4A3-SC8 is a modular dendritic macromolecule with cationic and lipophilic groups that enables small RNAs to prolong survival in an aggressive hepatocellular carcinoma model and can be used to synthesize lipid nanoparticles.

4A3-SC8 (100 mg/kg) is a biodegradable dendrimer with low toxicity and high delivery efficiency in mice carrying invasive liver cancer[1].

After intravenous injection of 1 mg/kg siRNA, 4A3-SC8 mediated the primary accumulation of siRNA in the liver, with lesser amounts in the spleen and kidneys, and resulted in greater than 95% knockdown of FVII in mice [1]. Following administration of 4A3-SC8 (100 mg/kg with 4 mg/kg control siRNA siCTR; i.v.; once), the mice exhibited illness and a significant weight loss of nearly 20% on the first day post injection [1].