Retina Society 2026: MCO-010 optogenetic therapy maintains visual acuity gains through 4 years in advanced RP

At the Retina Society 59th Annual Scientific Meeting in Los Angeles, California, Christine Kay, MD, presented 4-year durability data for MCO-010 (Nanoscope Therapeutics). MCO-010 is an intravitreally delivered, mutation-agnostic optogenetic therapy for patients with advanced retinitis pigmentosa (RP). Kay is a vitreoretinal surgeon and inherited retinal disease (IRD) specialist. She discussed the RESTORE and REMAIN data with Modern Retina.

Targeting bipolar cells, not photoreceptors

MCO-010 is an adeno-associated virus (AAV)–mediated therapy delivered as a single intravitreal injection. It sensitizes the remaining healthy bipolar cells to light. Because the therapy does not depend on surviving photoreceptors, it is intended for patients who have already lost them.

RESTORE: Primary end point met

The phase 2b/3 RESTORE trial enrolled patients with end-stage RP and photoreceptor loss. The trial had 3 arms of 9 patients each: high dose, low dose, and sham. Both dose groups met the primary end point, a mean improvement of 0.3 logMAR at week 52. This result was statistically significant compared with sham, which showed no improvement. Kay noted that 0.3 logMAR corresponds to approximately 3 lines, or 15 ETDRS letters. Many patients had off-chart acuity, so the investigators measured vision with low-vision scales such as the BRVT or Freiburg test.

REMAIN: Durability through year 4

REMAIN is the 4-year follow-up to RESTORE. "The REMAIN data shows durable visual acuity gains out to year four," Kay said. On average, 40% of treated patients gained 0.3 logMAR and maintained that gain for 4 years. Over the same period, patients in the sham arm stayed stable or declined, consistent with the natural history of RP. Kay noted that 10- and 20-year data are not yet available.

Selecting candidates

In Kay's view, the best candidates are patients with late-stage RP and visual acuity of 20/200 or worse. Patients with good acuity would not benefit. Patients with severe concomitant ocular disease are also poor candidates, such as those with end-stage glaucoma or ischemic optic neuropathy, as are patients with a cortical stroke. She added that longer-term data and trials across different levels of visual acuity are still needed.

Genetic testing remains standard of care

Treatment candidacy does not depend on genotype. Even so, "I'm a big proponent of genetic testing for inherited retinal disease patients, so it still should be done," Kay said. She described mutation-agnostic therapy as one approach among several. Gene augmentation therapies are still needed for patients with early-stage disease who have not lost acuity.

A message for eye care providers

Kay said some patients report being told that nothing can be done for them and that they do not need follow-up. "It's no longer okay to tell our RP patients that there's nothing that we can do for you or that we don't need to see you again," she said. Options range from low-vision evaluations to clinical trials. Voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) has been FDA approved since 2017 for RPE65-mediated disease.

The biologics license application (BLA) for MCO-010 has been accepted and awaits an FDA decision. "Now we wait," Kay said.