Net4CGD : Advancing Gene Therapy for a Rare Primary Immune Deficiency

Net4CGD : Advancing Gene Therapy for a Rare Primary Immune Deficiency

Participation of NET4CGD at the 3rd International Rare Diseases Research Consortium Conference - Conference Center University Pierre et Marie Curie - Paris, France (February 8-9, 2017)

Poster abstract #17:

A. Galy1, G. Santilli2, S. Stein3, J. Schwaeble4, U. Siler5, A. Magnani6, S. Pouillot7, K. Kuehlcke8, M. Schmidt9, T. Paprotka10, G. Honnet1, F. Mavilio1, M. Grez3, H. Serve4, J. Reichenbach5, S. Blanche6, H.B. Gaspar2, M. Cavazzana6, A.J. Thrasher2.

1. Genethon, Evry, France, 2. University College London, London UK, 3. Chemotherapeutisches Forschungsinstitut, Georg-Spreyer-Haus Stiftung, Frankfurt, Germany, 4. Klinikum der Johann Wolfgang Von Goethe Universitaet, Frankfurt, Germany, 5. Universitaet Zuerich, Zurich, Switzerland, 6. Assistance Publique - Hopitaux de Paris, Hopital Necker Enfants-Malades, Paris, France, 7. Genosafe, Evry, France, 8. Eufets, Idar-Oberstein, Germany, 9. Deutsches Krebsforschungszentrum (DKFZ), Heidelberg, Germany, 10. GATC Biotech, Constance, Germany.

The Net4CGD European consortium project (FP7) is focused on the clinical development of gene therapy for patients with the X-linked form of chronic granulomatous disease (X-CGD). This debilitating primary immunodeficiency is caused by an absence of NADPH oxidase activity affecting phagocyte function. The disease is marked by elevated susceptibility to bacterial and fungal infections, as well as an excessive inflammatory response leading to granuloma formation. Current treatments of X-CGD are not entirely satisfactory and prior attempts at autologous gene therapy have failed. A new gene therapy strategy has been developed and is based on the use of a lentiviral vector expressing high levels of the gp91phox transgene in myeloid cells. With encouraging preclinical results, an orphan drug designation was obtained.

The Net4CGD consortium aims to develop this new orphan drug by conducting phase I/II trials in eligible patients. The Net4CGD consortium includes 7 scientific and clinical expert centers and SMEs. Since the start of the Net4CGD project, we have manufactured several lots of clinical-grade vector and a European multi-centric gene therapy trial for X-CGD has been successfully initiated. At present, 5 European centers are currently open in 4 countries to recruit adult or pediatric patients. While the clinical studies are ongoing, efforts are ongoing to develop state-of the art biological analyses for assessment of hematopoietic stem cell quality and patient biological response monitoring and to ensure high-quality harmonization of products and procedures to facilitate future product registration.

Poster is available here.