At the 2025 annual meeting of the American Society of Retina Specialists (ASRS), new data brought encouraging news for families affected by retinitis pigmentosa (RP). Dr. David Liao, MD, PhD, presented 126-week (over 2 years) follow-up results from the RESTORE (REMAIN) study of MCO-010, an innovative optogenetic therapy for advanced RP.
What the Study Found
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Patients with advanced RP, including those with very limited vision (20/1600 or worse), received a one-time injection of MCO-010.
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After more than two years of follow-up, many patients showed meaningful and lasting improvements in vision.
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On average, patients gained the equivalent of 3 lines on a standard eye chart (Snellen).
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The therapy continued to show a favorable safety profile, with no major adverse effects reported.
How MCO-010 Works
Unlike traditional gene therapies that target specific genetic mutations, MCO-010 is gene-agnostic — meaning it can potentially help patients regardless of their exact RP mutation.
Here’s what makes it unique:
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Uses optogenetics to make non-photoreceptor retinal cells sensitive to light.
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Specifically targets bipolar cells, which remain functional even in advanced disease.
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Works across the visible light spectrum, using natural ambient light.
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Designed for patients who have already lost most of their photoreceptors, offering hope even at late stages.
What’s Next
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Nanoscope Therapeutics, the company behind MCO-010, has already submitted a rolling Biologics License Application (BLA) to the FDA.
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Additional studies are planned to collect more data and further evaluate outcomes.
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FDA updates could come as soon as 2026, potentially opening the door to broader patient access.
Why This Matters
For decades, patients with inherited retinal diseases like RP have been told to wait — that treatments were always “five or ten years away.” Now, those treatments are finally becoming a reality.
MCO-010 may not be a cure for everyone, but it represents a powerful new option — one that doesn’t depend on genetic testing and could benefit many who previously had no hope of therapy.
At A Race Against Blindness, we’re following these advancements closely. Every new study moves us closer to a world where vision loss from inherited diseases like RP and Bardet-Biedl Syndrome no longer feels inevitable.
Source: Modern Retina
