Alnylam Pharmaceuticals and Collaborators Publish New Pre-Clinical Research on RNAi-Mediated Silencing of PCSK9, a Genetically Validated Regulator of LDL Metabolism

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that new pre-clinical data from its hypercholesterolemia program was published in Proceedings of the National Academy of Sciences (Frank-Kamenetsky, M., et al (August 11, 2008) Proc. Natl Acad. Sci. USA, 10.1073/pnas.0805434105). The published, peer-reviewed data demonstrate for the first time that a chemically synthesized small interfering RNA (or siRNA, the molecules that mediate RNAi) targeting the gene proprotein convertase subtilisin/kexin type 9 (PCSK9) achieves acute and durable lowering of total cholesterol levels in both mice and rats, and LDL cholesterol levels in non-human primates. Human genetic studies have correlated increased levels of PCSK9 with an increased risk of cardiovascular disease (Abifadel et al., Nature Genetics, 34 (2): 154-6; 2003). Conversely, human genetic studies have confirmed a nearly 88 percent reduced risk of cardiac events in subjects with decreased levels of PCSK9 (Cohen et al., New England Journal of Medicine, 354: 1264-72; 2006).
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