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Key Highlights OPGx-BEST1 targets BEST1-associated inherited retinal diseases, including BVMD and ARB AAV-based gene therapy approach designed to deliver a functional BEST1 gene directly to retinal pigment epithelium cells Five participants enrolled in Cohort 1, including three with BVMD and two with ARB Four participants have been dosed, with the fifth scheduled for dosing this month Dominant BEST disease participants underwent in vitro confirmation to assess whether their mutations are amenable to gene augmentation Early sentinel participant data showed positive tolerability and biological activity after subretinal administration Three-month topline Cohort 1 data expected in September 2026 For BEST1-associated diseases, careful patient selection, mutation-specific validation, and multi-endpoint retinal assessments may be key to advancing gene augmentation strategies
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Patients should be monitored for signs of inadequate nutritional intake, particularly elderly patients or those with low baseline body weight, as determined relevant by the prescribing clinician
Microdosing may not provide the full therapeutic benefits of standard dosing, and any dosing adjustments should be made under medical supervision to ensure both safety and effectiveness
