Opus Genetics Completes 5-Patient Phase 1/2 OPGx-BEST1 Cohort
Opus Genetics enrolled five adult participants in Cohort 1 of its Phase 1/2 OPGx-BEST1 gene therapy study for Best Vitelliform Macular Dystrophy and autosomal-recessive bestrophinopathy, dosing four and scheduling the fifth this month. The company presented baseline demographics at ARVO and expects three-month topline results in September 2026.
1. Cohort 1 Enrollment Completed
Opus Genetics has enrolled five participants in the adaptive, open-label Phase 1/2 study of OPGx-BEST1, including three with Best Vitelliform Macular Dystrophy and two with autosomal-recessive bestrophinopathy. Four participants have received subretinal administration of the gene therapy and the fifth is scheduled for dosing this month.
2. Baseline Demographics Presented at ARVO
At the ARVO Annual Meeting, the company’s principal investigator shared baseline demographics for all five Cohort 1 participants, highlighting age ranges, disease mutation amenability confirmed via an in vitro platform, and initial tolerability observations from the sentinel patient.
3. Three-Month Topline Results Expected in September
Opus Genetics plans to report three-month topline data from Cohort 1 in September 2026, covering structural OCT measures, microperimetry, best-corrected and low-luminance visual acuity, and contrast sensitivity, with further data presentation slated for a medical conference later this year.