Alnylam Pharmaceuticals, Inc. (ALNY) Morgan Stanley Global Healthcare Conference September 11, 2012, 01:30 pm ET Executives John Maraganore - CEO Mike Mason - VP, Finance Analysts Dave Friedman - Morgan Stanley Presentation Dave Friedman - Morgan Stanley
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I think we've now clearly demonstrated that RNAi does work in humans; RNAi is the foundation for generating new medicines and we're excited to continue to executive on that plan and advance programs with a real focus on our transthyretin amyloidosis program and also on our Hemophilia program where we are targeting anti-thrombin as the foundation for building an exciting company going forward.Dave Friedman - Morgan Stanley So maybe if we can just start with one broad question and you guys have been around for a while and your progress appears to be you know accelerating every year. So if you can maybe just highlight from a technological standpoint the one or two key aspects over the last year or two in terms of making RNAi more and more drug-like and deliverable that you think people should understand the best in terms of understanding where you guys are headed with this? John Maraganore Yeah, I think there really are two things David that are notable; one is that, you know we've really industrialized and advanced lipid nanoparticle technology in clinical studies, these are liposomal formulation of small interfering RNAs and we've made substantial improvements in those molecules, in those delivery formulation that enable an appropriately robust knock down of target genes in animals and also in humans with a good therapeutic of index, which is important. The second part, the second key thing has been the advancements in so called conjugate sRNA technology which are in many ways simpler molecules. These are small molecules that are handed out to the small interfering RNA and mediate receptor uptick of these molecules of the cells and those data have panned out beautifully in animals system right now and we’re about to go to human trials also in our TTR program. So, both platforms with the nanoparticles, but also conjugate base delivery of small interfering RNAs really have been the key things that have enabled the advancement over the last couple of years. Of course before that let’s not forget the important underlying aspect of RNAi which is these double stranded RNA molecules and as triggers of this biology are incredibly potent and obviously have served us well in our overall drug discovery efforts.