Germany's atugen, a specialist in RNA interference technologies for switching off genes, has demonstrated the feasiblity of delivering a small interfering RNA (siRNA)-based drug and achieving a physiological response in animals.
In a series of repeat studies to test glucose tolerance in normal rodents, atugen's siRNA therapy was shown to be effective in regulating blood sugar levels. The results suggest that atugen's stabilised siRNA molecules can be delivered to their target cells after injection.
The compounds have already found widespread use as research tools, because they ceb specifically turn off the expression of a single protein by a gene. However, delivery has been held up as one of the major obstacles to the advancement of siRNA as a therapeutic technology.
In atugen's study, treatment with stabilised siRNA molecules (atuRNAi) via an intravenous infusion led to downregulation of a target which plays a significant role in regulating glucose metabolism in vivo.
Compared to the control group, atuRNAi produced lower peak glucose levels with a return to near normal levels after two hours. In contrast, in animals treated with inactivated siRNA molecules, glucose levels rose to very high levels and failed to revert to normal within two hours.
The preclinical in vivo studies made use of a type 2 diabetes disease model in which an insulin-independent signalling pathway was turned on.
atugen said it is now moving on to conduct animal studies of its atuRNAi molecules in various cancer models.