Intravitreal gene therapy that makes surviving bipolar cells light-sensitive in advanced retinitis pigmentosa. One-time injection restores vision regardless of genetic cause.
BLA accepted September 2026. PDUFA decision H1 2027. First optogenetic vision therapy to reach FDA review.
Updated 9 Sep 2026Checked 10 Oct0 updates this week
MCO-010 carries a gene encoding a broadband light-sensitive protein (opsin) engineered to respond across visible wavelengths.
A viral vector delivers the gene through the vitreous, crosses the retina and infects surviving bipolar cells.
In retinitis pigmentosa, rods and cones die but bipolar neurons survive. The opsin gene makes bipolar cells light-responsive.
Light-activated bipolar cells send signals to the optic nerve, restoring form vision even though the natural photoreceptors are gone.
| Spec | MCO-010 (sonpiretigene isteparvovec) |
|---|---|
| Indication | Advanced retinitis pigmentosa with severe vision lossR (reported) |
| RESTORE trial enrollment (high-dose arm) | 18 treated, 9 shamR (reported) |
| Vision gain at 52 weeks (high-dose) | ~3 lines (0.337 logMAR vs sham)R (reported) |
| Vision gain at 76 weeks (sustained) | ~5 lines (0.539 logMAR vs sham)R (reported) |
| Functional response at 12 months | 89% on composite testingR (reported) |
| Serious adverse events (2 years) | None treatment-relatedR (reported) |
R reported by the company
Nanoscope Therapeutics
Optogenetic gene therapy, retinal disease, vision restoration
Nanoscope develops optogenetic gene therapies for retinal disease. Founded 2017, Dallas. MCO-010 is the first optogenetic vision therapy with FDA BLA acceptance (Sept 2026); PDUFA H1 2027.
nanoscopetherapeutics.com (opens nanoscopetherapeutics.com)Company profile →
