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Alnylam Pharmaceuticals Inc., and MIT have announced discoveries related to a novel way to deliver RNAi therapeutics.
The Cambridge-based biotechnology company and collaborators from the David H. Koch Institute for Integrative Research at MIT published new data in the journal Proceedings of the National Academy of Sciences describing advancements in the discovery of so-called lipidoids, or lipid nanoparticles, to help deliver RNAi therapeutics.
Lipidoids are lipid-like materials identified as a vehicle for the delivery of RNAi therapeutics and originally described by Alnylam and MIT collaborators in research published in 2008 .
The study found that these lipid nanoparticles could silence several different genes in rodent liver targets. Researchers say this means they could potentially develop one drug that could attack a disease on several different fronts simultaneously. The lipid nanoparticles were also found to increase the potency of the RNAi therapeutic. This would enable researchers to use lower doses of the potential drugs.
“We are excited by the delivery performance of these new formulations,” said Daniel Anderson, Ph.D. of the Koch Institute, in a statement. “This work demonstrates that doses measured in micrograms per kilogram can provide potent gene silencing with RNAi in several species including primates. This greatly improved efficacy allows us to dramatically decrease the dose levels of LNPs, thereby widening the therapeutic index, and also opens the door to formulations that can simultaneously inhibit multiple genes or pathways.”
RNAi is a newer area of biotechnology that seeks to better understand how genes can be turned off and on in cells, to create a new approach in drug development by shutting off genes that cause diseases. Researchers and biotechnology companies have thus far found it a challenge to develop delivery methods for these potential drugs.
Alnylam recently was one of 28 life sciences companies awarded a total of $25 million in tax incentives by the Massachusetts Life Sciences Center.
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