Lecturer: Gerald Schumann (Paul-Ehrlich-Institute, Frankfurt)
Advanced Therapy Medicinal Products (ATMPs) are a specialized category of medicinal products for human use that are based on genes, tissues or cells and offer groundbreaking new opportunities for the treatment of disease and injury. They include somatic cell therapy medicinal products, tissue-engineered medicinal products, gene therapy medicinal products and combined ATMPs and are developed to address previously unmet medical needs, often for conditions that are difficult to treat with conventional therapies.
Human pluripotent stem cells (PSCs) are increasingly used as basis for the manufacturing of cell- and gene therapy medicinal products. Safety aspects related to pluripotent stem cell (PSC)-derived cell and gene therapy include tumorigenicity, genetic and epigenetic stability, insertional mutagenesis, immune reactions, differentiation efficiency and purity, manufacturing and quality control and long-term monitoring. After providing a brief overview of ongoing clinical trials with pluripotent stem cell-derived products, this presentation focusses on the potential risk of genomic destabilization PSC-derived products are exposed to during the manufacturing process.