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Alnylam

Pipeline Growth Strategy for RNAi Therapeutics in Three Strategic Therapeutic Areas, or “STArs”

In December 2014, we announced a pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.  The strategy builds on our progress in the advancement of RNAi therapeutics, including our “Enhanced Stabilization Chemistry” (ESC)-GalNAc conjugate technology, as a modular and reproducible platform for discovery of innovative medicines.  The three STArs will remain focused on liver-expressed and genetically validated or pathogen-derived disease targets, with biomarkers for assessment of clinical activity early in Phase 1 trials.  Across our three STArs, we believe that we can address major unmet needs in a wide range of diseases with high-impact, differentiated medicines, and continue to build what we believe to be one of the most robust pipelines in biotech.  By executing on this strategy, we believe we have the potential to make a meaningful difference in the lives of patients, and maximize value for shareholders.