Objective Primary immune deficiencies (PID) are inherited disorders of the adaptive and innate immune system marked by severe infections, autoimmunity and high risk of cancer. Treatment entails hematopoietic stem cell (HSC) transplantation from allogeneic donors, however in the absence of an HLA compatible donor, HSCT outcome is limited by delayed or suboptimal reconstitution and complications. SCID-X1 and ADA-SCID have been successfully treated with autologous gene corrected HSC, however, associated with safety issues inherent to first generation retroviral vectors. This project utilizes genetically modified HSC and their descendants as immunotherapeutic cells to build a healthy immune system in PID patients, and is carried out by clinical centres, scientists and industrial partners pioneering in the field of advanced therapies and aiming at broad clinical application of safe cell-based therapeutic products. Multicentre phase I/II clinical trials for SCID-X1 and WAS are ready to start. Disease targeted technology to cure ADA-SCID, V(D)J recombination defects and CGD by gene corrected HSC and novel approaches in IPEX and HLH to gene modify already committed cells will be investigated. Based on rigorous preclinical efficacy and toxicology evaluation, flanked by basic studies aimed at improving HSC homing capacity and thymic epithelium regeneration, new clinical trials will be implemented. The consortium will establish a technology platform to implement, harmonize and run controlled, standardized multicentre preclinical studies using state-of-the-art advanced therapy. Strict observance of good practice quality guidelines and regulation of medicinal product development will be ensured. The successful completion of the project will be instrumental to accomplish and broaden clinical application of medicinal products able to rebuild and modulate the immune system with an anticipated impact that extends beyond PID to acquired immune disorders, allogeneic HSCT and cancer treatment. Fields of science medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesbasic medicineimmunologymedical and health sciencesmedical biotechnologycells technologiesstem cellsmedical and health sciencesclinical medicineoncologymedical and health sciencesbasic medicinetoxicology Programme(s) FP7-HEALTH - Specific Programme "Cooperation": Health Topic(s) HEALTH.2010.1.4-1 - Translational research on cell-based immunotherapy. FP7-HEALTH-2010-single-stage Call for proposal FP7-HEALTH-2010-single-stage See other projects for this call Funding Scheme CP-IP - Large-scale integrating project Coordinator UNIVERSITA VITA-SALUTE SAN RAFFAELE EU contribution € 1 431 584,08 Address VIA OLGETTINA 58 20132 Milano Italy See on map Region Nord-Ovest Lombardia Milano Activity type Higher or Secondary Education Establishments Administrative Contact Maria Rosa Pedrazzi (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data Participants (19) Sort alphabetically Sort by EU Contribution Expand all Collapse all INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE France EU contribution € 1 415 500,00 Address RUE DE TOLBIAC 101 75654 Paris See on map Region Ile-de-France Ile-de-France Paris Activity type Research Organisations Administrative Contact Annick Bertault (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data UNIVERSITY COLLEGE LONDON United Kingdom EU contribution € 1 195 287,00 Address GOWER STREET WC1E 6BT London See on map Region London Inner London — West Camden and City of London Activity type Higher or Secondary Education Establishments Administrative Contact Greta Borg-Carbott (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG Germany EU contribution € 516 199,00 Address PAUL EHRLICH STRASSE 42-44 60596 Frankfurt See on map Region Hessen Darmstadt Frankfurt am Main, Kreisfreie Stadt Activity type Research Organisations Administrative Contact Manuel Grez (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data WEIZMANN INSTITUTE OF SCIENCE Israel EU contribution € 437 794,00 Address HERZL STREET 234 7610001 Rehovot See on map Activity type Higher or Secondary Education Establishments Administrative Contact Talia Tzahor (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data TECHNISCHE UNIVERSITAET DRESDEN Germany EU contribution € 361 240,00 Address HELMHOLTZSTRASSE 10 01069 Dresden See on map Region Sachsen Dresden Dresden, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Lars Henning (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM Participation ended Netherlands EU contribution € 791 531,64 Address DR MOLEWATERPLEIN 40 3015 GD Rotterdam See on map Region West-Nederland Zuid-Holland Groot-Rijnmond Activity type Higher or Secondary Education Establishments Administrative Contact Gerard Wagemaker (Prof.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data OSPEDALE PEDIATRICO BAMBINO GESU Italy EU contribution € 539 995,00 Address PIAZZA SANT ONOFRIO 4 00165 Roma See on map Region Centro (IT) Lazio Roma Activity type Research Organisations Administrative Contact Sonya Jane Martin (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data MEDIZINISCHE HOCHSCHULE HANNOVER Germany EU contribution € 1 346 634,00 Address Carl-Neuberg-Strasse 1 30625 Hannover See on map Region Niedersachsen Hannover Region Hannover Activity type Higher or Secondary Education Establishments Administrative Contact Frank Dittrich (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data AGC BIOLOGICS SPA Italy EU contribution € 678 270,00 Address VIA MEUCCI, 3 20091 BRESSO MI See on map Region Nord-Ovest Lombardia Milano Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Enrico Cappelli (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data MILTENYI BIOTEC GMBH Germany EU contribution € 395 720,00 Address FRIEDRICH EBERT STRASSE 68 51429 Bergish Gladbach See on map Region Nordrhein-Westfalen Köln Rheinisch-Bergischer Kreis Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Sabrina Schmitz (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data UNIVERSITAT BASEL Switzerland EU contribution € 557 538,00 Address PETERSPLATZ 1 4051 Basel See on map Region Schweiz/Suisse/Svizzera Nordwestschweiz Basel-Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Georg-A Hollaender (Prof.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data EUROFINS GENOMICS EUROPE PHARMA AND DIAGNOSTICS PRODUCTS & SERVICES SANGER PCR GMBH Germany EU contribution € 289 400,00 Address JAKOB STADLER PLATZ 7 78467 Konstanz See on map Region Baden-Württemberg Freiburg Konstanz Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Natascha Ostrowski (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data FINOVATIS France EU contribution € 276 900,00 Address 62 RUE DE BONNEL 69003 Lyon See on map Region Auvergne-Rhône-Alpes Rhône-Alpes Rhône Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact David Koubi (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data BIONTECH INNOVATIVE MANUFACTURING SERVICES GMBH Germany EU contribution € 49 998,00 Address VOLLMERSBACHSTRASSE 66 55743 IDAR OBERSTEIN See on map Region Rheinland-Pfalz Koblenz Birkenfeld Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Klaus Kuehlcke (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data HACETTEPE UNIVERSITESI Türkiye EU contribution € 355 974,36 Address HACETTEPE UNIVERSITESI BEYTEPE KAMPUSU REKTORLUK BINASI 06800 Cankaya Ankara See on map Region Batı Anadolu Ankara Ankara Activity type Higher or Secondary Education Establishments Administrative Contact Sevtap Benli (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data UNIVERSITAT ZURICH Switzerland EU contribution € 49 998,00 Address RAMISTRASSE 71 8006 Zurich See on map Region Schweiz/Suisse/Svizzera Zürich Zürich Activity type Higher or Secondary Education Establishments Administrative Contact Ivo Cotti (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION Netherlands EU contribution € 25 000,00 Address RIJNSBURGERWEG 10 2333 AA Leiden See on map Region West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek Activity type Research Organisations Administrative Contact Marco Van Der Most (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data EUROPESE VERENIGING VOOR GEN- EN CELTHERAPIE IVZW Belgium EU contribution € 27 002,00 Address TIENSESTEENWEG 250 3360 BIERBEEK See on map Activity type Other Administrative Contact Renee Watson (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data FONDAZIONE CENTRO SAN RAFFAELE Italy EU contribution € 1 146 169,92 Address VIA OLGETTINA 60 20132 Milano See on map Region Nord-Ovest Lombardia Milano Activity type Research Organisations Administrative Contact Maria Rosa Pedrazzi (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Total cost No data