Machine learning for RNA-targeting drug design

Fuente: arXiv
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Autores principales: Karroucha, Wissam, Oliver, Carlos, Stoven, Veronique, Mallet, Vincent
Formato: Preprint
Publicado: 2025
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author Karroucha, Wissam
Oliver, Carlos
Stoven, Veronique
Mallet, Vincent
author_facet Karroucha, Wissam
Oliver, Carlos
Stoven, Veronique
Mallet, Vincent
contents Targeting RNA with small molecules offers significant therapeutic potential. Machine learning could substantially accelerate preclinical drug discovery, from hit identification to lead optimization. Yet a fundamental limitation emerges: drug design machine learning models, tailored for proteins, are not readily applicable to RNAs because of fundamental differences between RNAs and proteins in both structural characteristics and interactions with small molecules. RNA-specific approaches have consequently emerged, primarily focusing on binding site identification and virtual screening. In this review, we comprehensively compare machine learning tools for RNA-targeting drug design according to the tasks they address, their methodology and their relevance in RNA-specific contexts. As open challenges will catalyze new method development, we emphasize the need for standardized, drug design-specific evaluation approaches. We provide clear guidelines to establish these standards along with a benchmark assessing the ability of current machine learning models to predict specific drug-RNA interactions.
format Preprint
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institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Machine learning for RNA-targeting drug design
Karroucha, Wissam
Oliver, Carlos
Stoven, Veronique
Mallet, Vincent
Biomolecules
Targeting RNA with small molecules offers significant therapeutic potential. Machine learning could substantially accelerate preclinical drug discovery, from hit identification to lead optimization. Yet a fundamental limitation emerges: drug design machine learning models, tailored for proteins, are not readily applicable to RNAs because of fundamental differences between RNAs and proteins in both structural characteristics and interactions with small molecules. RNA-specific approaches have consequently emerged, primarily focusing on binding site identification and virtual screening. In this review, we comprehensively compare machine learning tools for RNA-targeting drug design according to the tasks they address, their methodology and their relevance in RNA-specific contexts. As open challenges will catalyze new method development, we emphasize the need for standardized, drug design-specific evaluation approaches. We provide clear guidelines to establish these standards along with a benchmark assessing the ability of current machine learning models to predict specific drug-RNA interactions.
title Machine learning for RNA-targeting drug design
topic Biomolecules
url https://arxiv.org/abs/2512.15645