GalNAc (N-Acetylgalactosamine) technology has been successfully used for liver-targeted siRNA delivery in many RNAi biotech companies.
It has revolutionized the siRNA therapeutics field, which was recently demonstrated by the FDA approval of Givosiran, the world’s first-ever GalNAc-conjugated RNAi therapeutic for the treatment of patients with acute hepatic porphyria. Several other GalNAc conjugates are in the advanced clinical trials to treat a wide variety of diseases. The GalNAc conjugate is a small carbohydrate moiety attached to the end of the siRNA. It enables siRNA uptake by the liver hepatocytes by binding with high affinity and specificity to the asialoglycoprotein receptor (ASGPR).
To further expand our pipelines beyond the locally administrable diseases, OliX Pharmaceuticals has recently secured GalNAc platform to treat various liver diseases, including liver fibrosis and NASH.