MCO-010 at 3 years: gene-agnostic gains in retinitis pigmentosa
Bakall on 3-year REMAIN data for MCO-010 in retinitis pigmentosa: durable 3-line acuity gains, why genotype did not matter, and which patients responded best.
Gene replacement therapy for retinitis pigmentosa (RP) reaches only a single genetic subtype: voretigene neparvovec (Luxturna; Spark Therapeutics) is approved for confirmed biallelic RPE65 mutation–associated retinal dystrophy and requires viable retinal cells. For the far larger group of patients whose photoreceptors are already lost, no approved therapy restores vision, and management has largely been limited to low-vision support and disease monitoring. Optogenetics takes a different route, conferring light sensitivity on surviving inner retinal neurons rather than repairing the defective gene.