis of key visual measures drawn from BIRD-1, presented by Rodrigo Jorge of the University of São Paulo. A further FLORetina session will report three-month Cohort 1 results in BVMD and ARB patients, with Aaron Nagiel listed as presenter.
Opus chief executive George Magrath will separately present at Retina in Rio on near-normalisation of cone sensitivity using gene augmentation in children with LCA5, the company's lead programme targeting a distinct inherited retinal disease.
Market context
Inherited retinal disease gene therapy has attracted considerable commercial and clinical attention since the FDA approval of Spark Therapeutics' voretigene neparvovec (Luxturna) for RPE65-related dystrophy in 2017. That approval established the regulatory template for subretinal AAV delivery and demonstrated the pathway for single-administration treatments in rare retinal conditions. Since then, a number of clinical-stage companies, including REGENXBIO, Applied Genetic Technologies Corporation (AGTC, now part of Beacon Therapeutics), and 4D Molecular Therapeutics, have advanced programmes targeting distinct IRD gene mutations, creating a competitive but still largely unsaturated landscape given the breadth of the genetic causes involved.
Opus has built a pipeline of seven AAV-based candidates, spreading risk across multiple mutations rather than concentrating on a single indication. The BEST1 programme is the second most advanced behind OPGx-LCA5, and the company's willingness to present at peer-reviewed venues alongside its investor webcast is a positive signal for data quality and scientific engagement. Investors will be scrutinising visual acuity outcomes, the microperimetry data referenced in the Retina in Rio abstract title, and any adverse events in the treated eye before drawing conclusions about the programme's development trajectory.
No data have been disclosed in this announcement, and no regulatory submission timeline has been indicated. The Cohort 1 readout is an early-stage safety and proof-of-concept signal; a larger Cohort 2 expansion and eventual pivotal design will depend on what the September and October presentations show.