Opus Genetics (IRD) KOL event summary

Introduction and agenda

• Event focused on 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, efficacy assessments, 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.

Market insights and analysis

• Over 300 genes cause inherited retinal diseases (IRDs), affecting more than 180,000 people in the US, with most IRDs lacking approved treatments.

• LCA5-associated IRD is a severe, early-onset dystrophy with significant unmet therapeutic needs; OPGx-LCA5 targets biallelic LCA5 mutations using an AAV8 vector.

• Gene augmentation strategies using AAV vectors show promise, but commercialization faces challenges due to small patient populations and manufacturing scale.

• Regulatory agencies are increasingly supportive of rare disease therapies and novel endpoints.

• Strategic partnerships and focused production lines are used to overcome manufacturing barriers.