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Alnylam is working to build a deep pipeline of RNAi therapeutics, generated from a proprietary combination of technologies referred to as our "product engine". This technology platform for RNAi therapeutics integrates broadly applicable technologies that could possibly be applied to a variety of different siRNAs to create a broad range of drug products.

With the goal of accelerating the development of RNAi therapeutics, Alnylam is implementing a phased approach to product development. Using the current capabilities of our product engine, we are working to initiate development of Direct RNAi™ products - RNAi therapeutics that will be administered directly at sites of diseases, focusing on areas such as ocular, respiratory and central nervous system.

Alnylam is also working to extend its capabilities to enable the development of Systemic RNAi™ therapeutics, drugs that travel through the bloodstream to reach diseased parts of the body. These products may have the potential to treat a broad range of diseases including oncology, inflammation, metabolic and infectious diseases.

Alnylam has demonstrated world-wide scientific leadership in developing delivery solutions for Systemic RNATM therapeutic applications.

Nature

Alnylam's November 2004, Nature article demonstrated endogenous gene silencing with RNAi following systemic administration. Alnylam's Nature article was the first ever demonstration of such gene silencing via systemic delivery and was heralded as a landmark achievement in the field.


Nature

Within eighteen months, in March 2006, Nature published further research by Alnylam scientist demonstrating, in primates, that a systemically delivered RNAi therapeutic can potently silence an endogenous disease-causing gene in a clinically relevant manner. This research represents a major advance because it suggests that an RNAi therapeutic can be effective when delivered systemically using a dosage appropriate for application in future human clinical studies.