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Delivering RNAi Therapeutics
An important area of scientific focus at Alnylam is developing approaches for the delivery of small interfering RNAs, or siRNAs — the molecules that mediate RNAi — into diseased tissues and cells. Delivery of siRNAs is one of the most important success factors for the continued advancement of RNAi therapeutics; indeed, when we can successfully deliver therapeutics to sites of disease, the door is opened for the delivery of RNAi therapeutics to address a broad range of human disease.
At Alnylam, we have made significant progress in understanding and developing several different approaches for the delivery of RNAi therapeutics. In fact, delivery solutions for both direct and systemic administration of siRNAs are now accessible for the clinical advancement of this technology.
We have built a very powerful platform around multiple different technologies that have allowed us to very successfully deliver our molecules into cells following in vivo administration. These approaches include conjugates, antibodies, liposomes, and peptides, to name a few. In fact, we have now achieved gene silencing with our siRNAs directed to more than 25 disease targets, in more than 5 tissues, and in 6 species — including humans. Our technology is rapidly emerging as a robust platform that we believe has the potential to rival or even exceed the historical successes of other discovery platforms, such as monoclonal antibodies.
At the same time, it is our belief that different delivery approaches will be required to access different target tissues, and will require a long-term commitment to continue to expand and evolve this technology over time as part of realizing the broadest potential of RNAi therapeutics. Our internal delivery efforts and those with certain key external collaborators, including the MIT and Tekmira, continue to advance Alnylam's efforts for delivery of RNAi therapeutics.