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Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency

Ziel

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.

Aufforderung zur Vorschlagseinreichung

FP7-HEALTH-2010-single-stage
Andere Projekte für diesen Aufruf anzeigen

Koordinator

UNIVERSITA VITA-SALUTE SAN RAFFAELE
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Via Olgettina 58
20132 Milano
Italien
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Higher or Secondary Education Establishments
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€ 1 431 584,08
Kontakt Verwaltung
Maria Rosa Pedrazzi (Dr.)

Beteiligte (19)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Frankreich
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€ 1 415 500
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Rue De Tolbiac 101
75654 Paris
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Research Organisations
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Annick Bertault (Ms.)
UNIVERSITY COLLEGE LONDON
United Kingdom
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Gower Street
WC1E 6BT London
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Higher or Secondary Education Establishments
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Greta Borg-Carbott (Ms.)
CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG
Deutschland
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€ 516 199
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Paul Ehrlich Strasse 42-44
60596 Frankfurt
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Manuel Grez (Dr.)
WEIZMANN INSTITUTE OF SCIENCE
Israel
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Herzl Street 234
7610001 Rehovot
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Talia Tzahor (Ms.)
TECHNISCHE UNIVERSITAET DRESDEN
Deutschland
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Helmholtzstrasse 10
01069 Dresden
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Lars Henning (Dr.)
ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

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Niederlande
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Dr Molewaterplein 40
3015 GD Rotterdam
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Gerard Wagemaker (Prof.)
OSPEDALE PEDIATRICO BAMBINO GESU
Italien
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Piazza Sant Onofrio 4
00165 Roma
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Sonya Jane Martin (Dr.)
MEDIZINISCHE HOCHSCHULE HANNOVER
Deutschland
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Carl-neuberg-strasse 1
30625 Hannover
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Frank Dittrich (Mr.)
AGC BIOLOGICS SPA
Italien
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Via Meucci, 3
20091 Bresso Mi
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Enrico Cappelli (Dr.)
MILTENYI BIOTEC GMBH
Deutschland
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51429 Bergish Gladbach
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UNIVERSITAT BASEL
Schweiz
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Petersplatz 1
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Georg-A Hollaender (Prof.)
GATC BIOTECH AG
Deutschland
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Jakob Stadler Platz 7
78467 Konstanz
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Natascha Ostrowski (Ms.)
FINOVATIS
Frankreich
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Cours Lafayette 68
69003 Lyon
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David Koubi (Dr.)
BIONTECH INNOVATIVE MANUFACTURING SERVICES GMBH
Deutschland
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Vollmersbachstrasse 66
55743 Idar Oberstein
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Klaus Kuehlcke (Dr.)
HACETTEPE UNIVERSITESI
Türkei
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Hacettepe Universitesi Beytepe Kampusu Rektorluk Binasi
06800 Cankaya Ankara
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Sevtap Benli (Ms.)
UNIVERSITAT ZURICH
Schweiz
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Ramistrasse 71
8006 Zurich
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EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION
Niederlande
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EUROPESE VERENIGING VOOR GEN- EN CELTHERAPIE IVZW
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Renee Watson (Ms.)
FONDAZIONE CENTRO SAN RAFFAELE
Italien
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Maria Rosa Pedrazzi (Dr.)