IDEA #2NVH37 Nanoformulation Technology for Efficient Delivery of mRNA and DNA vaccines and therapeutics

The invention provides composition and methods for the accelerated preparation of biologically active mRNA or DNA vaccines and therapeutics as aqueous suspensions or lyophilized powders that retain stability and efficacy at ambient temperatures. Manufacture of current mRNA vaccines (for example the Pfizer-BioNTech’s BNT162 and Moderna’s mRNA-1273 COVID-19 vaccines) involves complex pharmaceutical formulations with exacting cold chain requirements for vaccine production, storage and transport. Existing formulations comprise of lipids and polymers to encapsulate the nucleic acid into microparticles suitable for systemic delivery. The complex multicomponent nature of the formulation contributes to higher production costs, whereas our two-component system is easier and faster to assemble. The lipid composition of existing technologies contributes to instability of the formulations at ambient temperatures, whereas our technology contains no lipids or liposomes and as such is more stable at refrigeration or room temperatures. Existing formulations have relatively large (high micromolar) particle sizes, whereas our nanoparticles are closer in size to the 100 nm range that characterizes nanoparticles that are more avidly endocytosed by mammalian cells. To effect intracellular release of the nucleic acid payload following cellular uptake ofthe medicated nanoparticles, liposomal formulations depend on slow disintegration of the matrix and/or passive diffusion of the entrapped nucleic acid out of the matrix, whereas our micellized protein matrix (MPM) nanoformulation is relatively stable at extracellular pH while disintegrating readily within the acidic intracellular environment to release essentially all of the payload. Moreover, our technology uses recombinant human proteins that can be produced to industrial scale and used for systemic administration in humans with less concern for immune reaction or rejection. As a platform technology, MPM nanotechnology can be quickly applied to new viral variants as well as to other infectious microbes or therapeutic goals.