FDA accepts Nanoscope's BLA for MCO-010; PDUFA date set for H1 2027

Nanoscope Therapeutics/MCO-010 (sonpiretigene isteparvovec)

The FDA accepted Nanoscope's BLA for MCO-010 (sonpiretigene isteparvovec) in September 2026, setting a PDUFA date in H1 2027. The therapy is designed as a one-time intravitreal injection to restore vision in advanced retinitis pigmentosa, making it the first optogenetic gene therapy to reach this milestone.

• RESTORE phase 2b/3 trial (27 patients) showed treated patients gained approximately 3 lines of vision at 1 year vs sham.
• Vision gains were sustained through 76 weeks follow-up (0.539 logMAR improvement).
• 89% of treated patients showed functional improvement on composite testing at 12 months.
• No treatment-related serious adverse events reported over 2-year follow-up; most common findings were manageable anterior chamber inflammation (44%) and elevated intraocular pressure (39%).