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

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.

Year of publication

2025

Source

Science DIrect

Author

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

You might also be interested in

ERDERA marked the close of its second year with an Open Session on ultra-rare diseases, bringing together European, regulatory and scientific perspectives on how innovation can reach patients.
The Joint Transnational Call is expected to open on 1 December 2026, with an information webinar planned for 14 December.
December 14 @ 3:00 pm - 4:00 pm
This information webinar on the 2027 Joint Transnational Call for Proposals is scheduled for 14 December 2026.
The assessment compares diagnosis, treatment access and national governance across 16 countries, examining gaps in rare disease support throughout the life course.