Friday, June 11, 2010

RNAi offers hope for drug discovery

As scientists continue to investigate the human genome, where we now have the complete genetic sequence, there have been remarkable developments in the understanding of disease and consequently a large increase in the number of molecular disease targets to impact a broad range of human disease. The discovery of RNAi is a major breakthrough in biology, and this technology has the potential to make a broad and significant impact. A company called Alnylam Pharmaceuticals is leading the effort in translating the science of RNAi into a robust drug discovery capability. With RNAi it is possible to target virtually any gene in the human genome involved in the causal pathway of disease. The possibility of targeting these previously "undruggable" targets with RNAi is transformative for new drug discovery.

I spoke to Alnylam's CEO John Maraganore:

There was an excellent webcast this week you can access here: (registration required)