Objective:

To present phase 2 findings for OCU410, an investigational gene therapy for geographic atrophy (GA).

Approach:

• Study Design: The phase 2 study enrolled two dose cohorts (medium-dose and high-dose) and a control arm, each with 17 patients.
• Therapeutic Mechanism: OCU410 is a one-time gene therapy that modulates RORA to lower inflammation and complement activation.

Key Findings:

• 34% reduction in GA growth in the medium dose compared to control.
• OCU410 was well tolerated with no cases of retinal vasculitis, occlusive retinal vasculitis, or endophthalmitis.

Interpretation:

OCU410 is a novel modifier gene therapy designed for the treatment of GA.

Conclusion:

A phase 3 program is expected to begin later this year.