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 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. This strategy leverages Alnylam's clinical progress on siRNA delivery, including recent human proof-of-concept data for systemic delivery.

By 2015, the company expects to have five RNAi therapeutic programs in advanced clinical development. These include ALN-TTR (for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment of treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia.

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.