Gene Therapy AdvancementsEncouraging clinical activity and a manageable safety profile were demonstrated for OPGx-LCA5, with improvements in visual acuity reported in 5 out of 6 patients.
Market PotentialAAV gene therapy portfolio, including multiple assets for inherited retinal diseases (IRDs) and supported by positive data from the lead program, collectively target an estimated >$10B market opportunity.
Regulatory ProgressOpus Genetics, Inc. is pursuing accelerated approval for its OPGx-LCA5 program, with significant progress in its clinical trials.