SMARTT Polymer Technology® Targets and Delivers mRNA to the Liver
PhaseRx SMARTT Polymer Technology® has previously been shown to effectively deliver siRNA to hepatocytes in the liver using an N-acetylgalactosamine targeting group. For mRNA delivery, a similar targeted multi-domain polymer has been developed. The mRNA and polymer self-assemble into a 70 nm nanoparticle. The mRNA condensation assessed by SYBRGold assay showed that 26% of the mRNA is dye inaccessible, indicating a strong interaction between the polymer and the mRNA. The mRNA formulation is freeze-thaw stable and imparts serum stability to the mRNA. Mice injected intravenously with polymer formulated FLUC mRNA showed luciferase expression specific to the liver 2-3 logs above background.
PhaseRx, Inc., is a privately held RNAi delivery technology company focused on enabling the broad development of RNAi therapeutics, a new class of pharmaceuticals with significant market potential, which has been hindered to date by the lack of effective delivery technology. PhaseRx was founded to solve a critical challenge in RNAi delivery: the need for a versatile, developable system that can be administered systemically and deliver RNAi drugs into the cell where they can reach and inhibit the drug targets. PhaseRx is deploying an integrated delivery system based on novel synthetic polymers that meets these challenges and delivers RNAi drugs into the cytoplasm where they can reach and inhibit the desired target of interest. This proprietary delivery system represents a breakthrough technology for the development of RNAi-based drugs.