Gene Editing for Cell Therapy
Dimitrios L. Wagner
Gene editing and synthetic biology: developing cell therapies for diseases with high unmet medical need.
Dr. Dimitrios L. Wagner, M.D. / Ph.D.
Charité – Universitätsmedizin Berlin
BIH Center for Regenerative Therapies
Augustenburger Platz 1
Telefon:+49 (0)30 450 524 205
Chronic diseases such as cancer and autoimmune diseases place a heavy burden on patients and the healthcare system. Long-term treatment with drugs with unspecific effects can also lead to serious side effects. Novel cell therapies allow us to exploit the potent properties of the body's own cells and to enable long-term and profound treatment successes by means of "living drugs". One example is CAR-T cell therapy, in which the body's own immune cells called T lymphocytes are equipped with artificial cancer-specific recognition receptors (Chimeric Antigen Receptors: CAR) to detect and eliminate blood cancer cells in the patient’s body.
Our junior research group develops platform technologies to design the genetic identity of cells and use it to solve medical problems. Here we focus on improving the latest gene editing technologies from basic research and try to optimize them for clinical application. The goal is to improve the properties of cell products and simplify production to reduce costs and improve access to these promising therapies for patients.
Currently, we are focusing on CRISPR-Cas-based methods that avoid viruses for gene delivery and instead use synthetic nucleic acids and recombinant proteins. By combining synthetic reagents with physical or chemical delivery modalities, we aim to develop safe and efficient techniques for the next generation of novel and effective cell therapies in Berlin.
In addition, we are interested in the immunological aspects that influence the efficiency and safety of cell and gene therapies in the patient's body. Among other things, we want to find out whether and how immune cells from healthy donors can be used for cell therapy of sick people. Our medium-term goal is to develop so-called "off-the-shelf" cell therapies, which are produced from material from healthy people and can thus be used for the rapid and safe treatment of many patients from a single manufacturing run.