This was the 13th MEDICA-Forum organized by BIO.NRW since the first event in 2010. Despite it being the 13th year, there was nothing unlucky about it – this was the most well-attended BIO.NRW MEDICA-Forum ever. It is therefore without doubt that the “Application of Gene and Cell Therapies Made in NRW” remains a hot topic.

The major significance of this technological achievement lies in the fact that gene and cell therapies (GCTs) can give seriously ill people, for whom there are no real treatment options, new prospects for the future.

Germany and also North Rhine-Westphalia (NRW) have a lot to offer in this field of high technology. In order to promote the promising GCT research approaches throughout Germany in a targeted manner and to bring them to application, a national strategy for gene and cell therapies was published in mid-2024.

In the opening talk of the MEDICA-Forum, Dr. Elke Luger presented the supporting measures and instruments of the National Network Office Gene and Cell Therapies in Berlin, of which she is the head. This central contact and exchange platform encompasses measures such as training, networking, regulatory support, the creation of a national GCT atlas, and much more. There is a particular focus on promoting the next generation of scientists in the implementation of the national GCT strategy. With “GeneNovate”, innovators and young entrepreneurs are being offered a modular training programme in areas such as business models, IP protection, the legal basis for establishing a start-up, financing, and funding. Above all, the goal is to develop innovative products and commercial solutions that can be used to help patients.

During the second talk at the BIO.NRW MEDICA-Forum, Prof. Dr. Jørgen Magnus, who is head of the Chair of Bioprocess Engineering at RWTH Aachen University and has a wealth of industry experience, focused on the manufacturing process of gene and cell therapies. He drew particular attention to a special aspect of cell cultivation for therapeutic use, which is that the cells themselves are the product of the process and also the focal point. In traditional cell culture methods, the cells serve to produce a substance and the focus is on this product. In the production of gene and cell therapies, it is the other way around. The phenotypic analysis of the cell product that has the most diverse possibilities of all omics technologies is therefore of particular importance. In addition, the production technologies currently available for cell therapies are unsuitable for large-scale application. Production methods using adeno-associated viruses (AAVs) have a lot of potential for optimization. However, many more experiments and statistical designs of experiments (DoEs) are needed to identify critical process parameters and to gain a sufficiently deep understanding of production processes and their quality attributes to ensure that the cell produced is ideally adapted to its function/task.

During his talk, Dr. Hatim Hemeda from PL BioScience GmbH spoke about the possibilities of optimizing cell cultivation in various fields of application. Alongside gene and cell therapies, innovative cell culture methods also offer new options in the field of tissue repair, for organs created in the laboratory, and for the healing of autoimmune diseases. The key element of all these potential applications is the cell and the optimization of the respective growth conditions for cell-specific cultivation. The method developed by PL BioScience uses human platelet concentrates that can no longer be used for transfusion. Platelet lysates are obtained from the concentrates, which are rich in valuable growth factors and cytokines. They enable and support the growth of particularly demanding cells, thus opening up new possibilities in the field of cell-based therapies and the application of extracellular vesicles.

In his talk at the BIO.NRW MEDICA-Forum, Prof. Dr. Klump spoke about application-oriented research in the context of gene and cell therapies. With regard to gene therapy, he referred to a publication from 2021 in The New England Journal of Medicine. Clinical investigational medicinal products with CRISPR-Cas9 gene editing for sickle cell anaemia and beta thalassaemia were successfully administered to two patients. Current approaches in research and clinical studies largely focus on gene and cell therapies. Stem cells are the most important target cells, as Prof. Klump demonstrated using the example of the haematopoietic cell system with its associated diseases. Cells and tissues derived from well-studied and characterized induced pluripotent stem cells are ready to be translated into applications. This is a resource with enormous potential for the future.

Dr. Apel from Miltenyi Biotec GmbH gave a talk on the applications and future prospects of gene and cell therapies, providing a good overview of the current state of activities – which cell types are being used and what is their clinical focus in each case. Almost three quarters of all clinical applications are in the field of oncology, and CAR T cells are used for therapy in nearly 50 % of cases. In addition to developments of new gene and cell therapies, the biopharmaceutical company Miltenyi Biomedicine GmbH has been offering clinical research and development programmes for a number of years now. There is a particular focus here on therapeutic options for haematological cancers. In addition, the company is working on regenerative therapies for the treatment of Alzheimer’s, Parkinson’s, and diabetes among elderly patients.

The BIO.NRW MEDICA-Forum, which ran for 90 minutes, was packed with expert knowledge as well as insights into exciting product developments and new technologies. We’re already looking forward to next year’s event!