Nanoscope Therapeutics reported 5-year follow-up data on its optogenetic therapy MCO-010, showing sustained safety in patients with advanced retinitis pigmentosa (RP) who received a single intravitreal injection during an earlier phase 1/2a study. The data come from the EXTEND study, which followed 10 participants and found no serious adverse events, treatment discontinuations, or new safety signals over the 5-year period.
MCO-010 uses Nanoscope’s multicharacteristic opsin platform to restore photosensitivity in degenerated retinal cells. According to the company, transient inflammation remained the most common treatment-related finding and was manageable. Participants also reported sustained improvements in quality-of-life measures, particularly in distance vision tasks and functional independence.
Earlier phase 1/2a results demonstrated that participants receiving the higher dose experienced statistically significant gains in visual acuity at one year. Those functional gains did not appear to diminish during the long-term follow-up, though efficacy outcomes from EXTEND were not formally reported. The data was reported at the 34th annual conference of the Vitreoretinal Society in India.
Nanoscope has begun a rolling submission of a biologics license application to the US Food and Drug Administration for MCO-010 for severe vision loss due to RP. The company is also pursuing development of the therapy for Stargardt disease and plans to begin a phase 2 program in geographic atrophy by late 2025. RP