The Medical Center – University of Freiburg (UKL-FR) is one of the largest medical centers in Europe, with representations of all medical specialties. The medical faculty is part of the Albert-Ludwigs-University Freiburg, which can be traced back to 1457, hence one of the oldest and most distinguished Universities in Germany. UKL-FR´s Institute for Cell and Gene Therapy (IZG) will be involved in CARAT. The institute was established in 2013 by merging the Department of Transfusion Medicine, which provides the Medical Center with blood products and all transfusion and transplantation related diagnostic services, with a GMP clean room facility, and newly established research units at the Center for Chronic Immunodeficiency. In consequence, the IZG combines an established facility for diagnostic services with specialized personnel for GMP-compliant manufacturing processes and research groups focusing on instituting new perspectives in cell and gene therapy for patients suffering from rare immunodeficiencies, HIV infection, and leukemia. The three major research goals of the institute are (i) to further improve safe genome editing tools (incl. TALENs, CRISPR-Cas) for therapeutic applications in human stem cells and T cells; (ii) to develop disease models and cell therapies based on induced pluripotent stem cells (iPSCs); and (iii) to translate cell and gene therapy efforts into the clinic.
UKL-FR is the leader of Work Package 3. The lab’s expertise in targeted genome engineering as well as viral (AAV and lentiviral vectors) and non-viral (GMP-compatible transfer of mRNA) gene transfer technologies will permit the fast development and evaluation of highly specific nucleases as well as their transfer and application in primary human cells. In collaboration with partners Miltenyi and PEI, GMP-compliant cell separation as well as nuclease and donor transfer protocols will be established. To evaluate the genetically modified CAR T-cells for improved anti-tumour activity partner UKL-FR will team up with OSR and INSERM.