NON-VIRAL GENE-TRANSFER MEDICINAL PRODUCT - DESIGN AND RESULTS OF GLP PRE-CLINICAL STUDY

As an approach to “therapeutic angiogenesis” we have developed a cell-based, non-viral gene-transfer medicinal product using autologous gene modified fibroblasts (GMF) to produce bFGF and VEGF165 temporarily, as a form of pharmacological local preconditioning before tissue ischemia prevails. We specified product-specific and general risk factors of the gene therapy approach and discussed them with public authorities and regulatory experts. Furthermore, we established GMP (good manufacture practice) production of a gene therapy medicinal product (GTMP) and carried out pre-clinical studies including toxicity study, tumorigenecity study, biodistribution / plasmidmigration, target tissue selectivity and environmental shedding.