SLN124

SLN124 is a synthetic, double-stranded short interfering RNA (siRNA) oligonucleotide conjugated to N-acetylgalactosamine (GalNAc) residues for targeted delivery to hepatocytes. It specifically targets and silences the TMPRSS6 gene in the liver, leading to increased endogenous hepcidin production—the master regulator of iron homeostasis. By increasing hepcidin levels, SLN124 reduces plasma iron and transferrin saturation, making it a promising therapy for rare iron-loading anemias such as beta-thalassemia and myelodysplastic syndromes (MDS), as well as myeloproliferative diseases like polycythemia vera. The drug is administered subcutaneously and has demonstrated safety, tolerability, and proof-of-mechanism in phase 1 clinical studies[1][4][5][6]. Developed by Silence Therapeutics, it has received FDA Fast Track Designation for polycythemia vera[3].

Other names

TMPRSS6 small interfering RNATMPRSS-6 small interfering RNATMPRSS 6 small interfering RNAmatriptase-2 siRNAmatriptase2 siRNAmatriptase 2 siRNA