RNAi therapeutics company Alnylam Pharmaceuticals, Inc., US, has announced the publication of new data in the journal Proceedings of the National Academy of Sciences (PNAS). The company had worked with collaborators from the David H. Koch Institute for Integrative Research at the Massachusetts Institute of Technology (MIT). The data published in the journal describes further advancements in discovery and development of novel ‘lipidoid’ formulations for the systemic delivery of RNAi therapeutics.
Lipidoids are lipid-like materials discovered for the delivery of RNAi therapeutics, and were originally described by Alnylam and MIT collaborators. In particular, the new research findings demonstrate the discovery of new lipidoid materials that facilitate significantly improved in vivo potency for RNAi therapeutics.
The new pre-clinical data describe a formulation based on a lipidoid known as ‘C12-200’ that was shown to enable gene silencing in vivo in rodents at doses below 0.01 mg/kg; demonstrate complete, rapid, and durable gene silencing in rodents as soon as 24 hours with protein levels returning to baseline within 20 to 35 days; specifically inhibit expression of as many as five target genes simultaneously after a single injection of an LNP formulation in rodents; and demonstrate potent and selective silencing of the clinically relevant gene transthyretin (TTR) at doses as low as 0.03 mg/kg in non-human primates.
PNAS claims to be one of the world's most-cited multidisciplinary scientific serials. It publishes research reports, commentaries, reviews, perspectives, colloquium papers and actions of the Academy. Coverage in PNAS spans the biological, physical, and social sciences. PNAS is abstracted and/or indexed in Index Medicus, PubMed Central, Current Contents, Medline, SPIN, JSTOR, ISI Web of Science and BIOSIS.
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