Approaches to in vivo CAR-T therapy technology and considerations of quality control and nonclinical research
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PUBLIC SUMMARY
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This review introduced the mechanisms, clinical advances and optimization strategies of in vivo CAR-T therapy.
This review focused on considerations for developing quality control strategies for in vivo CAR-T therapy.
This review focused on discussing key considerations for its nonclinical research.
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ABSTRACT
Chimeric antigen receptor T-cell (CAR-T) therapy has achieved remarkable success in the treatment of hematological malignancies. However, conventional ex vivo CAR-T therapies are constrained by complex manufacturing processes, long production cycles, and limited clinical applicability. To address these limitations and to simplify the treatment process and enhance accessibility, strategies for in situ delivery of CAR-encoding genes to endogenous T cells have been developed. This review summarizes the design of carriers and payloads for in vivo CAR-T therapy, provides an overview of recent advances in the field, and examines its persistent challenges and potential solutions. The article focuses on discussing key considerations related to quality control and nonclinical research for in vivo CAR-T products based on their characteristics and relevant guidelines for gene therapy and conventional CAR-T cell products, with the aim of providing insights for the translational application of this technology. -
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REFERENCES
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Qin X., Hou S., Sun Z., et al. (2026). Approaches to in vivo CAR-T therapy technology and considerations of quality control and nonclinical research. The Innovation Drug Discovery 1:100012. https://doi.org/10.59717/j.xinn-drugdisc.2026.100012
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Qin X., Hou S., Sun Z., et al. (2026). Approaches to in vivo CAR-T therapy technology and considerations of quality control and nonclinical research. The Innovation Drug Discovery 1:100012. https://doi.org/10.59717/j.xinn-drugdisc.2026.100012
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Figure 1.
In vivo CAR-T timeline
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Figure 2.
Development of chimeric antigen receptor (CAR) structure
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Figure 3.
Clinical trial indications and corresponding CAR targets of in vivo CAR-T therapy
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Figure 4.
Comparison of manufacturing and quality control processes of LNP-based and lentiviral-based in vivo CAR-T therapy
