Objective:

To present long-term results of Nanoscope Therapeutics’s MCO-010 optogenetic therapy for improving vision in patients with retinitis pigmentosa (RP).

Approach:

Key Findings:

• MCO-010 achieved a statistically significant improvement of 0.3 logMAR in BCVA, equating to approximately 3 lines or 15 ETDRS letters.
• 40% of patients receiving MCO-010 experienced this improvement compared to sham patients.
• Cumulative vision improvement was observed over a 3-year period.
• Patients with better baseline visual acuity and less retinal thinning had better visual outcomes.

Interpretation:

The data suggest that MCO-010 is a promising optogenetic therapy for patients with severe vision loss due to RP, demonstrating both efficacy and a positive safety profile.

Limitations:

• The study involved a small sample size of 27 patients.
• Long-term effects beyond 3 years are still unknown.

Conclusion:

MCO-010 shows durable benefits in visual acuity for patients with RP, and Nanoscope is pursuing FDA approval for this therapy.

Sources:

• Macula Society Meeting Presentation
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