Innovations in RNA therapeutics: a review of recent advances and emerging technologies

Abstract

The field of biomedical science has witnessed another milestone with the advent of RNA-based therapeutics. This review explores three major RNA molecules, namely: messenger RNA (mRNA), RNA interference technology (RNAi), and Antisense Oligonucleotide (ASO), and analyses U.S. Food and Drug Administration drugs from 14 RNA-based pharmaceutical companies in terms of targeted genes, diseases and types, clinical trials and status, the mode of delivery, and the year of production. Many of such drugs are clinically approved or pending approval by the U.S. Food and Drug Administration (FDA) alongside other leading drugs agencies. Regarding diseases, this article emphasizes cancer therapy, genetic diseases, viral infections, and two categories of drug delivery systems include viral vectors and nanoparticles. Despite the tremendous progress made, key issues associated with these delivery systems are stability, off-target activities of RNA payloads, efficiency in cellular uptake, and the innovative need for engineering techniques for modifications. This review emphasizes the transformative potential of RNA therapeutics and the role of innovative technologies in addressing clinical needs, paving the way for a new era in precision medicine.

Keywords: RNA interference; RNA-based therapeutics; antisense oligonucleotides; mRNA; siRNA.