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About Alnylam Overview
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. Alnylam is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers.
The meaning of our name, Alnylam
Located in the constellation Orion, Alnylam is the name of the center star of Orion's belt. The star has a luminosity that is 250,000 times greater than the sun, representative of the potential strength that RNAi therapeutics could bring to bear for human health.
In January 2011, we launched our "Alnylam 5x15" product strategy. The "Alnylam 5x15" strategy establishes a path for development and commercialization of novel RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need.
Products arising from this initiative share several key 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 RNAi delivery platform; the opportunity to monitor an early biomarker in Phase I clinical 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 paths for commercialization.By the end of 2015, we expect to have five such RNAi therapeutic programs in clinical development, including programs in advanced stages, on its own or with a partner. The "Alnylam 5x15" programs include: ALN-TTR02, an intravenously delivered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting TTR for the treatment of ATTR in patients with familial amyloidotic cardiomyopathy (FAC); 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 ALN-CC5 for the treatment of complement-mediated diseases, and other programs.
Alnylam has additional partnered programs in clinical or development stages, including ALN-VSP for the treatment of liver cancers.
Our leadership position on RNAi therapeutics and intellectual property have enabled us 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. We 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 VaxiRNATM 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 New England Journal of Medicine, Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.