Messenger RNA (mRNA)-based immunotherapies and protein in situ production therapies hold great promise for addressing theoretically all the diseases characterized by aberrant protein levels. The safe, stable, and precise delivery of mRNA to target cells via appropriate pharmaceutical strategies is a prerequisite for its optimal efficacy. In this review, we summarize the structural characteristics, mode of action, development prospects, and limitations of existing mRNA delivery systems from a pharmaceutical perspective, with an emphasis on the impacts from formulation adjustments and preparation techniques of non-viral vectors on mRNA stability, target site accumulation and transfection efficiency. In addition, we introduce strategies for synergistical combination of mRNA and small molecules to augment the potency or mitigate the adverse effects of mRNA therapeutics. Lastly, we delve into the challenges impeding the development of mRNA drugs while exploring promising avenues for future advancements.
Keywords: Pharmaceutical strategy; Stability; Target site accumulation; Transfection efficiency; mRNA.
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