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

1:1
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.

Acces this publication
CELL & GENE THERAPY INSIGHTS

Year of publication

2025

Source

Science DIrect

Author

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

You might also be interested in

ERDERA Accelerator webinar (1)

ERDERA launches Ethics & Regulatory webinar series with introductory webinar on ethics across the research continuum

ERDERA’s ethics and regulatory experts are launching a dedicated webinar series to help the rare disease research community navigate two fast-moving areas of biomedical research: Advanced Therapy Medicinal Products (ATMPs) and Artificial Intelligence.
webinar series ERDERA

Introductory webinar: Situating ethics in the research continuum

July 24 @ 3:00 pm - 4:00 pm
This webinar will present a theoretical framework for situating ethics across research activities. The framework will then be brought to life through a relevant case study discussion, giving participants a concrete reference point for the episodes ahead.
NMG workshop 2026

ERDERA National Mirror Groups workshop: Creating, evolving and utilising national registries to advance rare disease research

Held in Riga on 9–10 June, the workshop brought National Mirror Group experts, researchers, clinicians and policymakers together to exchange practical lessons on how national rare disease registries can better support research and alignment across countries.
2

ERDERA launches Clinical Trial Call 2026 to advance early‑phase clinical trials for rare diseases

ERDERA (the European Rare Diseases Research Alliance) has opened its Clinical Trial Call to support multinational early-phase clinical trials in rare diseases. The call will fund studies designed to generate robust clinical evidence, strengthen regulatory readiness and make rare disease trials more feasible across countries.

ERDERA's

search form