CMP-SCN

**CMP-SCN** is an investigational antisense oligonucleotide (ASO) developed by CAMP4 Therapeutics using its RNA Actuating Platform to upregulate endogenous **SCN1A** gene expression for the treatment of **Dravet syndrome**, a severe genetic epilepsy caused by **SCN1A** haploinsufficiency. It targets the **SCN1A natural antisense transcript (SCN1A-NAT)**, a regulatory RNA that suppresses **SCN1A** transcription, leading to increased **SCN1A** mRNA and protein levels (1.5-2 fold in non-human primate brain regions). Preclinical data demonstrate **25% SCN1A** upregulation in Dravet mouse models with **~70% reduction** in seizure number, frequency, amplitude, and duration, alongside wide CNS distribution and good tolerability following intrathecal administration. Licensed from **OPKO Health**, it was advancing toward IND filing by end-2022 (delayed to mid-2023 per funding reports).[1][2][3][7][8]