Opus Genetics said an independent data monitoring committee has recommended that the company’s phase 1/2 BIRD-1 trial of OPGx-BEST1 for Best disease proceed without modification. The committee completed its planned review of 1-month safety data from the first treated participant and supported advancing enrollment to the next cohort.

The study is designed to evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1, an AAV-based gene therapy intended to deliver a functional BEST1 gene to retinal pigment epithelium cells in a single subretinal injection. The treatment is being assessed in 2 dosing cohorts across multiple centers. Investigators will track functional and anatomic outcomes, including changes in visual acuity and retinal structure.

OPGx-BEST1 is based on preclinical data showing restoration of BEST1 protein expression and improvements in retinal function in disease models. Best disease and related bestrophinopathies, caused by mutations in the BEST1 gene, affect an estimated 9,000 people in the United States and can lead to progressive vision loss. RP