ION283

ION283 is an investigational antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals for the treatment of Lafora disease, a rare and fatal form of progressive myoclonus epilepsy. The drug is designed to target and degrade the messenger RNA (mRNA) of the glycogen synthase 1 (GYS1) gene, which encodes the enzyme responsible for glycogen synthesis in the brain. By reducing GYS1 expression, ION283 aims to prevent the overproduction of glycogen and the subsequent formation of toxic polyglucosan aggregates known as Lafora bodies, which are the hallmark of the disease. ION283 is administered via intrathecal injection and has received Orphan Drug Designation in both the United States and the European Union. In 2024, Ionis licensed the global development rights for ION283 to Noventia Pharma.

Other names

GYS1 ASOGYS-1 ASOGYS 1 ASOGys1-ASOGys-1-ASOGys 1-ASO