The data indicated that a single in vivo administration of MDRNA meroduplexes targeting apolipoprotein B mRNA formulated in a DiLA2 delivery vehicle showed robust activity in a mouse model.
The meroduplex siRNA decreased both ApoB mRNA and serum cholesterol levels. In addition, the formulated, chemically modified siRNA resulted in minimal body weight loss in mice while showing reduced levels of cytokine stimulation.
Michael Houston, vice president of chemistry and formulations at MDRNA, said: “The results of this preclinical work suggest that a unique combination of both a novel siRNA construct within a lipid-based delivery formulation has the potential to elicit a potent RNAi response and reduce off-target activity.”