Standardization of Lipid Nanoparticle Design for Targeted Drug Delivery in Multiple Organs through a Compounded Formulaic Approach

Abstract

There are many different diseases that affect a variety of different organs which makes designing drug compounds for their treatment very difficult. However, if the disease were treated using LNPs (lipid nanoparticles) and supplemental administration techniques that were developed specifically for the respective organs then we would be able to create a starting library for LNP constituents and supplemental techniques that could be utilized across a variety of organs. Lung-related diseases could utilize nebulized drug delivery 1 (NLD1) LNPs, brain-related diseases could utilize acid-degradable LNPs (AD-LNPs) with microbubble-FUS (focused ultrasound), and bone-related diseases could utilize aspartic acids LNPs (ASP6-LNPs). The LNP selection is a starting template for treatment. These diseases would incorporate relevant mRNA in their LNP encapsulation for the treatment of pneumothorax, glioblastoma, and osteogenesis imperfecta. The selection of mRNA can be switched to address different genetic anomalies seen within alternate diseases that affect the same organs. This would enable the exploration and experimentation of diseases that affect smaller disease populations as preliminary research in targeting would have already been established thus presenting a formulaic approach for the development of a therapy.