Alnylam 5x15 Programs

In January 2011, we launched our "Alnylam 5x15™" product strategy focused on the development of innovative RNAi therapeutics for genetically defined diseases addressing major unmet medical needs.

The "Alnylam 5x15" strategy strategy establishes a path for development and commercialization of novel RNAi therapeutics to address genetically defined diseases with high unmet medical need. Products arising from this initiative share several critical characteristics including: a genetically defined target and disease; the potential to have a major impact in a high unmet need population; the ability to leverage the existing Alnylam delivery platform; the opportunity to monitor an early biomarker in Phase I trials for human proof of concept; and the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated path for commercialization.

By the end of 2015, we expect to have five RNAi therapeutic programs in advanced clinical development, including programs in advanced stages, on its own or with a partner. These include ALN-TTR (for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD), ALN-PCS for the treatment of hypercholesterolemia, ALN-AS1 for the treatment of acute intermittent porphyria (AIP), ALN-TMP for the treatment of treatment of beta-thalassemia and iron-overload disorders, and other programs.

Alnylam intends to focus on developing and commercializing certain programs from this product strategy itself in the United States and potentially certain other countries; we will seek development and commercial partners for other core programs both in the United States and in other global territories.