We identified four formulations possessing higher intracellular protein expression ability in vitro even after aerosolization which were then assessed in in vivo studies. An increasing molar ratio of poly-(ethylene) glycol (PEG)-lipid significantly decreased size but also intracellular protein expression of mRNA. Generally, upon nebulization, LNP formulations showed increased particle size and decreased encapsulation efficiencies. Lipid nanoparticles (LNPs) showed stable physicochemical properties for at least 14 days of storage at 4 ☌, and most formulations exhibited high encapsulation efficiencies greater than 80%. In this study, we employed a Design of Experiments (DOE) strategy to screen a library of lipid nanoparticle compositions to identify formulations possessing high potency both before and after aerosolization. The development of a stable and efficacious mRNA pulmonary delivery system would enable high therapeutic concentrations locally in the lungs to improve efficacy and limit potential toxicities. Messenger RNA is a class of promising nucleic acid therapeutics to treat a variety of diseases, including genetic diseases.
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