Nanoscope Therapeutics Presents 5-Year Safety Data for MCO-010 Optogenetic Therapy in Retinitis Pigmentosa

Nanoscope Therapeutics has reported five-year long-term safety results from its EXTEND study, reinforcing the safety and sustained tolerability of MCO-010, an intravitreal optogenetic treatment for patients with advanced retinitis pigmentosa (RP). The findings support the potential of MCO-010 as a durable therapy that may restore functional vision following a single injection.

Long-Term Safety and Tolerability Confirmed

The EXTEND study followed participants from the original Phase 1/2a clinical trial, in which 10 patients with advanced RP received a single intravitreal dose of MCO-010. Over the five-year follow-up period, no serious adverse events or new safety concerns were observed. The consistent safety profile suggests strong long-term tolerability and durability of response.

“These results demonstrate the excellent long-term safety and tolerability of MCO-010, even 5 years after a single injection,” said Samarendra Mohanty, PhD, President and Chief Scientific Officer of Nanoscope.
“The absence of serious safety signals and strong participant retention underscore the potential of MCO-010 as a durable, noninvasive therapy for severe vision loss in RP patients.”

Sustained Functional Benefits in Vision and Daily Activities

In the initial study, participants who received the higher dose of MCO-010 achieved statistically significant improvements in visual acuity at one year. These improvements were maintained or enhanced through year five, with participants reporting notable gains in visual independence, distance navigation, and overall quality of life.

These functional benefits highlight the therapy’s continued impact beyond the early treatment period.

The EXTEND findings were presented at the 34th Annual Conference of the Vitreo Retinal Society – India.

Regulatory Pathway and Broader Clinical Development

Nanoscope is currently conducting a rolling Biologics License Application (BLA) with the U.S. FDA for MCO-010 in the treatment of RP. The therapy has also shown encouraging clinical signals in Stargardt disease.

MCO-010 has received multiple regulatory designations, including FDA Fast Track, FDA Orphan Drug, and FDA RMAT (Regenerative Medicine Advanced Therapy) status. Additionally, it holds EMA Orphan Designations covering rod- and cone-dominant dystrophies as well as macular dystrophies.