Volume 29, Issue 162, August 2025

mRNA Therapeutics Beyond Vaccines: Emerging Applications in Rare and Chronic Diseases

Marta Górska^1♦, Maciej Janowski¹, Jakub Górski², Paweł Czyż³, Antonina Strzałkowska¹

¹Central Teaching Hospital of Medical University of Lodz, Pomorska 251, 92- 213, Lodz, Poland
²Medical University of Lodz, Pomorska 251, 92-213, Lodz, Poland
³Szpital Miejski im. Św. Wincentego a Paulo w Gdyni, ul.Wójta Radtkego 18 Gdynia, Poland

^♦Corresponding author
Marta Górska, Central Teaching Hospital of Medical University of Lodz, Pomorska 251, 92-213, Lodz, Poland

ABSTRACT

Messenger RNA (mRNA) isn’t just about COVID-19 vaccines anymore, far from it. In just the past few years (especially starting around 2015), mRNA technology has begun to change the way we think about treating disease, not just some diseases, but a wide range: rare ones, chronic ones, even a few that used to be considered untreatable. This review takes a closer look at where mRNA might be headed next, not just beyond COVID-19, but beyond infectious diseases altogether. We're talking about cancer (with a big focus on personalized, tailor-made immunotherapies), rare inherited metabolic disorders (conditions such as cystic fibrosis and propionic acidemia have always limited treatment options), and autoimmune diseases too (like multiple sclerosis or type 1 diabetes, which affect millions but still lack accurate long-term solutions). In other words, mRNA isn't just a pandemic story - it’s becoming a much bigger story (a much more exciting one) across medicine. We examine not only what these therapies entail, but also how they are delivered (with lipid nanoparticles playing a significant role), how they are being tested (including ongoing clinical trials), and their safety and scalability (a critical consideration). The spotlight is on research and clinical trials spanning january 2015 to May 2025 - all aimed at giving us a clearer sense of what truly works, what’s still experimental, and what’s just about to break through. mRNA, which not so long ago was thought of mainly as a vaccine tool, is now (quite rapidly) transforming into something bigger: a broad therapeutic platform, and maybe - even more exciting - a real gamechanger for the future of medicine.

Keywords: mRNA therapeutics, lipid nanoparticles, CRISPR-Cas9 delivery, VEGF mRNA

Medical Science, 2025, 29, e150ms3698

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DOI: https://doi.org/10.54905/disssi.v29i162.e150ms3698

Published: 30 August 2025