Area C: Translation

This area will provide the platforms for translating new REBIRTH developments into the clinical practice and for controlling bio-safety in order to avoid adverse effects of novel drugs or complex medical products. Large and small animal models and pharmaco-toxicological assays will be used to explore unexpected risks both in vivo and in vitro. We have a strong focus on the genetic integrity of modified stem cells using state-of-the-art techniques to identify chromosome aberrations and mutations, and to assign risk categories to these changes. The large-scale production of modified stem cells and iPSCs will lead to an urgent need for quantitative assays for monitoring their genetic integrity. Therefore, it is our aim to establish a European reference centre for pro-active bio-safety assessment. Moreover, we are setting up an academia-based infrastructure for the large-scale production of regenerative products, including platforms for the generation of cell-based and cell-derived or (bio-)chemical regenerative products. Again, a strong focus will be on quality control of these products, and our state-of-the-art GMP facilities will enable clinical-grade regenerative products to be developed. We have established a well-structured framework for investigator- initiated clinical trials meeting all legal requirements under the new EU legislation, which are documented in a series of ongoing and planned clinical trials. These activities are all based on the intention to provide novel regenerative medicine to successfully treat patients, and to protect them from harm.