"The Product is the Process – Is it?" Manufacturing and Translation of ATMPs and Tissue- & Cell-based products

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Summary

Whether in regenerative medicine, cell therapy or gene therapy, research success stories increasingly bring innovative therapy and product concepts involving advanced therapy medicinal products (ATMPs) into the forefront.

Several products have already received pharmaceutical marketing authorisations and are in clinical use, while others are on the verge of doing so. A large number of other product initiatives are in the early stages of development or clinical trials. A complex and dynamic regulatory environment has concurrently emerged in this modern technological field.

The scientific lectures were focussed on the topic of innovative cell therapeutics and gene therapeutics for the treatment of congenital and acquired diseases of the haematopoietic system.

The keynote speaker, Professor Marina Cavazzana, Head of the Biotherapy Department at Necker Hospital Paris, focused on classical and new lentiviral vectors for gene therapy of sickle cell anaemia and beta thalassemia.

Professor Roland Meisel, Deputy Clinic Director and Head of Paediatric Stem Cell Therapy at the University Hospital Düsseldorf, presented the latest clinical data on "exa-cel", a drug based on ex vivo gene editing (GE).

Several lectures addressed regulatory issues. Concrete examples were provided by Professor Heiko von der Leyen's lectures on possibilities for CAR T cell production as well as by Dr Martin Schleef with regard to the production and quality control of plasmid-based vector systems and derivatives.

The first insights into the current and future use of AI systems (artificial intelligence) in medicinal product development and monitoring, presented by Liam Childs from the Paul-Ehrlich-Institut, were not to be missed.

The event is planned to be held again at the end of 2024.