RESEARCH TRIANGLE PARK, N.C., September 2, 2026
Opus Genetics, Inc. plans to present topline clinical data from Cohort 1 of the Phase 1/2 OPGx-BEST1 gene therapy program during an investor conference call and webcast on September 9, 2026. The company expects the upcoming presentation to provide the first topline clinical update from the cohort, while the full clinical dataset and additional analyses of visual function are scheduled for presentation at several leading ophthalmology meetings later this month and in October. The data are expected to provide further insight into the development of OPGx-BEST1 for BEST1-related inherited retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).
Opus Genetics Sets OPGx-BEST1 Data Readout
The investor webcast is scheduled for September 9 at 8:00 a.m. ET, when Opus Genetics will present topline results from Cohort 1 of the ongoing Phase 1/2 clinical study. Additional clinical analyses will follow at the Euretina Innovation Spotlight on September 30 and Euretina 2026 on October 4, where investigators are expected to discuss initial results from patients with BVMD and ARB caused by BEST1 mutations. Further presentations are planned at Retina in Rio on October 14 and the FLORetina meeting, including analyses examining structure-function relationships, microperimetry and other measures of retinal and visual function. The sequence of presentations is expected to provide a broader view of the program beyond the initial topline investor update.
Gene Therapy Targets BEST1-Related Retinal Disease
OPGx-BEST1 is an investigational AAV-based gene therapy designed to address the underlying genetic cause of BEST1-related inherited retinal disease. The therapy uses an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1 study is an adaptive, open-label Phase 1/2 trial evaluating the safety and efficacy of a single-eye subretinal administration of OPGx-BEST1 in adults with BVMD or ARB. By delivering a functional gene copy directly to affected retinal cells, the program is intended to pursue a potentially durable treatment approach for inherited retinal disorders associated with BEST1 mutations.
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Opus Advances Broader Retinal Gene Therapy Pipeline
The OPGx-BEST1 data update represents another clinical milestone for Opus Genetics as the company advances a broader pipeline of gene therapies intended to restore vision and prevent blindness in patients with inherited retinal diseases. The company is developing seven AAV-based programs, with OPGx-BEST1 and OPGx-LCA5 among its lead candidates. Additional programs target RDH12, MERTK, RHO, CNGB1 and NMNAT1. The upcoming clinical presentations will be particularly important for OPGx-BEST1 as the company moves from early clinical evaluation toward a more detailed assessment of safety, retinal structure and visual function. Further data from Cohort 1 could help define the next stage of clinical development for the investigational therapy in BEST1-related retinal degeneration.
Source: Opus Genetics, press relese
