Development of RNA-targeting small-molecule therapeutics

Walter J. Zahurancik; Vaishnavi Sidharthan; Roopa Biswas; Venkat Gopalan

doi:10.1016/j.tips.2026.04.011

Development of RNA-targeting small-molecule therapeutics

Walter J. Zahurancik^*
, Vaishnavi Sidharthan
, Roopa Biswas
, Venkat Gopalan

^*Corresponding author for this work

Anatomy, Physiology, and Genetics

Research output: Contribution to journal › Short survey › peer-review

Abstract

Development of robust RNA-targeting therapeutics to treat diseases requires an understanding of the structural plasticity of ligand-binding pockets in RNAs and the complexity of RNA interactions in vivo. Refocusing efforts toward new RNA-targeted compound libraries and large-scale RNA structural studies will help design successful screens and accelerate RNA-centered drug discovery.

Original language	English
Pages (from-to)	598-602
Number of pages	5
Journal	Trends in Pharmacological Sciences
Volume	47
Issue number	6
DOIs	https://doi.org/10.1016/j.tips.2026.04.011
State	Published - Jun 2026

Keywords

drug discovery
RNA

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10.1016/j.tips.2026.04.011

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title = "Development of RNA-targeting small-molecule therapeutics",

abstract = "Development of robust RNA-targeting therapeutics to treat diseases requires an understanding of the structural plasticity of ligand-binding pockets in RNAs and the complexity of RNA interactions in vivo. Refocusing efforts toward new RNA-targeted compound libraries and large-scale RNA structural studies will help design successful screens and accelerate RNA-centered drug discovery.",

keywords = "drug discovery, RNA",

author = "Zahurancik, \{Walter J.\} and Vaishnavi Sidharthan and Roopa Biswas and Venkat Gopalan",

note = "Publisher Copyright: {\textcopyright} 2026 Elsevier Ltd",

year = "2026",

month = jun,

doi = "10.1016/j.tips.2026.04.011",

language = "English",

volume = "47",

pages = "598--602",

journal = "Trends in Pharmacological Sciences",

issn = "0165-6147",

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TY - JOUR

T1 - Development of RNA-targeting small-molecule therapeutics

AU - Zahurancik, Walter J.

AU - Sidharthan, Vaishnavi

AU - Biswas, Roopa

AU - Gopalan, Venkat

PY - 2026/6

Y1 - 2026/6

N2 - Development of robust RNA-targeting therapeutics to treat diseases requires an understanding of the structural plasticity of ligand-binding pockets in RNAs and the complexity of RNA interactions in vivo. Refocusing efforts toward new RNA-targeted compound libraries and large-scale RNA structural studies will help design successful screens and accelerate RNA-centered drug discovery.

AB - Development of robust RNA-targeting therapeutics to treat diseases requires an understanding of the structural plasticity of ligand-binding pockets in RNAs and the complexity of RNA interactions in vivo. Refocusing efforts toward new RNA-targeted compound libraries and large-scale RNA structural studies will help design successful screens and accelerate RNA-centered drug discovery.

KW - drug discovery

KW - RNA

UR - https://www.scopus.com/pages/publications/105040116903

U2 - 10.1016/j.tips.2026.04.011

DO - 10.1016/j.tips.2026.04.011

M3 - Short survey

C2 - 42168044

AN - SCOPUS:105040116903

SN - 0165-6147

VL - 47

SP - 598

EP - 602

JO - Trends in Pharmacological Sciences

JF - Trends in Pharmacological Sciences

IS - 6

ER -

Development of RNA-targeting small-molecule therapeutics

Abstract

Keywords

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