Bringing research results of gene and cell therapy to the clinic faster

This is the English translation of a press release by the Leibniz Association.

Please find the original press release (in German) here:

https://www.leibniz-gemeinschaft.de/ueber-uns/neues/presse/pressemitteilungen/pressemitteilungen-single/newsdetails/forschungsergebnisse-der-gen-und-zelltherapie-schneller-in-die-klinik-bringen

Bringing research results of gene and cell therapy to the clinic faster

Leibniz scientists significantly involved in national strategy for gene and cell-based therapies.

The international competitiveness of Germany as a research and innovation hub in the field of gene and cell-based therapies (GCT) is to be strengthened in the long term, and access for patients to these novel treatment methods is to be ensured. This is the aim of the national strategy for gene and cell-based therapies, whose strategy paper was handed over to the Federal Minister of Education and Research, Bettina Stark-Watzinger, today at the Futurium in Berlin. Scientists from the Leibniz Association have significantly contributed to the paper, which was created under the leadership of the Berlin Institute of Health at Charité (BIH) on behalf of the Federal Ministry of Education and Research.

Five scientists from the Leibniz Association participated in five of the eight working groups—two of them as spokespersons—that developed the strategy paper through a multi-stakeholder process. From the perspective of the Leibniz participants, the primary focus now is to overcome the obstacles in translating research into clinical practice and to establish continuous coordination and collaboration between politics, science, and industry in the field of gene and cell-based therapies.

Philipp Beckhove, Scientific Director of the Leibniz Institute for Immunotherapy in Regensburg and Professor at the University of Regensburg, served as the spokesperson for Working Group I – Networking and Support of Stakeholders: “Gene and cell-based therapies enable a fundamental innovation—the reprogramming of cells into ‘intelligent’ drug factories. By this, we mean cells that can specifically target diseased organs, recognize disease activity, and heal. Gene and cell-based therapies are still a young discipline, but they have already achieved spectacular successes: from correcting genetic disorders to curing advanced tumors and previously incurable autoimmune diseases. Gene and cell-based therapies undoubtedly represent the medicine of the future and a tremendous source of future economic growth and prosperity. In Germany, cutting-edge science is being conducted in this field. However, translating this into the production of concrete therapeutics and clinical practice is significantly hindered by various obstacles, particularly in Germany. With our initiative, stakeholders in this field have come together to collectively identify ways to position Germany strongly in this field on the international stage. We hope that the federal and state governments will join us in pursuing these paths together.”

Sven Stegemann, Managing Director of the “fiT – Leibniz Joint Lab First in Translation” at the DWI – Leibniz Institute for Interactive Materials in Aachen, served as the spokesperson for Working Group V – Enhancing Quality and Capacity in the Field of GMP Production: “With the dynamic development and successes of gene and cell-based therapies, we are on the verge of the next quantum leap in treating previously untreatable diseases. The ‘National Strategy for Cell and Gene-Based Therapies’ creates important prerequisites to set the course for leveraging the potential available in Germany.
In Germany, there is still a lack of translation of research results into patient applications. To meet the increasing demand for gene and cell-based therapies for clinical studies and patient treatment, appropriate manufacturing capacities according to Good Manufacturing Practice (GMP) guidelines must be provided. Since the production of gene and cell-based therapies requires product-specific manufacturing technologies and operating such GMP units is very costly, it is essential to build and expand suitable GMP infrastructures, including the training of qualified personnel. To achieve this, we recommend, among other things, the creation of a central GMP and regulatory committee for gene and cell-based therapies that continuously analyzes needs and drives forward timely and demand-based advancements. Only in this way can Germany expand its existing strengths in gene and cell-based therapies and take on a leading role in the international competition.”

Eicke Latz, Scientific Director of the DRFZ Berlin and Professor at the Charité in Berlin, was a member of Working Group VI – Research and Development: “Research and development in the field of gene and cell-based therapies is currently experiencing an unprecedented upswing, promising far-reaching impacts on the treatment of numerous diseases. We are witnessing the emergence of the first gene-editing technologies that enable new, specific gene-based therapies, and the development of individual cell therapies that are not only successful in treating tumors but also in curing autoimmune diseases. Furthermore, new methods are being developed for more efficient and targeted delivery of genes and cells to specific parts of the body. Advances in stem cell technology and tissue engineering could lead to methods for regenerating damaged tissue or even growing entire organs in the lab. These promising developments are still in their early stages, but the rapid progress in these areas gives hope for significant breakthroughs in the coming years. It is crucial for Germany to play an active role in translating research into clinical practice. This requires collaboration between science, politics, and industry to adapt the prerequisites for efficient development of new drugs in Germany.”

Members of the Leibniz Association who participated in the development of the National Strategy include:

• Spokesperson for Working Group I – Networking and Support of Stakeholders: Philipp Beckhove (Leibniz Institute for Immunotherapy, Regensburg)
• Spokesperson for Working Group V – Enhancing Quality and Capacity in the Field of GMP Production: Sven Stegemann (Leibniz Joint Lab First in Translation at the DWI – Leibniz Institute for Interactive Materials, Aachen) and as a member: Matthias Edinger (Leibniz Institute for Immunotherapy, Regensburg)
• Member of Working Group III – Technology Transfer: Sven Stegemann (DWI, fiT)
• Member of Working Group IV – Standards, Norms, and Regulatory Framework: Simone Thomas (Leibniz Institute for Immunotherapy, Regensburg)
• Member of Working Group VI – Research and Development: Eicke Latz (DRFZ Berlin)

Further information on the National Strategy for Gene and Cell-Based Therapies can be found at https://www.bihealth.org/en/notices/new-perspectives-for-patients-national-strategy-for-gene-and-cell-therapies-presented-to-the-bmbf

Press contact for the Leibniz Association:
Christoph Herbort-von Loeper
Tel.: 030 / 20 60 49 – 471
Mobile: 0174 / 310 81 74
herbort@leibniz-gemeinschaft.de

The Leibniz Association

The Leibniz Association connects 96 independent research institutions. Their scope ranges from natural, engineering, and environmental sciences to economics, spatial, and social sciences, and the humanities. Leibniz Institutes address socially, economically, and ecologically relevant questions. They conduct both fundamental and applied research, also within the interdisciplinary Leibniz Research Alliances, maintain scientific infrastructures, and offer research-based services. The Leibniz Association emphasizes knowledge transfer, particularly through the Leibniz Research Museums. It advises and informs policymakers, scientists, businesses, and the public. Leibniz institutions maintain close collaborations with universities—such as in the form of Leibniz ScienceCampi—with industry, and other partners domestically and internationally. They undergo a transparent and independent evaluation process. Due to their national significance, the institutes of the Leibniz Association are jointly funded by the federal and state governments. The Leibniz Institutes employ around 20,500 people, including 11,500 scientists. The budget amounts to two billion euros.
www.leibniz-gemeinschaft.de