Optogenetic therapy shows promise in treating Stargardt disease: STARLIGHT Trial

The optogenetic therapy, MCO-010, showed notable safety and efficacy, with no serious adverse effects observed in the majority of patients with Stargardt disease treated in the Phase 2a STARLIGHT trial.

Significant improvements in visual acuity were noted, especially when utilizing a wearable magnifier.

In this open-label, multicenter 48-week clinical trial, the safety and efficacy of a single intravitreal injection of MCO-010 was evaluated in 6 patients with Stargardt disease. All participants harbored pathogenic genetic mutations in the ABCA4 gene.

At baseline there was a mean BCVA ETDRS letter score of 23. At 48 weeks, the 5 patients who experienced improvement had a mean improvement of 9 ETDRS letters. When employing a wearable magnifier, the mean improvement in BCVA was 16 and 19 ETDRS letters at weeks 24 and 48, respectively. Notably, 3 patients achieved a gain of 30 ETDRS letters in BCVA with a wearable magnifier.

At week 48, no serious adverse effects were reported. Notably, 1 patient experienced chronic uveitis, which was resolved by week 48. While this patient did experience a reduction in ETDRS letters, the overall safety profile remained positive.

Reference
Singer M, et al. MCO-010 Optogenetic Therapy to Treat Stargardt Disease: Safety and Efficacy through Week 48 in the Phase 2a STARLIGHT Clinical Trial. Presented at: Euretina 2023.