elsunersen

Elsunersen (formerly PRAX-222) is an antisense oligonucleotide (ASO) therapeutic being developed by Praxis Precision Medicines, in collaboration with Ionis Pharmaceuticals and RogCon, for the treatment of SCN2A developmental and epileptic encephalopathy (SCN2A-DEE). It is specifically designed to target and reduce the expression of the SCN2A gene and its encoded protein, the voltage-gated sodium channel Nav1.2. In patients with early-onset SCN2A-DEE, gain-of-function mutations lead to overactivity of Nav1.2, resulting in severe seizures and developmental delays. By lowering SCN2A mRNA and protein levels, elsunersen aims to restore balance to neuronal excitability and serve as a disease-modifying treatment. The drug is administered intrathecally and has received Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as PRIME designation from the EMA.

Other names

PRAX-222PRAX222PRAX 222elsunersen