ION337

ION337 is an investigational antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of Dravet syndrome, a severe, rare, genetic form of epilepsy. The drug utilizes Ionis' proprietary technology to enhance the potency and durability of ASOs that modulate splicing. Its mechanism of action involves targeting the SCN1A gene to ameliorate aberrant inclusion of poison exons, thereby aiming to restore the production of functional NaV1.1 protein and neuronal function. This approach seeks to address the underlying genetic cause of Dravet syndrome, which is often associated with pathogenic variants in SCN1A.