Background

SCN8A is a neuronal sodium channel gene with a role in regulation of neuronal excitability. Mutations in SCN8A lead to encephalopathy and severe recurrent seizures. Individuals with SCN8A encephalopathy often have their first seizure by 4 months of age and subsequently have poor development remaining nonverbal and nonambulatory. SCN8A induced seizures are also resistant to current epileptic therapeutics.

Technology Overview

This technology consists of an antisense oligonucleotide (ASO) method to target SCN8A mutations. This ASO was successfully used in a mouse model of SCN8A encephalopathy to delay seizure onset and increase survival (). Additionally, this ASO has proven effective in treating a related mutation on SCN1A, which causes a different epilepsy known as Dravet Syndrome. This may be due