LSP-SCN8

LSP-SCN8 is a splice modulating oligonucleotide (SMO) and antisense oligonucleotide drug developed by LifeSplice Pharma for the treatment of Dravet syndrome, a severe pediatric epilepsy. The drug works by directing alternative splicing of the SCN8A gene, which encodes the sodium channel protein type VIII alpha subunit (Nav1.6), leading to reduced expression or production of a non-functional version of this protein. By decreasing functional Nav1.6 in excitatory neurons, LSP-SCN8 aims to counterbalance neuronal hyperexcitability caused by loss-of-function mutations in SCN1A that underlie Dravet syndrome. Preclinical studies have shown that LSP-SCN8 can dramatically reduce seizures and mortality in mouse models of Dravet syndrome[1][2][3][4][5].