The Science of RNA Interference
RNAi is a natural biological process that regulates gene expression by cleaving target mRNA and reducing production of the related protein.1-4
RNAi therapeutics harness the natural RNAi mechanism for regulating gene expression to offer targeted knockdown of specific proteins.1-5
of RNAi therapeutics for regulating gene expression
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Summary of RNAi
An overview of RNAi discovery, how RNAi works, and the potential of RNAi therapeutics
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RNAi Mechanism of Action
Watch the full mechanism of action video for a comprehensive overview of the natural RNAi mechanism, the science of RNAi therapeutics, delivery platforms, and RNAi therapeutics in action
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Science of RNAi
Learn more about how Alnylam is turning Nobel Prize-winning science into action
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RNAi Therapeutics
Learn more about how Alnylam plans to further innovate RNAi therapeutics
WATCH NOWFeatures of RNAi Therapeutics and Delivery Systems
Highly selective for specific mRNA, with limited off-target effects6-9
Catalytic mechanism enables repeated cleavage of target mRNA1,10
Enhanced stability and duration of action1,7,9,11
Infrequent dosing intervals7,11-13
Delivery Systems
RNAi therapeutics have the potential to target any tissue in the body to reduce the expression of any target protein.1,5,6 Currently approved RNAi therapeutics target the liver (accurate as of February 2026).5,14 Research is ongoing to expand delivery to diverse tissues.6
The History of RNAi and Alnylam
Since its initial observation in 1990, RNAi has progressed from a naturally occurring biological mechanism to an approach now applied in clinical practice.5,21
References: 1. Friedrich M, Aigner A. BioDrugs. 2022;36:549-571; 2. Cuccato G, et al. BMC Syst Biol. 2011;5:19; 3. Martinez NJ, Gregory RI. RNA. 2013;19:605-612; 4. Varley AJ, Desaulniers JP. RSC Adv. 2021;11:2415-2426; 5. Traber GM, Yu AM. Mol Pharmacol. 2024;106:13-20; 6. Hu B, et al. Signal Transduct Target Ther. 2020;5:101; 7. Ranasinghe P, et al. Br J Pharmacol. 2023;180:2697-2720; 8. Lam JKW, et al. Mol Ther Nucleic Acids. 2015;4:e252; 9. Zhang Y, et al. Int J Mol Sci. 2022;23:2408; 10. Li Z, Rana TM. Acc Chem Res. 2012;45:1122-1131; 11. Brown CR, et al. Nucleic Acids Res. 2020;48:11827-11844; 12. Gareri C, et al. J Clin Med. 2022;11:3884; 13. Tang Q, Khvorova A. Nat Rev Drug Discov. 2024;23:341-364; 14. Jadhav V, et al. Nat Biotechnol. 2024;42:394-405; 15. Tam YYC, et al. Pharmaceutics. 2013;5:498-507; 16. Kaczmarek JC, et al. Genome Med. 2017;9:60; 17. Brown KM, et al. Nat Biotechnol. 2022;40:1500-1508; 18. An G. J Clin Pharmacol. 2024;64:45-57; 19. Brown K. Presented at: International Stroke Conference; February 5-7, 2025; Los Angeles, CA, USA; 20. Sloan K. Presented at: Annual Meeting of the Huntington Study Group; November 7-9, 2024; Cincinnati, OH, USA; 21. Napoli C, et al. Plant Cell. 1990;2:279-289; 22. Fire A, et al. Nature. 1998;391:806-811; 23. Soutschek J, et al. Nature. 2004;432:173-178; 24. The Nobel Prize. Nobel Prize in Physiology or Medicine 2006—Advanced information. Accessed February 2026. https://www.nobelprize.org/prizes/medicine/2006/advanced-information/; 25. ONPATTRO® (patisiran) Prescribing Information. Alnylam; 2023; 26. GIVLAARI® (givosiran) Prescribing Information. Alnylam; 2024; 27. OXLUMO® (lumasiran) Prescribing Information. Alnylam; 2025; 28. LEQVIO® (inclisiran) Prescribing Information. Alnylam; 2025; 29. AMVUTTRA® (vutrisiran) Prescribing Information; 30. QFITLIA® (fitusiran) Prescribing Information.
Abbreviations: ApoE, apolipoprotein E; ASGPR, asialoglycoprotein receptor; CNS, central nervous system; FDA, Food and Drug Administration; GalNAc, N-acetylgalactosamine; IT, intrathecal; IV, intravenous; LDL, low-density lipoprotein; LNP, lipid nanoparticle; mRNA, messenger ribonucleic acid; RISC, ribonucleic acid-induced silencing complex; RNA, ribonucleic acid; RNAi, ribonucleic acid interference; SC, subcutaneous; siRNA, small interfering ribonucleic acid.
MED-US-RNAi-2600014
