Safe & efficient somatic gene therapy


This project will explore the use of emerging, highly innovative gene-modifying and delivery technologies and capitalize on recent discoveries in gene expression to develop radical solutions to the problem of precisely controlling the fate and expression of exogenous genetic information in gene therapy. Our networks combines genome editing and exploitation of inhibitory RNA to correct genetic defects, and develops smart delivery vehicles based on advanced viral and non-viral delivery methods. Our efforts will stem from basic technology development driven by leading investigators who have already provided the seminal proof-of-principle of novel approaches to controlled gene transfer and persisting transgenesis. They will grow into a pro-active assessment of the pharmacokinetics and potential toxicology of the new approaches in ad hoc redesigned preclinical models, taking advantage of biomarkers defined in ongoing clinical trials and animal models. In this process, we will also establish novel assays for non-clinical and clinical monitoring of biosafety and efficiency. Both academic and industrial partners work hand-in-hand in this collaborative project, ensuring the highest possible degree of innovation and quality control. We will target both stem cells of different lineages ex vivo followed by transplantation into conditioned hosts and long-lived differentiated tissue cells in vivo. Studies will be conducted in disease entities for which animal models are available to study the acute and long-term consequences of the correction of inborn or acquired genetic defects in somatic cells. Among them, we will focus on diseases for which the need for improved gene transfer tools has been unequivocally shown in clinical experience (hemophilia and SCID-X1), and take advantage of our association with the clinical investigators who have led such trials to ensure access to the most relevant information, models, and patient material. We will thus pave the way to a clinical translation of innovative emerging technologies, and simultaneously establish a pipeline of radical solutions to remaining problems. This will eventually lead to the design of new clinical trials that should result in an increased therapeutic index for the disease of interest. Both animal models and pioneering clinical trials are essential to derive basic laws of dose finding, vector targeting, pharmacokinetics, toxicology and biomarkers for treatment monitoring.


GATC Biotech’s task:

GATC performs high-throughput sequencing and analysis of the genomic integration sites of gene therapy vectors.  A database will be developed which provides the project partners with bioinformatic analysis and statistics relating to the different gene therapy vectors and methods.


Participating partners:

ACIES, Lyon, Fance

Charité - Universitätsmedizin Berlin, Germany

École Polytechnique Fédérale de Lausanne, Switzerland

ETH Zürich, Switzerland

Europaeisches Institut fuer Forschung und Entwicklung von Transplantationsstrategien GmbH (EUFETS), Idar-Oberstein, Germany

Flanders Institute for Biotechnology Leuven, Belgium

Genethon, Evry, France

German Cancer Research Center (DKFZ), Heidelberg, Germany

Hannover Medical School, Laboratory for Experimantal Cell Therapy,  Germany

Inserm (Institut national de la santé et de la recherche médicale), Paris, France

Institute of Child Health London (UCL), UK                                    

Leiden University Medical Center (LUMC), Netherlands

Ludwig-Maximilians-Universität Muenchen (LMU), Germany

Lund University, Sweden

Max-Delbrueck-Center for Molecular Medicine (MDC), Berlin, Germany

Medical Center of Erasmus University Rotterdam, Netherlands

Molecular Medicine S.p.A. (MolMed), Milan, Italy

Paul-Ehrlich-Institute, Langen, Germany

Royal Holloway and Bedford New College (RHUL), University of London, UK

San Raffaele del Monte Tabor Foundation, Milan, Italy                             

Università di Modena e Reggio Emilia (Unimore), Modena, Italy


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