CELL-PID
Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency
| Coordinatore | UNIVERSITA VITA-SALUTE SAN RAFFAELE
Organization address
address: Via Olgettina 58 contact info |
| Nazionalità Coordinatore | Italy [IT] |
| Totale costo | 15˙799˙071 € |
| EC contributo | 11˙887˙735 € |
| Programma | FP7-HEALTH
Specific Programme "Cooperation": Health |
| Code Call | FP7-HEALTH-2010-single-stage |
| Funding Scheme | CP-IP |
| Anno di inizio | 2010 |
| Periodo (anno-mese-giorno) | 2010-11-01 - 2015-10-31 |
Partecipanti
| # | ||||
|---|---|---|---|---|
| 1 |
UNIVERSITA VITA-SALUTE SAN RAFFAELE
Organization address
address: Via Olgettina 58 contact info |
IT (MILANO) | coordinator | 1˙431˙584.08 |
| 2 |
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
Organization address
address: 101 Rue de Tolbiac contact info |
FR (PARIS) | participant | 1˙415˙500.00 |
| 3 |
MEDIZINISCHE HOCHSCHULE HANNOVER
Organization address
address: Carl-Neuberg-Strasse 1 contact info |
DE (HANNOVER) | participant | 1˙346˙634.00 |
| 4 |
UNIVERSITY COLLEGE LONDON
Organization address
address: GOWER STREET contact info |
UK (LONDON) | participant | 1˙195˙287.00 |
| 5 |
Fondazione Centro San Raffaele
Organization address
address: Via Olgettina 60 contact info |
IT (Milano) | participant | 1˙146˙169.92 |
| 6 |
ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM
Organization address
address: 's Gravendijkwal 230 contact info |
NL (ROTTERDAM) | participant | 791˙531.64 |
| 7 |
MOLECULAR MEDICINE SPA
Organization address
address: VIA OLGETTINA 58 contact info |
IT (MILANO) | participant | 678˙270.00 |
| 8 |
UNIVERSITAET BASEL
Organization address
address: Petersplatz 1 contact info |
CH (BASEL) | participant | 557˙538.00 |
| 9 |
OSPEDALE PEDIATRICO BAMBINO GESU
Organization address
address: PIAZZA SAN ONOFRIO 4 contact info |
IT (ROMA) | participant | 539˙995.00 |
| 10 |
CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG
Organization address
address: PAUL EHRLICH STRASSE 42-44 contact info |
DE (FRANKFURT) | participant | 516˙199.00 |
| 11 |
WEIZMANN INSTITUTE OF SCIENCE
Organization address
address: HERZL STREET 234 contact info |
IL (REHOVOT) | participant | 437˙794.00 |
| 12 |
MILTENYI BIOTEC GMBH
Organization address
address: FRIEDRICH EBERT STRASSE 68 contact info |
DE (BERGISCH GLADBACH) | participant | 395˙720.00 |
| 13 |
TECHNISCHE UNIVERSITAET DRESDEN
Organization address
address: HELMHOLTZSTRASSE 10 contact info |
DE (DRESDEN) | participant | 361˙240.00 |
| 14 |
HACETTEPE UNIVERSITESI
Organization address
address: HACETTEPE UNIVERSITESI BEYTEPE KAMPUSU REKTORLUK BINASI contact info |
TR (CANKAYA ANKARA) | participant | 355˙974.36 |
| 15 |
GATC BIOTECH AG
Organization address
address: JAKOB STADLER PLATZ 7 contact info |
DE (KONSTANZ) | participant | 289˙400.00 |
| 16 |
FINOVATIS
Organization address
address: COURS LAFAYETTE 68 contact info |
FR (LYON) | participant | 276˙900.00 |
| 17 |
EUROPAEISCHES INSTITUT FUER FORSCHUNG UND ENTWICKLUNG VON TRANSPLANTATIONSSTRATEGIEN GMBH
Organization address
address: Vollmersbachstrasse 66 contact info |
DE (IDAR-OBERSTEIN) | participant | 49˙998.00 |
| 18 |
UNIVERSITAET ZUERICH
Organization address
address: Raemistrasse 71 contact info |
CH (ZURICH) | participant | 49˙998.00 |
| 19 |
EUROPESE VERENIGING VOOR GEN- EN CELTHERAPIE IVZW
Organization address
address: TIENSESTEENWEG 250 contact info |
BE (BIERBEEK) | participant | 27˙002.00 |
| 20 |
EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION
Organization address
address: P. DEBYELAAN-HAEMATOLOGIE contact info |
NL (MAASTRICHT) | participant | 25˙000.00 |
Mappa
Word cloud
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Obiettivo del progetto (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.'
Introduzione (Teaser)
An EU-funded study aims to restore the faith in of the use of gene therapy for treatment of primary immune deficiencies (PIDs). The CELL-PID project utilises genetically modified haematopoietic stem cells (HSCs) as a cell-based therapy for building a healthy immune system in patients with PIDs.