Nanoscope Reports Long-Term Data for It’s Optogenetic Therapy in Retinitis Pigmentosa

Nanoscope Therapeutics recently reported 5-year safety results from its EXTEND study, offering long-term evidence supporting the safety and durability of MCO-010, an intravitreal optogenetic therapy for patients with advanced retinitis pigmentosa (RP).

The results of the EXTEND study—a follow-up to the company’s phase 1/2a trial that included 10 participants who had received a single intravitreal injection of MCO-010—confirmed no serious adverse events or new safety signals over 5 years, reinforcing MCO-010’s safety profile and suggesting long-term tolerability following a single administration.

“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.”

During the original trial, participants receiving the higher MCO-010 dose achieved statistically significant improvements in visual acuity after 1 year, which translated to sustained or enhanced vision-related quality-of-life scores through 5 years, with notable benefits in distance activities and visual independence.

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

Nanoscope has initiated a rolling Biologics License Application (BLA) with the US FDA for MCO-010 in RP and has reported encouraging data for its use in Stargardt disease. The therapy holds FDA Fast Track, Orphan Drug, and RMAT designations, as well as EMA Orphan designations covering non-syndromic and syndromic rod- and cone-dominant dystrophies and macular dystrophies.