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:

Below is a rough draft transcript of our conversation.

Alnylum is a company that is focused on creating a whole new class of medicines with a new discovery based on a new discovery in biology called RNA interference or RNAi as it's abbreviated and it's a technology and an approach that we think can transform the treatment of many human diseases because we can silence disease causing genes and basically by turning those genes off temporarily, we can make a difference in people's lives by treating their disease in ways that can't be done with today's medicines.
And the other part of the company, which is quite exciting, is that with what we've been doing over the years now, our technology and innovation is now becoming increasingly a game changer in much of a biomedical discovery, both in industry and in academia. So we've got partnerships with companies like Roche and Novartis and Cicada amongst others, Medtronic as well, and we've also been able to work real closely with major academic institutions like MIT and Plank and Stanford and UT Southwestern and work together on our technology. So it's a company that's hopefully what we believe is going to make a big difference in the treatment of disease with the types of approaches that we're taking. And then at the same time we're finding that we're able to work real closely across the entire biomedical world to basically advance our technology. 
Q: Tell us about your newest scientific advisory board 
That's a group that's focused on are one of the applications of our technology focused on transforming biologics manufacturing. You know, there's a whole range of medicines that are used today that are collectively called the biologic system: includes recombinant proteins like the use of factor eight for hemophilia or monoclonal antibodies like the use of the drug Avastin for cancer. And then also vaccines, and we've recently started up an effort where we're using our technology to fundamentally transform how those types of products are made. And so the group you're referring to was a recent assembly of some world renowned scientists that are going to help advise us on, on our biotherapeutics effort. But Alnylum overall has had a very active scientific advisory board since 2002 with people like a Phil Sharp and Tom and Bob Langer amongst other people that had been advising or company. 
One of the fun things and exciting things about what we've done, is that as we've developed our advanced technology, we've also realized that the role of RNA in general in human biology is far broader than we ever expected. And that allowed us to understand more about micro RNA; these are small RNA that are present in all of ourselves and then we figure out the technologies that allow us to either turn up or turn them down. And by what we've been able to learn is that by sometimes turning them down you can treat specific diseases and sometimes by turning them up you can treat other diseases.

And so a company that we've cofounder with Isis called Regulus. This is an exciting effort that's a focused on micro RNA therapeutics and we and Isis both own 50 percent of that company. So we have a high a retention of the ownership there. We're excited about where that's going and there's a lot of interest in the pharmaceutical industry for where that's going as well.