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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

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Related Experiment Video

Updated: Dec 23, 2025

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Published on: August 18, 2023

Chemically Programmable and Switchable CAR-T Therapy.

Junpeng Qi¹, Kohei Tsuji^2,3, David Hymel²

¹Department of Immunology and Microbiology, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL, 33458, USA.

Angewandte Chemie (International Ed. in English)

|April 25, 2020

Summary

This summary is machine-generated.

This study introduces a novel method to enhance small molecule drugs by enabling them to activate chimeric antigen receptor T-cells (CAR-Ts). This approach improves small molecule cancer therapy by leveraging their unique binding capabilities with immune cell activation.

Keywords:

CAR-Ts antibodies antitumor agents cell-surface receptors immunotherapy

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Enhancing Chimeric Antigen Receptor-Extracellular Vesicles (CAR-EV) Technology: The Future of Cancer Therapy

Enhancing Chimeric Antigen Receptor-Extracellular Vesicles (CAR-EV) Technology: The Future of Cancer Therapy

Published on: September 19, 2025

Area of Science:

Biotechnology
Immunology
Oncology

Background:

Cell surface macromolecules are primary drug targets, but small molecules can access unique binding pockets with diagnostic and therapeutic potential.
Small molecules face limitations in biodistribution, half-life, and immune interaction compared to antibodies.

Purpose of the Study:

To develop a method for enabling small molecules to recruit and activate chimeric antigen receptor T-cells (CAR-Ts).
To overcome the limitations of small molecules in cancer therapy by integrating them with CAR-T cell technology.

Main Methods:

Utilized a CAR-T platform incorporating a chemically programmed antibody fragment (cp-Fab) as a switch.
Developed a system targeting folate binding proteins on cancer cells.

Main Results:

The cp-Fab/CAR-T system demonstrated potent and specific eradication of folate-receptor-expressing cancer cells.
Proof-of-concept studies confirmed efficacy both in vitro and in vivo.

Conclusions:

Small molecules can be engineered to activate CAR-T cells, offering a new therapeutic strategy.
This chemically programmed CAR-T system shows promise for targeted cancer therapy by combining small molecule specificity with immune cell effector function.