Advances in RNA Therapeutics: Classes, Innovations and Clinical Applications

RNA therapeutics represent a transformative class of treatments capable of correcting genetic errors, modulating gene expression, and enabling targeted intervention across a wide range of diseases. These platforms—including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), messenger RNAs (mRNAs), and microRNAs (miRNAs)—have progressed from proof-of-concept to clinical reality, with multiple FDA-approved drugs for conditions such as transthyretin amyloidosis, spinal muscular atrophy, and COVID-19. This review highlights the mechanisms, clinical milestones, and delivery innovations that have propelled RNA-based therapies forward. This article examines emerging carriers—including lipid nanoparticles, aptamer conjugates, peptides, and exosomes—that address critical challenges such as RNA instability, off-target effects, and organ-specific delivery. Advances in RNA editing, AI-guided sequence design, and self-amplifying RNA platforms are poised to further expand RNA’s therapeutic scope. The review also discusses applications across cardiovascular, neurological, oncological, and infectious diseases, as well as opportunities in regenerative and personalized medicine. While barriers in delivery, scalability, and regulatory adaptation remain, ongoing innovations are rapidly closing the gap between potential and practice. RNA therapeutics are no longer experimental—they are reshaping the future of medicine.