Alnylam Pharmaceuticals in collaboration with the Massachusetts Institute of Technology (MIT) published new information explaining an innovative technique to deliver RNAi therapeutics systemically with the help of synergistic blends of innovative lipid-like substances known as “lipidoids.”
The findings were released in the Molecular Therapy journal. The research results showed that binary blends of lipidoids can be combined in a single lipid nanoparticle (LNP) structure to deliver synergistic gene silencing in vivo and in vitro.
The study involves preparing 3,500 LNP formulations and determining their impact on gene silencing. The results of the study proved that synergistic gene silencing can be obtained by using materials that integrated productive endosomal escape as well as efficient cellular uptake. The pre-clinical information demonstrated that inefficient single lipidoids could be combined together to activate robust silencing of the Factor VII mRNA in vivo and the luciferase mRNA in vitro. It is expected that this binary formulation technique can be used with any siRNA delivery material that uses blends of special components to carry out different steps in the delivery process.
Kevin Fitzgerald, Senior Director of Research at Alnylam stated that the data obtained formulates original techniques to deliver RNAi therapeutics in a systematic manner. He added that the published data demonstrates that binary blends of some lipidoids will integrate balanced lipid nanoparticle formulations with delivery properties of synergistic siRNA.
Source: http://www.alnylam.com/