GalNAc siRNA#5

GalNAc siRNA#5 is a GalNAc-conjugated, chemically modified small interfering RNA (siRNA) designed to target and silence claudin-3 (CLDN3), a key tight junction protein expressed in hepatocytes and cholangiocytes. Developed by the University of Bern, the Agency for Science, Technology and Research (A*STAR) in Singapore, and its spin-out CALDRE, the therapeutic is designed to treat cholestatic and fibrotic liver diseases, including progressive familial intrahepatic cholestasis type 3 (PFIC3), primary biliary cholangitis (PBC), and primary sclerosing cholangitis (PSC). By downregulating claudin-3, the siRNA modulates the paracellular barrier between liver cells, which enhances bicarbonate-rich bile flow, reduces toxic bile acid accumulation, and protects liver tissue from necrosis and progressive fibrosis. In preclinical models of PFIC3 (Abcb4-deficient mice), subcutaneous administration of GalNAc siRNA#5 significantly reduced serum markers of liver injury (such as ALT, AST, ALP, bilirubin, and plasma bile acids) and prevented hepatic necrosis and fibrosis.

Other names

ClagalsiRclaudin-3 siRNAclaudin3 siRNAclaudin 3 siRNA