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Updated: Jul 19, 2025

MISSION esiRNA for RNAi Screening in Mammalian Cells
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Published on: May 12, 2010

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25 years of maturation: A systematic review of RNAi in the clinic

Ida Juhl Corydon1, Bjørn Kristensen Fabian-Jessing1,2, Thomas Stax Jakobsen1,2

  • 1Department of Biomedicine, Aarhus University, Høegh-Guldbergs Gade 10, 8000 Aarhus C, Denmark.

Molecular Therapy. Nucleic Acids
|August 16, 2023
PubMed

Insights

RNA interference (RNAi) therapeutics, enabling sequence-specific gene knockdown, have seen significant advancement. The landscape shows increasing clinical applications and development towards targeting extra-hepatic tissues.

Area of Science:

  • Biotechnology
  • Genetics
  • Pharmacology

Background:

  • RNA interference (RNAi) therapeutics offer precise gene knockdown by targeting RNA molecules via complementary base-pairing.
  • 2023 marks the 25th anniversary of RNAi discovery, highlighting its therapeutic potential.
  • The field has evolved significantly, with advancements in delivery systems and clinical applications.

Purpose of the Study:

  • To systematically review published and ongoing clinical trials of RNAi-based therapeutics.
  • To analyze the current landscape of clinically applied RNAi, focusing on therapeutic maturation, delivery platforms, administration routes, and targets.
  • To understand the trajectory and future potential of RNAi therapeutics.

Main Methods:

  • A systematic review of Web of Science, PubMed, and Embase databases was conducted for clinical trials from January 1, 1998, to December 30, 2022.
  • Data extraction followed a predefined protocol for 90 published trials (from 81 articles).
  • Ongoing clinical trials were identified from ClinicalTrials.gov, adding 48 trials to the analysis.

Main Results:

  • A total of 90 published and 48 ongoing clinical trials utilizing RNAi were included.
  • Contemporary trials predominantly employ N-acetylgalactosamine delivery with subcutaneous administration or lipid nanoparticle delivery with intravenous administration.
  • Five RNAi therapeutics targeting the liver for severe diseases have been approved.

Conclusions:

  • RNAi therapeutics have demonstrated substantial growth over the last decade.
  • The increasing number of ongoing trials suggests a promising future for RNAi-based medicines.
  • Future developments are expected to expand RNAi therapeutic applications beyond hepatic tissues.
Keywords:
LNPMT: Oligonucleotides: Therapies and ApplicationsN-acetylgalactosamineRNA interferenceRNAiclinical trialsmiR-shRNAshRNAsiRNAsystematic review

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