RC001

RC001 is a first-in-class RNA editing therapeutic developed by RecoRNA Bio for the treatment of Dravet syndrome (DS). It utilizes a chemically optimized guide RNA to recruit endogenous adenosine deaminases acting on RNA (ADAR) enzymes to specific sites on *SCN1A* transcripts. This targeted editing is designed to upregulate the expression of the voltage-gated sodium channel Nav1.1, addressing the underlying genetic haploinsufficiency caused by loss-of-function mutations in the *SCN1A* gene. Preclinical studies in mouse models of Dravet syndrome demonstrated molecular correction, reduced seizure frequency, and improved biomarkers. Safety and pharmacodynamic profiles have been evaluated in rats and non-human primates, showing broad distribution of editing activity across brain regions. Based on these findings, a first-in-human investigator-initiated study was launched in China to evaluate its safety and therapeutic potential in children with Dravet syndrome.