Running interference into the clinic

Writing a story on therapeutics based on RNA interference, so far I’ve interviewed half a dozen experts, most of them research executives at biotechs leading that charge. There’s a steady stream of early clinical trials with RNAi drugs, almost all targeting liver-expressed genes. In the last week of May, for example, Tekmira launched a phase 1/2 trial for liver cancer and Benitec dosed the first patient in a phase 1 hepatitis B trial. The experts emphasize that animal models have proved highly predictive in studying the effects of RNAi and claim that the delivery obstacles that plagued the first wave of RNAi drugs have been overcome, at least for the liver. They also suggest that regulatory authorities, so far, have been pleased by the clear biomarkers and consistent data available for RNAi agents. Of course, everyone is also properly guarded at this stage of clinical research. I’m looking forward to getting more perspectives on June 13 at the Koch Institute’s symposium on RNA biology, cancer and therapeutics.

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