Roadmap to discovery and early development of an mRNA loaded LNP formulation for liver therapeutic genome editing

Introduction mRNA therapeutics were a niche area in drug development before COVIDvaccines. Now they are used in vaccine development, for non-viral therapeuticgenome editing, in vivo chimericantigen receptor T (CAR T) celltherapies and protein replacement. mRNAis large, charged, and easily degraded by nucleases. It cannot get into cells,escape the endosome, and be translated to a disease-modifying protein without adelivery system such as lipid nanoparticles (LNPs).