IRD OPGX-BEST1 gene therapy data supports pivotal trial advancement
OPGX-BEST1 phase I/II in BVMD and ARB showed strong safety and promising efficacy. All participants had functional gains and most had structural improvements, with FDA alignment on a 3-decibel microperimetry endpoint and patient-reported outcomes.
The phase I/II trial of OPGX-BEST1 in BVMD and ARB showed strong safety and promising efficacy, with all participants experiencing functional gains and most showing structural improvements. FDA alignment on a 3-decibel microperimetry endpoint and patient-reported outcomes paves the way for a pivota…