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- Title
RNA interference in the era of nucleic acid therapeutics.
- Authors
Jadhav, Vasant; Vaishnaw, Akshay; Fitzgerald, Kevin; Maier, Martin A.
- Abstract
Two decades of research on RNA interference (RNAi) have transformed a breakthrough discovery in biology into a robust platform for a new class of medicines that modulate mRNA expression. Here we provide an overview of the trajectory of small-interfering RNA (siRNA) drug development, including the first approval in 2018 of a liver-targeted siRNA interference (RNAi) therapeutic in lipid nanoparticles and subsequent approvals of five more RNAi drugs, which used metabolically stable siRNAs combined with N-acetylgalactosamine ligands for conjugate-based liver delivery. We also consider the remaining challenges in the field, such as delivery to muscle, brain and other extrahepatic organs. Today's RNAi therapeutics exhibit high specificity, potency and durability, and are transitioning from applications in rare diseases to widespread, chronic conditions. With six approved drugs, siRNA is now an established therapeutic modality poised for expansion.
- Publication
Nature Biotechnology, 2024, Vol 42, Issue 3, p394
- ISSN
1087-0156
- Publication type
Article
- DOI
10.1038/s41587-023-02105-y