SPVN20

SPVN20 is an adeno-associated virus (AAV) gene therapy developed to restore visual acuity and color vision in patients with late-stage retinitis pigmentosa (RP), specifically those with dormant but viable cone cells. The therapy is mutation-agnostic, meaning it can be used regardless of the underlying genetic cause of RP. Its mechanism involves delivering a gene encoding a variant of the G-protein coupled inwardly rectifying potassium channel (GIRK channel)—normally not expressed in the retina—into cone cell bodies via an IVT-permissive AAV capsid. This reactivates opsin signaling and restores function to non-functional cones by modulating GPCR pathways, aiming to improve light responses even after significant photoreceptor degeneration[1][3][5][8]. The program is currently in preclinical development, with first-in-human trials planned[7].

Other names

Adeno-associated virus based gene therapy SparingVision