Objective:
To evaluate the long-term efficacy and safety of MCO-010, an optogenetic gene therapy, in patients with advanced retinitis pigmentosa (RP).
Key Findings:
- Both treatment arms showed statistically significant BCVA improvements at 52 weeks compared to sham.
- Long-term follow-up through 152 weeks demonstrated sustained BCVA improvements of +0.265 LogMAR (high dose) and +0.456 LogMAR (low dose).
- Greater efficacy was associated with better baseline visual acuity and less retinal thinning.
- No treatment-related serious adverse events were reported over 3 years.
Interpretation:
The findings suggest that MCO-010 provides durable, clinically meaningful visual improvement in advanced RP patients, with treatment response influenced by anatomic and disease-stage variables.
Limitations:
- Small sample size of 27 patients may limit generalizability.
- Long-term effects beyond 3 years are not yet established.
Conclusion:
MCO-010 represents a promising optogenetic therapy for retinal degeneration, with potential to revolutionize treatment for low-vision patients.
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