Breaking barriers in cell and gene therapy: advancing ATMPs through innovation and collaboration

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Effective collaboration between industry, academia, and patient organizations is essential to advance standardized platform technologies. A recent example is the newly launched European Rare Diseases Research Alliance (ERDERA).

In recent years, a slew of advanced therapy medicinal products (ATMPs), particularly cell and gene therapies, have demonstrated the transformative power of these therapies. CAR-T cell therapy, for example, has revolutionized the field in the treatment of patients with various types of relapsed/refractory (R/R) B-cell malignancies including diffuse large B-cell lymphoma (DLBCL), B-cell acute lymphoblastic leukemia (B-ALL), follicular lymphoma (FL), mantle cell lymphoma (MCL), and multiple myeloma (MM). CAR-T cell application in solid tumors, however, remains elusive due to a variety of challenges in the biology. This not withstanding, results published in 2024 brought renewed optimism for the use of CAR-Ts in solid tumors, by showing that targeting two brain tumor-associated proteins instead of one showed promise
as a strategy for reducing tumor growth in patients with recurrent glioblastoma (GBM), an aggressive form of brain cancer.

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CELL & GENE THERAPY INSIGHTS

Year of publication

2025

Source

Science DIrect

Link to cite

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Author

Anton Ussi, Toni Andreu, Florence Bietrix, and David Morrow

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