Efficacy increase of lipid nanoparticles in vivo by inclusion of bis(monoacylglycerol)phosphate - Supplementary data

Aim: To investigate the effect of incorporating bis(monoacylglycerol)phosphate (BMP) lipid into a
lipid nanoparticle and the functional transport of mRNA by the formulated nanoparticles in vivo.
Materials & methods: The nanoparticles were prepared from ionizable lipid, 1,2-distearoyl-sn-glycerol-3-
phosphocholine, cholesterol, 1,2-dimyristoyl-sn-glycerol PEG 2000, BMP and formulated mRNA encoding
human erythropoietin. We measured the effect of BMP on physicochemical properties and impact on
functional efficacy to transport mRNA to its target cells/tissue as measured by protein expression both in
vitro and in vivo. Results: Lipid nanoparticles composed of BMP displayed increased endosomalmembrane
fusion and improved mRNA delivery to the cytosol. Conclusion: The results establish the foundation for
future development of these nanoparticulated entities by designing new BMP derivatives and correlating
structures to enhanced pharmacokinetic profiles.