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Abstract
Three mRNA products are projected to be approved by 2024 for infectious diseases and rare metabolic conditions.
- mRNA therapeutics include diverse molecular architectures like modified nucleosides and advanced platforms such as self-amplifying RNA and circular RNA.
- Lipid nanoparticle delivery faces challenges such as endosomal escape limitations and the need for organ-targeted delivery systems.
- Clinical applications of mRNA technologies span vaccines, personalized cancer treatments, protein replacement therapies, and CRISPR genome editing.
- Translational challenges include issues with stability, targeting specific tissues, immunogenicity, and scaling up manufacturing processes.
- Regulatory science and equitable access for low- and middle-income countries are critical areas needing attention, with specific recommendations for capacity-building.
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