siJAK1

siJAK1 is a small interfering RNA (siRNA) therapeutic candidate designed to selectively silence the expression of Janus kinase 1 (JAK1). Developed under the European Union's Horizon 2020 NEWDEAL project, siJAK1 is intended for the localized treatment of inflammatory bowel disease (IBD). The therapeutic approach involves complexing the siRNA with lipid nanoparticles (such as CL40) to facilitate delivery to colonic phagocytic cells, including macrophages and neutrophils, via intrarectal administration. By inducing the degradation of JAK1 mRNA within the intestinal mucosa, the drug aims to minimize systemic immunosuppression typically associated with oral small-molecule JAK inhibitors. Preclinical studies in mouse models of dextran sulfate sodium (DSS)-induced colitis have demonstrated successful reduction of JAK1 mRNA levels, although this specific target silencing did not result in significant amelioration of intestinal inflammation in that model.

Other names

JAK1 siRNAJAK-1 siRNAJAK 1 siRNAJanus kinase 1 siRNA