The ongoing Phase 2b/3 RESTORE clinical trial has revealed promising results for MCO-010, an optogenetic therapy aimed at treating retinitis pigmentosa (RP). The study, led by Allen Ho, MD, at Wills Eye Health System, assessed the efficacy of MCO-010 in patients with advanced RP over 52 weeks.

The trial involved 27 patients divided into three groups: low-dose MCO-010, high-dose MCO-010, and a sham control. MCO-010, delivered via an AAV2 vector, targets bipolar cells in the retina to express a photosensitive opsin protein, restoring light sensitivity.

Results showed significant improvements in best-corrected visual acuity (BCVA). Low-dose patients exhibited mean improvements of 0.171, 0.207, 0.438, and 0.337 logMAR at weeks 16, 24, 36, and 52, respectively. High-dose patients showed improvements of 0.077, 0.220, 0.228, and 0.301 logMAR at the same intervals. Statistically significant gains were observed at week 36 and maintained through week 52, with P-values of 0.0021 (low-dose) and 0.0355 (high-dose).

Importantly, MCO-010 was well tolerated, with no serious adverse effects reported. These findings suggest that MCO-010 could be a viable treatment for restoring vision in patients with advanced RP.