GalNAc Delivery

Overview

N-acetylgalactosamine sugar ligand conjugated to oligonucleotides for targeted hepatocyte delivery via ASGPR receptor, enabling the portfolio of approved siRNA and ASO drugs targeting the liver and liver-produced proteins. GalNAc is a sugar ligand modified to bind the asialoglycoprotein receptor on hepatocytes, enabling precise delivery of siRNA and ASO drugs specifically to liver cells and catalyzing a wave of approved drugs targeting liver-produced proteins. The major investor enthusiasm upswing in the oligonucleotide field correlates closely with GalNAc delivery proving efficacy for liver targeting; it is seen as a proof-of-concept that targeted delivery unlocks investment cycles for new modalities. (Segment 3 (10:00-15:00))