AAO 2026: MCO-010 sustains vision gains in RP through 4 years
Long-term data from the RESTORE trial shows that the optogenetic therapy MCO-010 provides sustained vision improvements for patients with retinitis pigmentosa. The treatment uses light-sensitive proteins to restore visual signaling in the retina.
Why it matters
This represents a breakthrough in gene-agnostic therapy for patients suffering from progressive, untreatable vision loss.
A single intravitreal injection of the optogenetic therapy sonpiretigene isteparvovec (MCO-010; Nanoscope Therapeutics) produced visual acuity gains in patients with severe vision loss from retinitis pigmentosa (RP) that were sustained through 4 years, according to data presented by Kenneth C. Fan, MD, MBA, a vitreoretinal and inherited retinal disease specialist at Retina Consultants of Texas in Houston. Fan presented the 4-year REMAIN follow-up data from the phase 2b/3 RESTORE trial at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting in New Orleans, Louisiana, and discussed them with Ophthalmology Times.¹
Patients with RP face “a very tragic, progressive vision loss,” Fan said, and no FDA-approved gene-agnostic therapy exists for them. MCO-010 is a disease-agnostic therapy that delivers a light-sensitive opsin protein to retinal bipolar cells. The bipolar cells can then serve as a proxy for lost photoreceptors, sending signals to the brain when light is present.
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