s overarching strategy, the DPCs used in ARC-520 employ active ligand-mediated targeting specific to a
receptor on hepatocytes. This approach results in high-potency knockdown of a target gene, validated in mice, rats, and non-human primates and it has demonstrated a low toxicity profile in primates enabling long term dosing.
"The promotion of ARC-520 as a clinical candidate, so soon after acquiring the Roche RNAi assets, represents an important milestone for Arrowhead," said Dr.