C-TERP-SCN1A antisense oligonucleotide

C-TERP-SCN1A antisense oligonucleotide is a systemically administered, CNS-penetrant antisense oligonucleotide (ASO) therapy developed by QurCan Therapeutics for the treatment of Dravet Syndrome. Dravet Syndrome is a severe form of epilepsy primarily caused by loss-of-function mutations in the SCN1A gene, which leads to sodium channel haploinsufficiency. This therapeutic leverages QurCan's proprietary Target-Engineered Responsive Polymer (TERP) technology, a polymer-lipid hybrid nanoparticle platform designed to facilitate efficient blood-brain barrier (BBB) transcytosis via dual receptor-mediated transport. By delivering the ASO systemically, the drug aims to increase cerebral Nav1.1 protein levels, thereby reducing seizure incidence and severity and preventing sudden unexpected death in epilepsy (SUDEP). Preclinical studies in mouse models have demonstrated that intravenous administration of C-TERP-SCN1A results in significant brain exposure and functional rescue of the Dravet phenotype.

Other names

C-TERP-SCN1AC-TERP-SCN-1AC-TERP-SCN 1A