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npj Digital Medicine· 2025Q1· Review

AI-driven virtual cell models in preclinical research: technical pathways, validation mechanisms, and clinical translation potential

Chunyu Ma, Han Zhang, Yiwei Rao, Xinyu Jiang et al.

Short summary

AI-driven virtual cell models, integrating omics data with deep learning, can predict drug responses and disease progression with high precision, potentially reducing animal testing and optimizing therapies.

AI-generated from the title and abstract; the full text is not read.

Key points

  • Virtual cell models integrate omics data (transcriptomics, proteomics) with deep learning (generative models, GNNs).
  • They enable high-precision predictions of drug responses, gene perturbations, and disease progression.
  • A closed-loop workflow uses computational evaluation and experimental verification (CRISPR, organoids).
  • Applications include personalized drug screening and disease modeling, aiming to reduce animal testing.
  • Challenges remain in regulatory acceptance, data privacy, and model interpretability.

AI-generated from the title and abstract; the full text is not read.

Abstract

AI-driven virtual cell models show the potential to transform the paradigm of life sciences research by integrating multimodal omics data (e.g., single-cell transcriptomics and proteomics) with advanced algorithms such as deep generative models and graph neural networks to enable high-precision predictions of drug responses, gene perturbations, and disease progression. These models enable high-precision predictions of drug responses, gene perturbations, and disease progression. This review outlines the technical pathways and validation mechanisms of virtual cells, emphasizing a closed-loop workflow from computational evaluation to experimental verification using CRISPR assays and organoid platforms. The applications of virtual cells in personalized drug screening and disease modeling are highlighted, showcasing their potential to reduce animal testing and optimize therapy. However, challenges in regulatory acceptance, data privacy, and model interpretability remain. Global policy and standardization trends are driving clinical translation, and future advancements will involve cross-disciplinary integration and greater standardization to enhance the impact of virtual cells in precision medicine and drug discovery.

The authors' abstract, as published at the source. npj Digital Medicine, 2025 · DOI ↗

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Drug DiscoveryPharmacology, Toxicology and Pharmaceutics