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Review
. 2024 Mar 23;403(10432):1192-1204.
doi: 10.1016/S0140-6736(23)02444-3. Epub 2024 Mar 7.

mRNA-based therapeutics: looking beyond COVID-19 vaccines

Hamideh Parhiz  1 , Elena N Atochina-Vasserman  1 , Drew Weissman  2
Affiliations
Review

mRNA-based therapeutics: looking beyond COVID-19 vaccines

Hamideh Parhiz et al. Lancet. .

Abstract

Recent advances in mRNA technology and its delivery have enabled mRNA-based therapeutics to enter a new era in medicine. The rapid, potent, and transient nature of mRNA-encoded proteins, without the need to enter the nucleus or the risk of genomic integration, makes them desirable tools for treatment of a range of diseases, from infectious diseases to cancer and monogenic disorders. The rapid pace and ease of mass-scale manufacturability of mRNA-based therapeutics supported the global response to the COVID-19 pandemic. Nonetheless, challenges remain with regards to mRNA stability, duration of expression, delivery efficiency, and targetability, to broaden the applicability of mRNA therapeutics beyond COVID-19 vaccines. By learning from the rapidly expanding preclinical and clinical studies, we can optimise the mRNA platform to meet the clinical needs of each disease. Here, we will summarise the recent advances in mRNA technology; its use in vaccines, immunotherapeutics, protein replacement therapy, and genomic editing; and its delivery to desired specific cell types and organs for development of a new generation of targeted mRNA-based therapeutics.

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Conflict of interest statement

Declaration of interests In accordance with the University of Pennsylvania policies and procedures and our ethical obligations as researchers, we report that HP and DW are named on patents that describe the use of nucleoside-modified mRNA as a platform to deliver therapeutic proteins and vaccines and target lipid nanoparticles to specific organs and cells to deliver therapeutic mRNAs. ENA-V and DW are named on a patent that describes the methods of use of nucleoside-modified mRNA-LNP vaccine for norovirus. We have disclosed those interests fully to the University of Pennsylvania, and we have in place an approved plan for managing any potential conflicts arising from licensing of our patents.

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