NaV1.1 selective potentiator

NaV1.1 selective potentiators (such as the lead preclinical candidate XPC-837 developed by Xenon Pharmaceuticals) are small molecule positive allosteric modulators of the voltage-gated sodium channel subunit alpha type 1 (NaV1.1, encoded by the SCN1A gene). Loss-of-function mutations in SCN1A reduce NaV1.1 expression in GABAergic inhibitory interneurons, leading to an imbalance of inhibition and excitation that causes Dravet syndrome and other severe epilepsies. By selectively stabilizing the open state and destabilizing the inactivated state of NaV1.1 channels, these potentiators increase interneuron excitability and restore synaptic inhibition, offering a potential disease-modifying precision therapy for Dravet syndrome.

Other names

NaV1.1 selective potentiatorsNaV-1.1 selective potentiatorsNaV 1.1 selective potentiatorsNaV1.1 sodium channel openersNaV-1.1 sodium channel openersNaV 1.1 sodium channel openers