Introduction :  First a pioneer of human genome research, Genethon now explores the new therapeutic hypotheses stemming from the extraordinary progresses of contemporary biology.

Today, Genethon is a non-profit biotherapy organisation,  financed at 88.7% by the AFM, Under the management of Dr Anne-Marie Masquelier, CEO, Genethon is a unique structure where researchers, technicians, chemists and physicians elaborate technologies to develop innovative therapeutics, like gene therapy or cell therapy, to treat rare or orphan diseases. Scientist researchers of Genethon focus on therapeutic tools for in vivo or ex vivo gene transfer, on rare diseases and specifically on neuromuscular diseases as well as on immune system or skin diseases. These pathologies are real models to help accelerate the research towards new therapeutic concepts which can subsequently be adapted to more well-known pathologies.

For gene therapy and cell culture, a large engineering team then develops vector manufacturing processes that are transferred to our GMP production service, recently accredited as Gene and Cell Therapy Establishment (Etablissement de thérapie génique et cellulaire -ETGC). The latter provides GMP quality controlled vectors, not only for Généthon’s own projects but for numerous collaborations or external requests. Today we can manufacture vectors for use in gene therapy clinical trials.
Involved in WP : 7, 8 and ILC.

www.genethon.fr

Head of Regulatory Affairs and Quality Assurance in Genethon since 2001.
Didier Caizergues has a Higher Diploma (DESS) in Health and Pharmaceutical Legislation, Faculty of  Pharmacy, University of Paris XI.
 

Otto-Wilhelm MERTEN has a degree in biotechnology and is the head of the department of bioprocess development of Genethon since 1997. He has a large scientific experience gained at the Inst. Pasteur (Paris/F) as well as at the Sandoz Research Inst. (Vienna/A). He was participant in different EU sponsored projects. His contribution to Treat-NMD is mostly in the domain of bioproduction related issues.

Maria Antonietta Zanta-Boussif is Head of Molecular Evaluation section in Genethon, Research and Translational Development. She has a PhD Thesis from L. Pasteur University, Strasbourg, France. She contributes to Treat-NMD with her Bio analytics, cell and Molecular biology and  virology skills.
 

Hafedh Haddad is a medical doctor with a degree in clinical research. He joined GENETHON in 2005 to lead its 1st clinical project: the gamma-sarcoglycanopathy (LGMD 2C) project. Currently in charge of the clinical operations for GENETHON's neuromuscular and rare diseases clinical projects.

Previously, Dr Haddad worked on spinal muscular atrophy, Duchenne muscular disease and congenital myasthenic syndromes at the INSERM, CNRS and the Institute of Myology in Paris.

Preclinical and clinical pharmacology Department Head Research and Development and PhD in Human Genetics since 1996.

Her expertise  is  in Drug Mechanisms of Action; Gene Functional Annotation and Biomarker discovery; Pathologic gene selection and validation, Human genetics and genomics. She currently leads the Preclinical studies and clinical monitoring department at Genethon.
 

With a diploma of Biotechnology (European Educational Program - University of Aberdeen, Scotland) and a Master's degree in Cellular Biology (Nice-Sophia Antipolis University, France), Françoise has been working at Genethon since 1991. A first position as scientific information specialist led her to develop her skills and knowledge in internet resources searching and information management.

Following a professional training in multimedia in 1999, she participated in the development of Genethon’s websites in 2002 and 2006.

As an information manager and webmaster, she is now in charge of the website updates and the editorial and technical coordination of Genethon’s Newsletter.
Genethon, as a partner of Treat-NMD, regularly publishes news related to the research programmes, events, etc. organized by the European Network of Excellence for the treatment of neuromuscular diseases.
 

Marie-Odile OTT – PhD, was trained as a molecular biologist in developmental biology. She has expertise in  European affairs and coordination in European projects. She was National Contact Point for “Life Science, biotechnology and genomic for health priority during the 6th European framework programme.

She managed the ERANET programme “Alliance-O” 2005-2007 and she joined Genethon in 2007 as partnership Development Representative