Obiettivo This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD). This rare primary immune deficiency of phagocytes is caused by mutations in the gp91phox gene. Affected patients are highly-susceptible to infections and develop inflammatory granulomas. Several members of the Net4CGD consortium have already attempted hematopoietic gene correction of X-CGD using gp91 gammaretroviral gene transfer vectors. While functional correction and clinical benefit was initially achieved, problems arose, linked to insertional mutagenesis, vector silencing and lack of long-term engraftment. Net4CGD proposes future trials to achieve (i) effective transduction of hematopoietic cells, (ii) physiological expression of the transgene and (iii) long-term engraftment of gene modified cells. A new lentiviral vector (LV) was developed to express gp91phox in myeloid cells. Encouraging results obtained in preclinical studies and through the compassionate treatment of a patient, prompt us to test the LV in a multi-center study in several European centers expert in CGD, under the sponsorship of a rare disease specialist. The tasks include i) Manufacturing clinical grade vector to support clinical studies, ii) Conducting a multi-center phase I/II trial in eligible X-CGD patients, with LV gene-modified autologous hematopoietic stem cells to evaluate the safety and efficacy of the procedure iii) Ensuring high-quality and harmonization of products and procedures to facilitate future product registration iv) Obtaining state-of-the art information on biological efficacy and safety in patients by assessing immune restoration and large-scale integrome data. If positive, this study will be used to register the orphan drug. The treatment is expected to improve patients’ quality of life and will reduce the economical burden of CGD. Results should benefit other RD. Campo scientifico medical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesmedical biotechnologycells technologiesstem cellsnatural sciencesbiological sciencesgeneticsmutation Programma(i) FP7-HEALTH - Specific Programme "Cooperation": Health Argomento(i) HEALTH.2012.2.4.4-1 - Preclinical and/or clinical development of substances with a clear potential as orphan drugs Invito a presentare proposte FP7-HEALTH-2012-INNOVATION-1 Vedi altri progetti per questo bando Meccanismo di finanziamento CP-FP - Small or medium-scale focused research project Coordinatore ASSOCIATION GENETHON Contributo UE € 2 250 140,00 Indirizzo RUE DE L INTERNATIONALE 1 BIS 91002 Evry Francia Mostra sulla mappa Regione Ile-de-France Ile-de-France Essonne Tipo di attività Research Organisations Contatto amministrativo Jean-Philippe Marin (Mr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato Partecipanti (10) Classifica in ordine alfabetico Classifica per Contributo UE Espandi tutto Riduci tutto UNIVERSITY COLLEGE LONDON Contributo UE € 449 858,00 Indirizzo GOWER STREET WC1E 6BT LONDON Mostra sulla mappa Tipo di attività Higher or Secondary Education Establishments Contatto amministrativo Greta Borg-Carbott (Ms.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG Germania Contributo UE € 270 000,00 Indirizzo PAUL EHRLICH STRASSE 42-44 60596 Frankfurt Mostra sulla mappa Regione Hessen Darmstadt Frankfurt am Main, Kreisfreie Stadt Tipo di attività Research Organisations Contatto amministrativo Robert Dornberger (Mr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato KLINIKUM DER JOHANN WOLFGANG VON GOETHE UNIVERSITAET Germania Contributo UE € 110 250,00 Indirizzo THEODOR STERN KAI 7 60596 Frankfurt Am Main Mostra sulla mappa Regione Hessen Darmstadt Frankfurt am Main, Kreisfreie Stadt Tipo di attività Higher or Secondary Education Establishments Contatto amministrativo Birgit Rosiejak (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato University of Zurich Svizzera Contributo UE € 300 000,00 Indirizzo RAMISTRASSE 71 8006 ZURICH Mostra sulla mappa Tipo di attività Higher or Secondary Education Establishments Contatto amministrativo Janine Reichenbach (Prof.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato ASSISTANCE PUBLIQUE HOPITAUX DE PARIS Francia Contributo UE € 350 455,00 Indirizzo 55 BOULEVARD DIDEROT 75012 Paris Mostra sulla mappa Regione Ile-de-France Ile-de-France Val-de-Marne Tipo di attività Research Organisations Contatto amministrativo Agnès Dauvergne (Ms.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato GENOSAFE SAS Francia Contributo UE € 852 000,00 Indirizzo RUE DE L'INTERNATIONALE 1 91000 EVRY Mostra sulla mappa Regione Ile-de-France Ile-de-France Essonne Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Severone Pouillot (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato BIONTECH INNOVATIVE MANUFACTURING SERVICES GMBH Germania Contributo UE € 751 808,00 Indirizzo VOLLMERSBACHSTRASSE 66 55743 IDAR OBERSTEIN Mostra sulla mappa Regione Rheinland-Pfalz Koblenz Birkenfeld Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Klaus Kuehlcke (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato DEUTSCHES KREBSFORSCHUNGSZENTRUM HEIDELBERG Germania Contributo UE € 300 000,00 Indirizzo IM NEUENHEIMER FELD 280 69120 Heidelberg Mostra sulla mappa Regione Baden-Württemberg Karlsruhe Heidelberg, Stadtkreis Tipo di attività Research Organisations Contatto amministrativo Ina Krischek (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato EUROFINS GENOMICS EUROPE PHARMA AND DIAGNOSTICS PRODUCTS & SERVICES SANGER PCR GMBH Germania Contributo UE € 240 096,00 Indirizzo JAKOB STADLER PLATZ 7 78467 Konstanz Mostra sulla mappa Regione Baden-Württemberg Freiburg Konstanz Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Janet Kenklies (Ms.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato FINOVATIS Francia Contributo UE € 125 000,00 Indirizzo 62 RUE DE BONNEL 69003 Lyon Mostra sulla mappa Regione Auvergne-Rhône-Alpes Rhône-Alpes Rhône Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo David Koubi (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato