CUR-1916

CUR-1916 is an investigational synthetic single-stranded oligonucleotide therapy developed to treat Dravet syndrome, a severe genetic epilepsy. It utilizes the AntagoNAT technology platform to specifically bind and suppress SCN1ANAT, a natural antisense transcript that inhibits expression of the healthy SCN1A gene. By blocking SCN1ANAT, CUR-1916 increases production of functional sodium channel protein from the remaining healthy copy of SCN1A in patients with Dravet syndrome. This approach aims to address both seizure symptoms and underlying disease mechanisms by restoring more normal levels of Nav1.1 protein in neurons[1][2][4][10].

Other names

CUR-1916CUR1916CUR 1916OPK88001OPK-88001OPK 88001