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RNA-based drugs: current, imminent and possible therapeutic applications
Sebastiano A Torrisi1, Federica Geraci2, Lidia Diolosà3
1Department of Biomedical and Biotechnological Sciences, University of Catania, Catania, Italy; Oasi Research Institute-IRCCS, Unit of Translational Neuropharmacology and Translational Neurosciences, Troina, Italy.
Pharmacology & Therapeutics
|November 20, 2025
Summary
Messenger RNA (mRNA) vaccines have saved millions of lives, paving the way for novel RNA-based therapeutics. These versatile drugs offer cost-effective treatments for previously undruggable targets across diverse diseases.
Area of Science:
- Pharmacology
- Biotechnology
- Drug Discovery
Background:
- Messenger RNA (mRNA) vaccines have demonstrated significant success in combating the COVID-19 pandemic, saving millions of lives.
- The development of RNA-based therapeutics represents a paradigm shift in medicine, offering promising applications beyond infectious diseases.
- RNA-based drugs are characterized by ease of design, manufacturing, and potential cost-effectiveness.
Purpose of the Study:
- To provide a comprehensive pharmacological analysis of the current RNA therapeutics landscape.
- To review approved RNA-based drugs and key investigational candidates.
- To highlight clinical progress, pharmacological mechanisms, and challenges in RNA drug development.
Main Methods:
- Literature review of approved and investigational RNA-based drugs.
- Analysis of pharmacological mechanisms, drug delivery challenges, tolerability, and clinical outcomes.
- Exploration of RNA-based drug repurposing strategies.
Main Results:
- RNA-based drugs can target previously undruggable pharmacological targets, enabling personalized treatments for difficult-to-treat diseases.
- These therapeutics show versatility across various diseases, including cancer, neurodegenerative, cardiovascular, metabolic, rare genetic, and infectious diseases.
- Drug repurposing leveraging shared pathophysiological mechanisms can accelerate clinical impact.
Conclusions:
- RNA-based therapeutics represent a significant advancement in medicine with broad therapeutic potential.
- Addressing challenges in drug delivery and optimizing tolerability are crucial for clinical success.
- The versatility and potential for repurposing of RNA-based drugs offer a promising future for personalized medicine.
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