Cell and gene therapies in Leipzig: stronger through networking

Medicine has developed rapidly in recent years, particularly in the field of immunotherapies and innovative cell and gene therapies. These promising therapeutic approaches are able to tackle previously difficult-to-treat diseases more effectively, in some cases even with the potential to cure them. However, there is a significant hurdle between perspective and reality: High costs and complex authorisation procedures are slowing down the widespread use of these therapies. However, more intensive collaboration and the seamless integration of different development phases could pave the way for more efficient, tailored and, above all, economically viable solutions. Leipzig is leading the way in this respect.

Currently many academic research institutions and pharmaceutical companies are driving the development of cell and gene therapies (ZGT) forward: Because these are not only considered a future „wonder weapon“ against cancer, they could also become a gamechanger for numerous hereditary diseases. Recently developed gene therapy drugs, for example, utilise adeno-associated viruses (AAV), which themselves do not cause diseases, but replace defective human genes with functional genes . This type of ZGT alone could according to assumptions be used to treat around 7,000 common diseases 

Challenges in the development of cell and gene therapies

However, as with most modern therapies, the technical and human resources required for the development, authorisation and introduction of ZGT are enormous. The production costs per patient-specific cell therapy are between EUR 200,000 and 300,000. Gene therapeutics for very rare diseases are significantly higher at up to EUR 2 million. Although treatment methods such as chemotherapy or stem cell transplantation with permanent immunosuppression are similarly time-consuming, labour-intensive and cost-intensive, especially in view of social follow-up costs such as lost work and patient care, they are already firmly anchored in the healthcare system. As a result, CBT is not yet available to large sections of the population.

CGTs would be more economically competitive if the time and associated costs for basic research, early drug development, GLP studies, clinical trials, production of test samples, clinical trials and commercial applications were optimised. This could be achieved by better dovetailing and holistic integration of these elements in one place or from a single source. So far, however, such integration has been the exception rather than the rule, especially in Europe. This is due to the fact that only a few pharmaceutical companies cover all the necessary components of the development and production of cell and gene therapeutics, including clinical testing and approval, themselves

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Holistic approach necessary

Drug pipelines are largely filled by academic research and smaller start-ups, GLP studies and the clinic are often outsourced to specialised companies or application-oriented research institutions. The cost-intensive clinical testing is carried out by financially strong pharmaceutical companies and the production of authorised drugs by contract manufacturers with corresponding production capacities. ZGTs that are seeking authorisation as an Advanced Therapy Medicinal Product (ATMP) are far more complicated and costly to develop than conventional drugs. This is mainly due to the personalised therapeutic approaches and the biological starting material. Living cells, whether from patients themselves or from healthy donors, can only be standardised to a limited extent and require biological expertise in handling and processing.Both the clinical testing and the process transfer to GMP-compliant manufacturing processes according to pharmaceutical standards require in addition to the appropriate infrastructure the expertise for all development steps as well as the close connection to clinical studies and application centres.

Leipzig: bridging the gap between research and clinical application

In Europe, this necessary bridge between research and clinical application is currently being built in Leipzig in particular. The Fraunhofer Institute for Cell Therapy and Immunology IZI has more than 17 years of expertise in the production of ZGT and the monitoring of studies and approval processes. Here, experimental stages of drug development are transferred to application through GLP testing, process development and the GMP-compliant production of clinical test samples. The Fraunhofer IZI was already involved in the production of the clinical test samples for the European approval study for the world's first CAR-T cell therapy called Kymriah (manufacturer Novartis). Since 2006, the Fraunhofer IZI has produced more than 3,500 cell therapy samples for clinical trials, including over 500 CAR-T cell products. There is currently nothing comparable in Europe.

The Fraunhofer IZI also addresses the increasing demand for cell and gene therapeutics by developing new technologies for the (viral and non-viral) modification of immune cells together with clinical and industrial partners. New effector cells for cell-based therapies, such as natural killer cells (NK cells) and macrophages, are also being investigated and tested. A central focus of Fraunhofer IZI research is the development of CAR-NK cells for the treatment of solid tumours, which have not yet been effectively addressed by CAR-T cell therapies.

SaxoCell hub

Close cooperation with certified and experienced application centres in the immediate vicinity promotes a climate of innovation and enables the rapid transfer of knowledge and applications. These centres include the Leipzig and Dresden University Hospitals and Chemnitz Hospital. Thanks to their close links to the academic world, a seamless transition from research to clinical practice is guaranteed. The Fraunhofer IZI is also a key member of the SaxoCell innovation cluster. This consortium of Saxon research institutes, hospitals and industrial partners is the only future cluster funded by the BMBF that brings together ZGT with automation technologies, digitalisation and artificial intelligence.

Perspectives for Leipzig in the life sciences

Thanks to this networked life sciences & ecosystem, Leipzig has the potential to continue to assert itself as a leading European hub for regulatory testing, process development and ZGT production. The city and region are also making a name for themselves in future-oriented sectors such as diagnostics, digital health, robotics and artificial intelligence. The conditions for future growth are good, as city and state politicians have not only recognised the strategic importance of these technologies: All stakeholders involved are also aware that, in addition to the provision of appropriate funding instruments and financial resources, further networking and cooperation with each other will be crucial for the future.

Article from the "Kapitalmarkt-Portal - goinpublic.de" from 26/09/2023