Nav1.1

Nav1.1

Loss of function of Nav1.1 as a result of genetic mutations results in an aberrant increase in neuronal excitability in the brain leading to seizures. Hundreds of mutations have been identified in the SCN1A gene encoding Nav1.1 and many have been directly associated with seizure disorders or predicted to be pathogenic. Furthermore, dysfunction of Nav1.1 many play a role in certain deficits in cognition processes. Ligand has established a state-of-the-art drug discovery platform for Nav1.1 and identified novel chemical matter that enhances the function of Nav1.1 in cellular assays. These compounds can potentially be optimized leading to treatments for rare epilepsies and rescue of cognitive disorders.