European Network for the Advancement of Clinical Gene Transfer and Therapy

The field of gene therapy has matured and the prospects are exciting and hopeful, particularly since some treatmentshave now been shown to be effective in the clinic. However, precise quality and safety standards for clinical genetransfer have yet to be defined. In this context, defining optimal methods for the production of standard vector systemswould pave the way for accelerated development and improved safety. This would be of enormous value to industry,individual investigators and regulators.The goal of this proposal is the creation of a European Network for the Advancement of Clinical Gene Transferand Therapy (CLINIGENE) integrating multidisciplinary research and development in gene therapy as well asmobilising all major stake holders involved in the development of gene therapy medicinal products: academia,industry, regulatory bodies, clinics and patients. The network will generate platform databases for particular vectorswith respect to their safety and efficacy to ensure product manufacturing according to well-defined quality and safetystandards in order to accelerate clinical trials. This will be achieved by compiling all available information and thenranking test and control methods by comparison, and through validation by expert partners.The joint programme of activities comprises1. Integration activites: sharing facilities, exchange and high-level training of personnel, e-communication andcollaboration with the ESGT.2. Research activities: six horizontal activities serving integration towards the generation of reference/standard profiledata-bases - AAV, γ-retrovirus, lentivirus, adenovirus, genetically-modified cells & non-viral vectors - and fourvertical activities defining a path to optimised clinical protocols - quality and efficacy (manufacture), safety (pharmtoxand virus safety); pre-clinical models and novel assessment tools, clinical trials.3. Dissemination activities: training, high-level education, communication (including a web-site with scientific &medical data-bases) and management of shared information and intellectual property rights.Within a strong integration plan, the Network is planning for flexibility in order to adapt to : (i) progresses recorded ina stepwise manner and (ii) novelty arising during the CLINIGENE workprogramme.


Academic Partners

FCSR-TIGET, San Raffaele Telethon Institute for Gene Therapy, Milano, Italy

Institute of Ophthalmology, University College London,
Div. of Molecular Therapy, London, United Kingdom

TIGEM - Telethon Institute of Genetics and Medicine, Napoli, Italy

Orphanet - INSERM SC11
Hôpital Broussais, Paris, France

Medizinische Hochschule Hannover
Dept. Experimental Hematology, Hannover, Germany

INSERM, France

CBATEG - Autonomeous Univ. of Barcelona, Centre de Biotec. Animal i Teràpia,
Bellaterra, Spain

Academy of Sciences of the Czech Republic,
Institute of Molecular Genetics,
Prague, Czech Republic

IBET - Instituto de Biologia Experimental e Technologica, Oeiras, Portugal

Paul-Ehrlich-Institut, Langen, Germany

Ecole Normale Supérieure de Cachan, Cachan Cedex, France

Royal Holloway & Bedford University, School of Biological Science,
Egham, Surrey, United Kingdom

Mayo Clinic, Molecular Medicine Programme, Rochester, United States

Karolinska Institutet, Department of Medicine, Huddinge University Hospital,
Stockholm, Sweden

Technischen Universität München, Institut für Experimentelle Onkologie und Therapieforschung, München, Germany

Helmoltz Centre for Infection Research, Braunschweig, Germany

Klinik für Neurologie der Universität zu Köln,
Labors für Gentherapie & Molekulares Imaging am MPI für Neurologische Forschung, Köln, Germany

APHP - UMR 7087, UPMC/CNRS-CERVI, Hôpital de la Pitié, Paris, France

University of Ulm, Division of Gene Therapy, Ulm, Germany

Institut de Biotechnologie - Univ. de Lausanne, Lausanne, Switzerland

Genethon, Evry, France

CNRS, UMR 8121, Institut Gustave-Roussy, Villejuif, France

CHU Hotel Dieu Nantes, Laboratoire Therapie Genique, Nantes, France

The Hebrew University, Hadassah Medical School, Jerusalem, Israel

ISTEM (Unité INSERM), Evry Cedex, France

INSERM, Faculté de Sciences Pharma. et Biologiques, Paris, France

German Cancer Research Center (DKFZ), NCT-Heidelberg im Otto-Meyerhof-Zentrum, Heidelberg, Germany

University of Eastern Finland, A.I.Virtanen Institute, Kuopio, Finland

Industry Partners

Bioalliance Pharma, Paris, France

Bioreliance Ltd, Stirling, Scotland, United Kingdom

CellGenix, Freiburg, Germany

CleanCells, Bouffere, France

Epixis, Paris, France

Genosafe, Evry, France

Oxford BioMedica, Oxford

Plasmid Factory, Bielefeld, Germany

Transgene, Strasbourg, France




Hansjörg Hauser


École normale supérieure de Cachan (F)


Funding Agency