From rare to prevalent: dawn of the RNA therapeutics era

Jun Zhou , Erica Shu

Targetome ›› 2025, Vol. 1 ›› Issue (1) : e004

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Targetome ›› 2025, Vol. 1 ›› Issue (1) :e004 DOI: 10.48130/targetome-0025-0005
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From rare to prevalent: dawn of the RNA therapeutics era
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RNA therapeutics / Rare diseases / Chronic diseases / Multidisciplinary

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Jun Zhou, Erica Shu. From rare to prevalent: dawn of the RNA therapeutics era. Targetome, 2025, 1(1): e004 DOI:10.48130/targetome-0025-0005

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Author contributions

The draft of the manuscript was prepared by Zhou J and Shu E. Both authors reviewed the results and approved the final version of the manuscript.

Data availability

Not applicable.

Acknowledgments

We gratefully acknowledge Ms. Rui Zhang and Ms. Xianrong Lin at China Pharmaceutical University for their valuable assistance in research information collection. This work was supported by grants from the Open Project of State Key Laboratory of Natural Medicines (Grant No. SKLNMZZ2024JS33), and the Fundamental Research Funds for the Central Universities (2632025TD05).

Conflict of interest

The authors declare that they have no conflict of interest.

References

[1]

Henley MJ, Koehler AN. 2021. Advances in targeting 'undruggable' transcription factors with small molecules. Nature Reviews Drug Disco-very 20:669-688

[2]

Pathmanathan S, Grozavu I, Lyakisheva A, Stagljar I. 2022. Drugging the undruggable proteins in cancer: a systems biology approach. Current Opinion in Chemical Biology 66:102079

[3]

Dang CV, Reddy EP, Shokat KM, Soucek L. 2017. Drugging the 'undrug-gable' cancer targets. Nature Reviews Cancer 17:502-508

[4]

Costales MG, Matsumoto Y, Velagapudi SP, Disney MD. 2018. Small molecule targeted recruitment of a nuclease to RNA. Journal of the American Chemical Society 140:6741-6744

[5]

Ghidini A, Cléry A, Halloy F, Allain FHT, Hall J. 2021. RNA-PROTACs: degraders of RNA-binding proteins. Angewandte Chemie 60:3163-3169

[6]

Dey SK, Jaffrey SR. 2019. RIBOTACs: small molecules target RNA for degradation. Cell Chemical Biology 26:1047-1049

[7]

Zhang P, Liu X, Abegg D, Tanaka T, Tong Y, et al. 2021. Reprogram-ming of protein-targeted small-molecule medicines to RNA by ribonu-clease recruitment. Journal of the American Chemical Society 143:13044-13055

[8]

Costales MG, Aikawa H, Li Y, Childs-Disney JL, Abegg D, et al. 2020. Small-molecule targeted recruitment of a nuclease to cleave an onco-genic RNA in a mouse model of metastatic cancer. Proceedings of the National Academy of Sciences of the United States of America 117:2406-2411

[9]

Opalinska JB, Gewirtz AM. 2002. Nucleic-acid therapeutics: basic princi-ples and recent applications. Nature Reviews Drug Discovery 1:503-514

[10]

Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, et al. 2018. Patisiran, an RNAi therapeutic, for hereditary transthyretin amy-loidosis. The New England Journal of Medicine 379:11-21

[11]

Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, et al. 2018. Nusinersen versus sham control in later-onset spinal muscular atrophy. The New England Journal of Medicine 378:625-635

[12]

Frampton JE. 2023. Inclisiran: a review in hypercholesterolemia. Ameri-can Journal of Cardiovascular Drugs 23:219-230

[13]

Mak LY, Gane E, Schwabe C, Yoon KT, Heo J, et al. 2023. A phase I/II study of ARO-HSD, an RNA interference therapeutic, for the treatment of non-alcoholic steatohepatitis. Journal of Hepatology 78:684-692

[14]

Gane E, Lim YS, Kim JB, Jadhav V, Shen L, et al. 2023. Evaluation of RNAi therapeutics VIR-2218 and ALN-HBV for chronic hepatitis B: results from randomized clinical trials. Journal of Hepatology 79:924-932

[15]

Magoola M, Niazi SK. 2025. Current progress and future perspectives of RNA-based cancer vaccines: a 2025 update. Cancers 17(11):1882

[16]

Weber JS, Carlino MS, Khattak A, Meniawy T, Ansstas G, et al. 2024. Individualised neoantigen therapy mRNA-4157 (V940) plus pembro-lizumab versus pembrolizumab monotherapy in resected melanoma (KEYNOTE-942): a randomised, phase 2b study. The Lancet 403:632-644

[17]

Rojas LA, Sethna Z, Soares KC, Olcese C, Pang N, et al. 2023. Personal-ized RNA neoantigen vaccines stimulate T cells in pancreatic cancer. Nature 618:144-150

[18]

Paunovska K, Loughrey D, Dahlman JE. 2022. Drug delivery systems for RNA therapeutics. Nature Reviews Genetics 23:265-280

[19]

Naeem S, Zhang J, Zhang Y, Wang Y. 2025. Nucleic acid therapeutics: past, present, and future. Molecular Therapy Nucleic Acids 36:102440

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