Ready-To-Use Lipid-Based Nanoparticles Solution as an Innovative Alternative to LNPs for the Delivery of RNA Therapeutics

Ionizable lipid nanoparticles (LNPs) have been widely used for in vivo delivery of RNA therapeutics with the particularity that they predominantly end up targeting the liver. The current challenge is the use of commercially available lipids to achieve specific formulations that can ensure wider biodistribution of the RNA once delivered to target different organs and cell types mRNA/in vivo-jetRNA^®+ nanoparticles and LNPs were characterized by DLS to assess size and zeta potential and mRNA encapsulation efficiency was assessed using the RiboGreen assay. Here we demonstrate how our ready-to-use in vivo-jetRNA^®+ transfection reagent can efficiently deliver mRNA to different organs. Firstly, we evaluated in vivo biodistribution using both systemic and local administration routes (intravenous, intraperitoneal and intramuscular). Secondly, we performed an in vivo toxicity study in mouse post-delivery of mRNA-in vivo-jetRNA^®+ nanoparticles. In addition to biodistribution, efficacy and toxicity, we assessed the stability of mRNA encapsulated within in vivo-jetRNA^®+ when stored at 4 °C or room temperature for 1 month.