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RNA Therapeutics: Delivery Problems and Solutions-A Review
Natalia Pozdniakova1, Evgenii Generalov2, Alexei Shevelev3
1Blokhin National Medical Research Center of Oncology, Ministry of Health of the Russian Federation, 115522 Moscow, Russia.
Pharmaceutics
|October 29, 2025
Summary
RNA therapeutics show great promise but face delivery challenges. Novel delivery methods and RNA modifications are key to expanding their use for widespread diseases like cancer and autoimmune disorders.
Area of Science:
- Biotechnology
- Molecular Medicine
- Drug Delivery Systems
Background:
- RNA-based therapeutics offer significant potential for treating various diseases.
- Current RNA delivery technologies face challenges in tissue targeting, selectivity, and side effects, hindering clinical progress.
Purpose of the Study:
- To critically assess the current landscape of RNA-derived medicines and their therapeutic applications.
- To examine advancements in RNA modification, delivery systems, and molecular mechanisms for treating prevalent diseases.
Main Methods:
- Review of existing literature on mRNA vaccines and RNA-based therapeutics.
- Analysis of clinical trial data for modified short RNA drugs (siRNAs, miRNA mimetics/inhibitors).
- Evaluation of evolving delivery methods like lipid nanoparticles, viral vectors, and exosomes.
Main Results:
- Approved RNA therapies predominantly target rare diseases due to delivery limitations.
- RNA-mediated macrophage reprogramming shows promise for enhancing delivery and efficacy.
- Novel approaches in RNA modification and targeted delivery are crucial for broader applications.
Conclusions:
- Advancements in RNA modification, targeted delivery, and understanding molecular mechanisms are essential for expanding RNA therapeutics to prevalent diseases.
- Overcoming delivery hurdles is critical to unlocking the full therapeutic potential of RNA-based medicines.
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