Advances in development of "lipidoid" formulations for systemic delivery of RNAi therapeutics ❋ Editors (2010)
The presentation described advances in discovery and development of novel "lipidoid" formulations for the systemic delivery of RNAi therapeutics. ❋ Unknown (2010)
Proceedings of the National Academy of Sciences (PNAS) describing further advancements in discovery and development of novel "lipidoid" formulations for the systemic delivery of RNAi therapeutics. ❋ Unknown (2009)
These new lipidoid materials comprise part of Alnylam's second generation lipid nanoparticle (LNP) platform. ❋ Unknown (2010)
In particular, the new pre-clinical research findings demonstrate the discovery of new lipidoid materials that facilitate significantly improved in vivo potency for RNAi therapeutics. ❋ Unknown (2010)
Additional approaches include novel lipidoid formulations, mimetic lipoprotein particles (MLPs), siRNA conjugation strategies, and single-stranded RNAi, amongst others. ❋ Unknown (2010)
These new data describe the further development of lipidoid formulations for the systemic delivery of small interfering RNAs (siRNAs), the molecules that mediate RNAi.
* demonstrating that lipidoid formulations achieve delivery of greater than 90\% of the administered siRNA dose to the liver and maintain robust in vivo activity following repeat administration over a period of several months, and indicating no evidence of neutralizing antigenicity or tachyphylaxis; and,
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 ❋ Unknown (2009)
Nature Biotechnology 26, 561-569 (01 May 2008)), showed successful delivery of siRNAs encapsulated in lipidoid formulations when administered in multiple animal species including mice, rats, and non-human primates.
