IRD OPGX-BEST1 gene therapy data supports phase III advancement
Phase I/II OPGX-BEST1 gene therapy in BVMD and ARB reported strong safety and promising efficacy, with functional gains for all participants and structural improvements for most. FDA alignment on a composite microperimetry and PRO endpoint supports phase III.
The phase I/II trial of OPGX-BEST1 gene therapy in BVMD and ARB showed strong safety and encouraging efficacy, with all participants posting functional gains and most showing structural improvement. FDA alignment on a composite microperimetry and PRO endpoint clears the way for phase III.