Messenger RNA (mRNA) is emerging as a new tool for therapeutic intervention, providing a transient, programmable platform for the treatment of complex diseases. Unlike DNA-based therapies, mRNA does not integrate into the genome and is considered more safe. The success of mRNA vaccines, combined with advances in mRNA technology and unmet needs in cardiovascular disease (CVD), has led to increased interest in expanding the use of this technology beyond infectious diseases. However, there are several challenges to overcome, including mRNA stability, delivery efficiency, immunogenicity, and regulatory standardization. This review examines advances in mRNA design, delivery platforms, and therapeutic applications in CVDs, emphasizing the need for a clear regulatory framework to facilitate clinical translation. The establishment of consistent regulatory guidelines will be a critical step in ensuring the safety and efficacy of mRNA-based cardiovascular therapies, potentially facilitating their widespread adoption. The future direction of research in mRNA formulation, personalized medicine approaches, and the refinement of regulatory policies will determine the landscape of next-generation cardiovascular therapeutics.

Messenger RNA (mRNA) based therapeutics in transforming cardiovascular care: Progress, regulatory challenges and future perspectives / M. Khan, U.M. Musazzi, S. Manellari, N. Mouawad, G. Damiano, R. Rinaldi, A. Raucci, S. Costantino, F. Paneni, P. Minghetti, G. Pompilio, M.C. Vinci. - In: PHARMACOLOGICAL RESEARCH. - ISSN 1043-6618. - 218:(2025 Aug), pp. 107847.1-107847.15. [10.1016/j.phrs.2025.107847]

Messenger RNA (mRNA) based therapeutics in transforming cardiovascular care: Progress, regulatory challenges and future perspectives

M. Khan
Primo
;
U.M. Musazzi
Secondo
;
S. Manellari;G. Damiano;P. Minghetti;G. Pompilio
Penultimo
;
2025

Abstract

Messenger RNA (mRNA) is emerging as a new tool for therapeutic intervention, providing a transient, programmable platform for the treatment of complex diseases. Unlike DNA-based therapies, mRNA does not integrate into the genome and is considered more safe. The success of mRNA vaccines, combined with advances in mRNA technology and unmet needs in cardiovascular disease (CVD), has led to increased interest in expanding the use of this technology beyond infectious diseases. However, there are several challenges to overcome, including mRNA stability, delivery efficiency, immunogenicity, and regulatory standardization. This review examines advances in mRNA design, delivery platforms, and therapeutic applications in CVDs, emphasizing the need for a clear regulatory framework to facilitate clinical translation. The establishment of consistent regulatory guidelines will be a critical step in ensuring the safety and efficacy of mRNA-based cardiovascular therapies, potentially facilitating their widespread adoption. The future direction of research in mRNA formulation, personalized medicine approaches, and the refinement of regulatory policies will determine the landscape of next-generation cardiovascular therapeutics.
Cardiovascular diseases (CVD); Regenerative medicine; Regulatory affairs; mRNA delivery systems; mRNA-based therapeutics;
Settore CHEM-08/A - Tecnologia, socioeconomia e normativa dei medicinali e dei prodotti per il benessere e per la salute
Settore MEDS-13/C - Chirurgia cardiaca
   National Center for Gene Therapy and Drugs based on RNA Technology (CN3 RNA)
   CN3 RNA
   MINISTERO DELL'UNIVERSITA' E DELLA RICERCA
   CN00000041
ago-2025
1-lug-2025
Article (author)
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/1174777
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