Product Platform

Our strategy at Alnylam is to lead the translation of the science of RNAi into a robust drug discovery capability and to build a significant product pipeline of innovative medicines that are generated from a proprietary combination of technologies referred to as our "product platform." Our efforts are well underway in a time when the pace of biomedical discovery is faster than ever and the hunger for innovative medicines is greater than ever.

The power and potential of RNAi is that it allows for the targeting of virtually any disease-causing gene in the genome. In order to prioritize our efforts, we focus on disease areas with significant unmet medical need, where the biology around the target is well characterized and understood, and where our delivery technologies can achieve efficient and specific effects.

In 2011, we launched our "5x15" strategy with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for transthyretin-mediated amyloidosis (ATTR), ALN-PCS for severe hypercholesterolemia, and ALN-HPN for refractory anemia. As part of this initiative, we expect to have 5 RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for respiratory syncytial virus (RSV) infection, ALN-VSP for liver cancers, and ALN-HTT for Huntington's disease.