BRIEF—Nanoscope files first BLA for gene-agnostic retinal therapy

US biotech Nanoscope Therapeutics has begun a rolling submission to the American medicines regulator for MCO-010, the first gene-agnostic therapy seeking approval for retinitis pigmentosa (RP).

Unlike most gene therapies tailored to specific mutations, MCO-010 targets the broader RP population—an inherited condition linked to over 100 genes—by activating dormant retinal cells regardless of the underlying mutation. The therapy is delivered through a one-time, in-office injection and does not require surgery or genetic testing.

The submission follows positive results from the Phase IIb RESTORE trial, where patients showed meaningful vision gains of more than three lines on an eye chart at 52 weeks, with benefits sustained through three years and no serious safety issues reported.

If approved, MCO-010 would be the first treatment to restore vision in legally blind RP patients without needing genetic matching. Completion of the full BLA is expected in early 2026, and the application is eligible for priority review under its fast-track designation.