Drug intelligence / Profile preview

SPVN06

Development stage
Phase 2
Lead developer
SparingVision
Modality
Gene Therapies
Administration
Subretinal
01

Overview

SPVN06 is a gene therapy developed by SparingVision for the treatment of inherited retinal diseases, primarily targeting retinitis pigmentosa (RP) and cone-rod dystrophies. It is designed to slow or stop the degeneration of cone photoreceptors, which can lead to blindness. The therapy works by delivering DNA encoding two isoforms—Rod-derived Cone Viability Factor (RdCVF), a neurotrophic factor that supports cone survival, and RdCVFL, an antioxidant enzyme that protects cones from oxidative stress—using an adeno-associated virus (AAV) vector via subretinal injection. This mutation-agnostic approach aims to provide benefit regardless of the patient’s genetic background and may also be applicable in dry age-related macular degeneration (AMD). As of early 2024, SPVN06 is in Phase 2 clinical trials for RP and cone-rod dystrophies[1][2][3][4][5][6][7][8].

Other names
RdCVFrod-derived cone viability factorRdCVFLrod-derived cone viability factor longNXNL1 gene therapyNXNL-1 gene therapyNXNL 1 gene therapy
02

Targets

RdCVF (Rod-derived cone viability factor)RdCVF–BSG1 (Rod-derived cone viability factor–Basigin-1 protein-protein interface)

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