Harnessing RNAi as a gene silencer

Drug delivered: Gene-silencing RNAi
Innovation: Delivering RNAi in clinical trials
Research organizations: Alnylam Pharmaceuticals, Vall d'Hebron Institute of Oncology

Alnylam Pharmaceuticals ($ALNY) and the Vall d'Hebron Institute of Oncology (VHIO) in Spain are working together to improve the delivery of the potentially powerful genetic RNAi drug concept.

Using RNAi drugs would "silence" certain traits by interfering with the genetic code--for instance, by attaching to the gene that codes for a specific type of cancer, RNAi can turn that expression off, so to speak. But getting those drugs there isn't easy.

Alnylam and VHIO used nanoparticles in a Phase I clinical trial to get the RNAi drug to silence genes that express cancer-inducing proteins. The nanoparticles allow the RNA to enter the nucleus of the cell in high concentrations while retaining its all-important shape. In the 41-patient trial in patients with metastasized liver cancer, the drug showed promising results.

The delivery of RNAi drugs is big news in the pharma industry, as many large companies have tried to do it and cut back, including Novartis ($NVS), Merck ($MRK) and Roche ($RHHBY).

Harnessing RNAi as a gene silencer

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