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Early clinical trial results show vision gains in patients with BEST1-related disease
02-10-2026 | 4 Min.Opus Genetics has shared encouraging early results from its ongoing Phase 1/2 clinical trial for OPGx-BEST1, a potential one-time gene therapy for people with BEST1-related retinal disease. All five patients enrolled in the clinical trial had clinically meaningful improvement in visual function, according to Opus Genetics. The greatest functional improvements were observed in participants with less advanced disease.- Beacon Therapeutics reported positive results from the Phase 3 pivotal trial of laru-zova, its potential gene therapy treatment for X-linked retinitis pigmentosa. A significant proportion of patients improved dim-light vision by 15 or more letters after treatment with either high or low doses of laru-zova compared to the group that was not treated, according to preliminary results from the VISTA clinical trial. Beacon plans to begin seeking FDA approval later this year. If approved, this would be the first treatment available for XLRP, one of the more severe forms of retinitis pigmentosa that predominantly affects males.
- The U.S. Food and Drug Administration accepted Dallas-based Nanoscope Therapeutics' biologics license application for MOGENRY, an emerging treatment for adults with retinitis pigmentosa and severe vision loss. The investigational treatment does not require a genetic diagnosis or specific gene mutation, giving MOGENRY the potential to become the first approved gene-agnostic treatment to improve vision in people with severe vision loss from RP.
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