Alnylam and Collaborators at MIT Publish Research on the Discovery of 'Lipidoids'

Novel Class of Lipid-Based Molecules for the Delivery of RNAi Therapeutics

02-May-2008

Alnylam Pharmaceuticals, Inc. 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 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.

In the new research, Akinc et al. describe the discovery and synthesis of a novel class of lipid-based molecules called "lipidoids". These lipidoids were used to form entirely new formulations of siRNAs, enabling their delivery to a broad range of tissues in vivo. The lipidoid molecules were created through a new combinatorial synthesis scheme that allows for simple, high-speed production. The rapid synthesis enables the development of a large library of over 1,200 structurally diverse lipidoids, which can ultimately be customized for different RNAi therapies and drug delivery approaches. In an exclusive license agreement with MIT, Alnylam has secured all rights to the lipidoid technology for delivery of RNAi therapeutics for all uses.

In the study, researchers described the discovery of lipidoids and evaluated the delivery of lipidoid formulations with siRNA and anti-miRNA oligonucleotides in multiple animal species including mice, rats, and non-human primates. The lipidoid formulations demonstrated potent, specific, and durable effects on gene expression in multiple tissues, including liver, lung, and peritoneal macrophages.

Further, the lipidoid formulations also proved effective for delivery of anti-miRNA oligonucleotides or "antagomirs" that are used to suppress miRNA activity. Also, the delivery technology was used to successfully deliver two different siRNAs at the same time, with no apparent competition between the two siRNAs, demonstrating the potential of a multi-targeting strategy for formulations of RNAi therapeutics.

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