National Strategy for Gene and Cell Therapies opens up new perspectives for patients

In fall 2022, the Federal Ministry of Education and Research (BMBF) commissioned the Berlin Institute of Health at Charité (BIH) to coordinate the creation of a national strategy for gene- and cell-based therapies. Around 150 experts from various stakeholder groups drew up the paper and developed a roadmap for improving healthcare and strengthening Germany as a location for gene- and cell-based therapies. Several experts from the German Centers for Health Research (DZG), to which the DZL belongs, were also represented in the various working groups.

Gene and cell therapies (GCTs) are some of the key technologies driving innovation in biomedical research and patient care. They are not only used to modulate disease processes and alleviate symptoms, but also directly address the genetic causes of diseases. This opens promising perspectives for patients suffering from severe and rare diseases for which no treatment currently exists.

In order to improve patient access to gene and cell therapies and to strengthen Germany as an international hub for research and innovation in this field, the BMBF commissioned the BIH in fall of 2022 to coordinate and oversee the development of a National Strategy for Gene and Cell Therapies. This strategy was presented to the Federal Minister of Education and Research, Bettina Stark-Watzinger, on June 12, 2024.

“The National Strategy for Gene and Cell Therapies is an important step towards securing and expanding Germany's position as a centre of biomedical innovation. Our declared aim is to create new treatment options for patients in the long term. I am very pleased that we have succeeded in bringing together so many stakeholders from different areas and jointly developing the National Strategy. This collaboration between science, industry, the public sector and society is an important key to success. I would like to thank all those involved for their great commitment. With this spirit of optimism, we should now move forward together in a national network." says Federal Research Minister Bettina Stark-Watzinger.

Interdisciplinary measures in eight action areas

The National Strategy is chiefly characterized by its multi-stakeholder approach, which brought together a wide range of views and interests from science, business, politics, society and patient groups. Several experts from the DZG were also represented in the various working groups formed for this purpose.

The fields of action include the following topics:

I. Stakeholder networking and support
II. Training and development of skills
III. Technology transfer
IV. Standards, norms and regulatory framework
V. Improvement of quality and capacity for good manufacturing practice (GMP) production
VI. Research and development
VII. Marketing authorization and translation into patient care
VII. Interaction with society

The strategy emphasizes the tremendous potential that the cutting-edge field of gene and cell therapy has for improving patient care and the healthcare industry and for strengthening Germany as a location for pharmaceuticals and innovation. Yet measures must be taken to accelerate the translation of findings from Germany’s strong basic research programs into clinical practice, while at the same time making gene and cell therapies not only safe and efficient but also affordable and widely accessible.

What’s next for the National Strategy for Gene and Cell Therapies?

The proposed measures will now be implemented step by step in collaboration with all stakeholders. A number of other activities have already been initiated alongside the drafting of the strategy. These include the creation of a National Network Office for Gene and Cell Therapies, the establishment of the nationwide entrepreneurship program GeneNovate, the offering of low-threshold advice on regulatory issues and the laying of the groundwork for funding to support researchers and projects in the field of gene and cell therapy. The Network Office is tasked with building a national GCT community that brings together all stakeholder groups through independent, cross-site information exchange and networking. A total of 48 million euros is available for the design and implementation of the strategy's measures in the period 2023-2026.

Download: National Strategy for Gene and Cell therapies

Source: BIH press release