Conférence Dr Anne GALY, DR Inserm UMR 951 PDF Imprimer Envoyer
Vendredi, 28 Mars 2014 16:18
Conférence Dr Anne GALY, DR Inserm UMR 951

Gene therapy strategies for rare primary immune deficiencies

UMR 951 Inserm
Immunologie et Biothérapies Innovantes
1bis rue de l'Internationale - Evry

Vendredi 4 avril 2014

Amphi 3 - Faculté de Médecine, 22 av. Camille Desmoulins - Brest

Gene therapy strategies for rare primary immune deficiencies


Gene therapy provides remarkable possibilities to treat monogenic disorders and in the process, helps to better understand these diseases while advancing the field of biomedical technology. We have developed a multi-center international strategy to rapidly assess the safety and efficacy of gene therapy in severe primary immune deficiencies such as Wiskott Aldrich Syndrome (WAS) or X-linked chronic granulomatous disease. Each of these diseases has an estimated incidence of 4 to 10 cases per million live births and our goal is to provide patients and families, an access to treatment for these very rare cases. An international strategy is developed to conduct phase I/II studies in Europe and in the US. Patients are transplanted with gene-modified autologous hematopoietic stem cells following disease-adapted conditioning regimens. Gene transfer vectors used in these studies are SIN lentiviral vectors (LV) that express the therapeutic cDNA at physiological levels in target cells. Robust manufacturing capacity of clinical-grade LV was established at Genethon to supply multiple clinical centers with high titer of standardized purified vector lots in quantities sufficient to treat infants and young adults. Encouraging results have been obtained in the WAS trials in which patients have now been treated without safety issues. Ongoing efforts aim to optimize technologies and models that will expedite the development of these new treatments.

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