Artificial Intelligence and the Transformation of Cell and Gene Therapy Development

Cell and Gene Therapy (CGT) represents a paradigm shift in medicine, offering curative potential for previously intractable diseases. However, the complexity, high cost, and manufacturing challenges inherent in developing, producing, and administering these therapies hinder their widespread accessibility. This review examines the critical and increasingly synergistic role of Artificial Intelligence (AI) and Machine Learning (ML) in overcoming these barriers across the entire CGT lifecycle, from discovery and construct design to smart manufacturing, clinical translation, and regulatory applications. We analyze how AI-driven approaches fundamentally differ from conventional methods, facilitating rapid construct optimization, generating highly predictive translational models, enabling the vision of autonomous, digital-twin-driven manufacturing, and establishing new paradigms for pharmacovigilance and regulatory oversight. The integration of AI is not merely an incremental improvement but a foundational transformation, positioning CGT to move from niche, bespoke treatments to scalable, accessible, and highly personalized medical modalities. We conclude by discussing current gaps, particularly data scarcity and regulatory uncertainty, and outlining a roadmap to realize the full potential of AI-enabled CGT.

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Publication Details

Journal
Pharmaceutics
Published
2026-03-13
DOI
https://doi.org/10.3390/pharmaceutics18030356
Primary Topic
CAR-T cell therapy research
Type
article
Field-Weighted Citation Impact
0.00
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article

Artificial Intelligence and the Transformation of Cell and Gene Therapy Development

Rominder Singh, Gaël Debauve, Jeewon Joung, Maya A. Singh et al.
Pharmaceutics
CAR-T cell therapy research
article

Artificial Intelligence and the Transformation of Cell and Gene Therapy Development

Rominder Singh, Gaël Debauve, Jeewon Joung, Maya A. Singh, Jared R. Auclair
article en

Abstract

Cell and Gene Therapy (CGT) represents a paradigm shift in medicine, offering curative potential for previously intractable diseases. However, the complexity, high cost, and manufacturing challenges inherent in developing, producing, and administering these therapies hinder their widespread accessibility. This review examines the critical and increasingly synergistic role of Artificial Intelligence (AI) and Machine Learning (ML) in overcoming these barriers across the entire CGT lifecycle, from discovery and construct design to smart manufacturing, clinical translation, and regulatory applications. We analyze how AI-driven approaches fundamentally differ from conventional methods, facilitating rapid construct optimization, generating highly predictive translational models, enabling the vision of autonomous, digital-twin-driven manufacturing, and establishing new paradigms for pharmacovigilance and regulatory oversight. The integration of AI is not merely an incremental improvement but a foundational transformation, positioning CGT to move from niche, bespoke treatments to scalable, accessible, and highly personalized medical modalities. We conclude by discussing current gaps, particularly data scarcity and regulatory uncertainty, and outlining a roadmap to realize the full potential of AI-enabled CGT.

PharmaceuticsVol. 18(3)
Ministry of Food and Drug Safety (KR), Northeastern University (US), University of Washington (US), UCB Pharma (Belgium) (BE)
Industry, innovation and infrastructure
Openalex Percentile: Top 23%
CAR-T cell therapy research
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