MILESTONES

A new dawn for RNAi drugs

Credit: GiroScience / Alamy Stock Photo

The 10th of August, 2018 marked a new era for the field of RNA therapeutics, with the first approval of an RNA interference (RNAi)-based drug by the United States Food and Drug Administration. The drug — patisiran (Onpattro) — is approved for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis (hATTR). This rare and devastating neurodegenerative disease is caused by deposition of amyloid fibrils formed by misfolded transthyretin protein. A double-stranded small interfering RNA composed of two modified 21‑mer oligonucleotides and encapsulated in a lipid nanoparticle formulated for hepatocyte uptake, patisiran silences transthyretin mRNAs in the liver to reduce serum levels of the protein. The approval of patisiran brings new hope to patients with hATTR who previously had no effective treatment options.

Further reading

Garber, K. Alnylam launches era of RNAi drugs. Nat. Biotechnol. 36, 777–778 (2018).

Setten, R. L. et al. The current state and future directions of RNAi-based therapeutics. Nat. Rev. Drug Discov. 18, 421–446 (2019)

doi: 10.1038/d42859-019-00091-3

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