IRD OPGX-BEST1 gene therapy meets FDA-aligned microperimetry endpoint
IRD says Phase 1/2 low-dose OPGX-BEST1 in BVMD and ARB showed strong safety and encouraging efficacy, with 75% of evaluable participants meeting an FDA-aligned microperimetry endpoint and noted structural and functional improvements.
The Phase 1/2 low-dose cohort of OPGX-BEST1 gene therapy in BVMD and ARB showed strong safety and encouraging efficacy, with 75% of evaluable participants meeting the FDA-aligned microperimetry endpoint. The update also cited notable structural and functional improvements, especially in earlier-stage disease. Pivotal…