The brief
- MCO-010 is gene-agnostic optogenetic therapy for retinitis pigmentosa of any genetic cause.
- RESTORE trial: high-dose arm gained 3 lines at 52 weeks, sustained 5 lines at 76 weeks. 89% functional improvement.
- FDA BLA accepted Sept 2026. PDUFA decision expected H1 2027.
Technical approach
Gene-agnostic design
Works for any genetic cause of RP
Unlike therapies targeting specific mutations, MCO-010 encodes a synthetic opsin for bipolar cells regardless of what broke photoreceptors.
Optogenetic protein
Synthetic broadband light sensor
A synthetic opsin engineered to respond across visible wavelengths, enabling light sensing without photoreceptors.
Delivery
One-time intravitreal injection
A single dose delivered directly into the eye; the vector crosses the retina to transduce surviving bipolar cells, which then become light-sensitive.
Mechanism
Bipolar cell sensitization
In RP, rods and cones die but bipolar cells survive. Opsin genes make bipolar cells light-responsive and signal the optic nerve.
