About Alnylam Overview

Alnylam is developing an entirely new class of innovative medicines based on a breakthrough discovery in biology known as RNA interference, or RNAi. With RNAi technology, we have the opportunity to treat disease and impact the lives of patients in a fundamentally new way by silencing disease-causing genes upstream of today's medicines.

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 has additional partnered programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection and ALN-VSP for the treatment of liver cancers. The company's leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, Ascletis, Monsanto, Genzyme, and The Medicines Company. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam's VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts