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Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency

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.

Call for proposal

FP7-HEALTH-2010-single-stage
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Coordinator

UNIVERSITA VITA-SALUTE SAN RAFFAELE
Address
Via Olgettina 58
20132 Milano
Italy

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Maria Rosa Pedrazzi (Dr.)
EU contribution
€ 1 431 584,08

Participants (19)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 1 415 500
Address
Rue De Tolbiac 101
75654 Paris

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Activity type
Research Organisations
Administrative Contact
Annick Bertault (Ms.)
UNIVERSITY COLLEGE LONDON
United Kingdom
EU contribution
€ 1 195 287
Address
Gower Street
WC1E 6BT London

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Greta Borg-Carbott (Ms.)
CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG
Germany
EU contribution
€ 516 199
Address
Paul Ehrlich Strasse 42-44
60596 Frankfurt

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Activity type
Research Organisations
Administrative Contact
Manuel Grez (Dr.)
WEIZMANN INSTITUTE OF SCIENCE
Israel
EU contribution
€ 437 794
Address
Herzl Street 234
7610001 Rehovot

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Talia Tzahor (Ms.)
TECHNISCHE UNIVERSITAET DRESDEN
Germany
EU contribution
€ 361 240
Address
Helmholtzstrasse 10
01069 Dresden

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Lars Henning (Dr.)
ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

Participation ended

Netherlands
EU contribution
€ 791 531,64
Address
Dr Molewaterplein 40
3015 GD Rotterdam

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Gerard Wagemaker (Prof.)
OSPEDALE PEDIATRICO BAMBINO GESU
Italy
EU contribution
€ 539 995
Address
Piazza Sant Onofrio 4
00165 Roma

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Activity type
Research Organisations
Administrative Contact
Sonya Jane Martin (Dr.)
MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
EU contribution
€ 1 346 634
Address
Carl-neuberg-strasse 1
30625 Hannover

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Frank Dittrich (Mr.)
AGC BIOLOGICS SPA
Italy
EU contribution
€ 678 270
Address
Via Meucci, 3
20091 Bresso Mi

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Enrico Cappelli (Dr.)
MILTENYI BIOTEC GMBH
Germany
EU contribution
€ 395 720
Address
Friedrich Ebert Strasse 68
51429 Bergish Gladbach

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Sabrina Schmitz (Ms.)
UNIVERSITAT BASEL
Switzerland
EU contribution
€ 557 538
Address
Petersplatz 1
4051 Basel

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Georg-A Hollaender (Prof.)
GATC BIOTECH AG
Germany
EU contribution
€ 289 400
Address
Jakob Stadler Platz 7
78467 Konstanz

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Natascha Ostrowski (Ms.)
FINOVATIS
France
EU contribution
€ 276 900
Address
Cours Lafayette 68
69003 Lyon

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
David Koubi (Dr.)
BIONTECH INNOVATIVE MANUFACTURING SERVICES GMBH
Germany
EU contribution
€ 49 998
Address
Vollmersbachstrasse 66
55743 Idar Oberstein

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Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Klaus Kuehlcke (Dr.)
HACETTEPE UNIVERSITESI
Turkey
EU contribution
€ 355 974,36
Address
Hacettepe Universitesi Beytepe Kampusu Rektorluk Binasi
06800 Cankaya Ankara

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Sevtap Benli (Ms.)
UNIVERSITAT ZURICH
Switzerland
EU contribution
€ 49 998
Address
Ramistrasse 71
8006 Zurich

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ivo Cotti (Mr.)
EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION
Netherlands
EU contribution
€ 25 000
Address
Rijnsburgerweg 10
2333 AA Leiden

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Activity type
Other
Administrative Contact
Marco Van Der Most (Mr.)
EUROPESE VERENIGING VOOR GEN- EN CELTHERAPIE IVZW
Belgium
EU contribution
€ 27 002
Address
Tiensesteenweg 250
3360 Bierbeek

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Activity type
Other
Administrative Contact
Renee Watson (Ms.)
FONDAZIONE CENTRO SAN RAFFAELE
Italy
EU contribution
€ 1 146 169,92
Address
Via Olgettina 60
20132 Milano

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Activity type
Research Organisations
Administrative Contact
Maria Rosa Pedrazzi (Dr.)