CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced the publication of a new study in Nature Biotechnology by Alnylam scientists and collaborators from the David H. Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology (MIT). The new research (Akinc et al., Nature Biotechnology advance online publication, 27 April 2008 (DOI:10.1038/nbt1402)) documents the design and synthesis of a new class of lipid-based molecules called “lipidoids”, which were used to form novel nanoparticle formulations for systemic delivery of RNAi therapeutics. The results of the studies showed successful delivery of lipidoid formulations of small interfering RNAs (siRNAs), the molecules that mediate RNAi, in multiple animal species including mice, rats, and non-human primates that together demonstrate potent, specific, and durable effects on gene expression in multiple tissues, including liver, lung, and peritoneal macrophages. Further, the new paper demonstrates applications of the same technology for delivery of microRNA (miRNA) antagonists.
