Opus Genetics (IRD) KOL event summary

Introduction and agenda

• Event focused on presenting 6-month safety and efficacy data for OPGx-LCA5, an AAV-based gene therapy for LCA5-associated inherited retinal disease, including detailed patient-level data, clinical endpoints, and a live Q&A session.

• Agenda included company updates, disease overview, clinical results, and discussion of unmet needs and next steps in the development program.

KOL background and credentials

• Panel featured leading experts in gene therapy and retinal diseases from University of Pennsylvania, Sierra Eye Associates, and Vitreoretinal Associates Florida, including Jean Bennett, Tomas Aleman, Christine Kay, and Arshad Khanani.

• Speakers have extensive experience in clinical trials, gene therapy innovation, and IRD patient care.

Market insights and analysis

• IRDs are caused by over 300 genes, affect more than 180,000 people in the US, and have significant unmet needs with limited approved treatments.

• Gene augmentation via AAV vectors offers promise, but commercialization faces challenges such as small patient populations, manufacturing scale, and high clinical standards.

• Strategic alliances with patient organizations, focused production lines, and bespoke manufacturing partnerships are key to efficient trial enrollment and high-quality production.