This transcript has been edited for clarity.
Hello, my name is Christine Nichols Kay, MD. I’m a vitreoretinal surgeon and an inherited retinal disease (IRD) specialist at Vitreoretinal Associates in Gainesville, Florida. I had the pleasure of presenting at the Macula Society meeting in February 2026 in San Diego on Nanoscope data. I gave a presentation on vision improvement outcomes in retinitis pigmentosa (RP), a multivariate analysis from the 3-year RESTORE and REMAIN data from the MCO-010 optogenetic gene therapy trial.
Nanoscope’s multicharacteristic opsin, MCO-010, is a disease-agnostic optogenetic therapy that sensitizes bipolar cells to light, restoring vision in patients with low vision and late-stage photoreceptor loss. The way that it does this is that the multicharacteristic opsin protein has been optimized to target bipolar cells, as compared to some other platforms that target other cells, like ganglion cells. This allows it to be active across the visible light spectrum and sensitive at ambient light levels, so no goggles are needed for this therapy. It also has rapid kinetics, which allows for tracking of fast movements of objects without blur.
The RESTORE trial that I presented randomized 27 patients with RP to a low or a high dose of MCO-010 vs a sham intravitreal injection group. These patients had advanced RP with severe vision loss, with a baseline visual acuity worse than 1.9 logMAR. For those of us who don’t think in logMAR, that’s a 20/1600 Snellen visual acuity in the study eye.
Patients were given a single intravitreal injection in the study eye at day 0. Patients receiving MCO-010 also received an oral corticosteroid regimen of prednisone for approximately 3 weeks, beginning 3 days prior to treatment with the intravitreal therapy. The primary endpoint was change from baseline in best-corrected visual acuity (BCVA) at week 52.
MCO-010 achieved its primary endpoint with statistical significance. A clinically meaningful 0.3 logMAR visual improvement was seen, which is approximately 3 lines, or 15 ETDRS letters. This was observed in patients with RP and severe vision loss as compared to the sham patients. Approximately 40% of the active treatment patients gained this 0.3 logMAR improvement in visual acuity, which was excellent to see.
There was also cumulative vision improvement over a 152-week, or 3-year, period. I presented the 152-week data at the Macula Society meeting, demonstrating this magnitude of durable benefit in visual acuity for patients.
The new data I presented this year at Macula Society included correlation analyses showing that patients with the best visual outcomes had better baseline visual acuity and less retinal thinning, or more maintained retinal structure of greater than 150 µm of retinal thickness on optical coherence tomography (OCT).
Importantly, the safety profile was positive. Adverse events were mild to moderate; they generally resolved and were controlled with topical therapy. There were no serious adverse events.
Regarding future development, Nanoscope has initiated a rolling biologics license application (BLA) submission with the US Food and Drug Administration (FDA). I was excited to be able to deliver positive safety and efficacy results from the 152-week data from the RESTORE and REMAIN trial in RP for this optogenetic therapy program. Thank you so much. RP
