Charité – Universitätsmedizin Berlin, Germany (coordinator) | CHARITE
The CHARITÉ – UNIVERSITÄTSMEDIZIN BERLIN is one of Europe’s largest University hospitals (about 18,000 employees, >1,000,000 patients/year).
Cell and Gene Therapy is one of the main clinical research areas of the Charité.
With the two structural units BCRT and BeCAT, CHARITE has been able to combine expertise in gene and cell therapies from the early development pipeline to clinical application, resulting in comprehensive and synergistic benefits and making the field attractive to scientists and clinicians.
Charité´s Berlin Center for Advanced Therapies (BeCAT) is a research and development center for innovative ATMP-based therapy concepts. The goal of the center is to bring these novel therapy concepts into clinical practice quickly and in the sense of precision medicine in order to contribute to the improvement of quality of life, survival and health economy.
Dimitrios Laurin Wagner
Medical Center – University Hospital Freiburg, Germany | UKLFR
The Universitätsklinikum Freiburg (UKLFR) is one of the largest medical centers in Europe, with representations of all medical specialties. The Institute for Transfusion Medicine and Gene Therapy (ITG) provides the hospital with blood products and all transfusion and transplantation related diagnostic services. Its research goals are (i) to improve genome editing tools (TALEN and CRISPR-Cas) for therapeutic applications in human cells, (ii) to develop preclinical in vitro and in vivo disease models and genotoxicity assays to validate the developed approaches, and (iii) to translate immune cell and stem cell therapies to the clinic for the treatment of certain types of cancers, HIV infection and primary immunodeficiencies.
Of particular interest to geneTIGA are the developed assays to characterize off-target effects of genome editors. In particular, partner UKLFR has developed and owns intellectual property on two novel NGS-based methods (Abnoba-Seq and CAST-Seq) that enable researchers to nominate and quantify on- and off-target effects following CRISPR or TALEN-mediated genome editing in a truly unbiased manner.
Lead CAR technology
Carla Fuster Garcia
Lead Genotox assays
Technische Universität Braunschweig, Germany | TU-BS
The Technische Universität Braunschweig (TUBS) is the oldest university of technology in Germany and was founded in 1745 as Collegium Carolinum. TUBS is a member of the TU9, an association of the leading German universities of technology. The departments of Biotechnology and Medical Biotechnology of the TU Braunschweig have a long lasting experience in antibody development for basic research, diagnostics and therapy. The main focus of the departments headed by Stefan Dübel and Michael Hust is antibody engineering, phage display and biomarker identification. The antibody engineering activities resulted in several spin-off companies and currently two antibodies are in clinical studies. They got several innovation awards for their translational research activities.
The competence of this partner is antibody generation and antibody engineering. TUBS brings in geneTIGA the recognized expertise in CAR receptor identification and design.
Oslo University Hospital, Norway | OUS
OUS brings extensive experience in the identification of specific T-cell receptors (TCRs) and the generation of redirected T-lymphocytes using transgenic TCRs, an alternative approach to CAR-T.
The Institute for Cancer Research, Oslo University Hospital (OUH), Oslo, Norway (http://ous-research.no/institute/) is the largest cancer institute in Norway. The Comprehensive Cancer Center (CCC) of OUH Radiumhospitalet is one of the largest Cancer Centers in Europe with its own Phase I/II clinical trial unit and a large academic facility for good manufacturing practices (GMP) production of cells for immunotherapy, housing a newly established Center for Advanced Cell and Gene Therapy (ACT). These units are integrated with the Department of Cancer Immunology (https://ous-research.no/immunology/) and clinical departments, giving OUH a competitive edge in bringing “in-house” discoveries to early clinical trials.
Morten Milek Nielsen
Aarhus University, Denmark | AU
Aarhus University is Denmark’s second-largest university with 38,000 students and ranks among the top 100 universities in the world in several international rankings. The Department of Biomedicine at Aarhus University bridges the divide between natural science and clinical medicine, and houses several research leaders in the field of gene therapy and gene editing.
In geneTIGA, researchers from Aarhus University will contribute with long-standing experience in viral gene vectors and precise gene editing technologies for efficient genetic engineering of optimized immune cells.
University of Oxford, UK (associated partner with own funding from UKRI) | UOXF
The University of Oxford (UOXF) is a world-leading centre of biomedical research with close links to the Oxford University Hospitals NHS Trust, where cutting edge clinical trials are conducted. UOXF has a broad portfolio of preclinical in vivo models with the corresponding read-out techniques, which are essential for testing the efficacy and safety of the cell product candidates, also from a regulatory point of view. In addition, a broad biobank of samples from IgAN patients is available.
ETH Zurich, Switzerland (associated partner with own funding from SERI) | ETHZ
With ETHZ, regularly ranked amongst the top 10 universities in the world according to QS, an outstanding expertise in gene editing is coming on board by providing access to numerous tools for optimizing efficacy and analyzing safety, including validated assays and methodologies used in ongoing clinical genome editing trials to treat hematopoietic diseases.
Prof. Jacob Corn and his team (https://cornlab.com/), located at the Institute of Molecular Health Sciences, has a long record on implementing genome editing technologies on different mammalian systems, and has made substantial contributions to the genome engineering field over the years; from fundamental discoveries on DNA editing requirements in human cells to practical applications of manipulating DNA repair outcomes in disease relevant models.
Particularly relevant to the geneTIGA project is the interrogation of the specificity and safety of genome editing tools by developing DISCOVER-seq, a broadly applicable approach for unbiased Off-target identification in cells and tissues. Apart from offering improved specificity in Off-target site detection, this cell-based method also provides an excellent framework for analyses in systems that cannot be assessed with other state of the art Off-target identification workflows, due to toxicity.
The Genome Engineering and Measurement Lab (GEML), a Knowledge Hub in ETHZ lead by Dr. Zacharias Kontarakis, brings expertise in applying a wide portfolio of genome engineering profiling methods, including DISCOVER-seq, and has already contributed to the development of proof of concept gene therapy processes in non-human primates.
Working closely with the European partners, the ETHZ team will select, design, and optimize safe and efficient next-generation genome editing technologies for the development of IgA-nephropathy treatments.
Professor of Genome Biology, Team leader
Head of the Genome Engineering and Measurement Lab
TissUse GmbH, Germany | TissUse
TissUse, a biotechnology SME, is an international pioneer in innovative microfluid-based multi-organ-on-a-chip technologies. With its expertise, we will jointly succeed in developing the vascularization of the system and the organoids, making the technology attractive as a disease model for testing the efficacy and safety of our gene-edited T-cell products and beyond.
CheckImmune, Germany | CHI
CHI, spin-off of CHARITE, is dedicated to the development, validation and application of immunological tests based on state-of-the-art platforms for final product characterization and therapy monitoring of patients in early clinical trials regarding safety, PK/PD, mode-of-action and surrogate markers, and has a broad international reputation in this field.
geneTIGA-Project Lead at CheckImmune and Head Development & Exploration Laboratory
Chief Executive Officer
Innovation Acta, Italy | INN-ACTA
INN-ACTA is a SME experienced in managing EC funded projects. INN-ACTA’s expertise ranges from grants management to dissemination activities (e.g. printed materials, flyers, brochures, newsletters, project websites, videos), training activities, organization of meetings, conferences, workshops and Project events.
European Kidney Patients Association, Austria | EKPF
EKPF is the European umbrella organization of the numerous national kidney disease patient advocacies. It will serve as a bridge between the consortium and patient organizations to inform relevant patient groups about the latest developments and to build trust for the novel therapy options using gene editing approaches and, on the other hand, to get feedback regarding needs and requests from them to the consortium.
Juan Carlos Julián Mauro (Federación Nacional ALCER)