Core Insights - Opus Genetics, Inc. announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the ARVO 2025 Meeting, showcasing advancements in gene therapies for inherited retinal diseases [1][3] - The company is focusing on its lead candidate OPGx-LCA5, which is currently in a Phase 1/2 trial, with promising 12-month data indicating visual improvement in adult patients [4][3] - Additional presentations will cover pre-clinical results for OPGx-MERTK and OPGx-RDH12, expanding the company's pipeline in treating various retinal disorders [5][7] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies for inherited retinal diseases and other ophthalmic disorders [9] - The company's pipeline includes AAV-based investigational gene therapies targeting mutations in genes associated with bestrophinopathy, Leber congenital amaurosis (LCA), and retinitis pigmentosa [9] - The most advanced program, OPGx-LCA5, is designed to address mutations in the LCA5 gene and is currently undergoing a Phase 1/2 open-label, dose-escalation trial [9] Upcoming Presentations - The presentation titled "Recovery of cone mediated vision in severe ciliopathy after gene augmentation; One year results from a Phase I/II trial of LCA5-LCA (OPGx-LCA5)" will take place on May 4, 2025, highlighting the efficacy of OPGx-LCA5 [4] - Another presentation on OPGx-MERTK will discuss its evaluation in a rat model of retinitis pigmentosa, scheduled for May 8, 2025 [5] - A study on the ocular tolerability of OPGx-RDH12 will be presented on May 5, 2025, focusing on its delivery in cynomolgus primates [6][7] Clinical Trials and Regulatory Status - The ongoing Phase 1/2 trial of OPGx-LCA5 has shown that all three adult patients experienced visual improvement at six months, with new data indicating sustained efficacy for a year [4][3] - The company plans to advance OPGx-LCA5 into a pivotal Phase 3 trial, contingent on continued safety and efficacy [3] - Phentolamine Ophthalmic Solution 0.75% is currently in Phase 3 trials for treating presbyopia and reduced dim light vision, with FDA Fast Track Designation granted for chronic night driving impairment [9]
Opus Genetics Announces Presentations at Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting