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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.
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Coordinator

UNIVERSITA VITA-SALUTE SAN RAFFAELE

Address

Via Olgettina 58
20132 Milano

Italy

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 431 584,08

Administrative Contact

Maria Rosa Pedrazzi (Dr.)

Participants (19)

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INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 1 415 500

UNIVERSITY COLLEGE LONDON

United Kingdom

EU Contribution

€ 1 195 287

CHEMOTHERAPEUTISCHES FORSCHUNGSINSTITUT GEORG-SPEYER-HAUS STIFTUNG

Germany

EU Contribution

€ 516 199

WEIZMANN INSTITUTE OF SCIENCE

Israel

EU Contribution

€ 437 794

TECHNISCHE UNIVERSITAET DRESDEN

Germany

EU Contribution

€ 361 240

ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

Netherlands

EU Contribution

€ 791 531,64

OSPEDALE PEDIATRICO BAMBINO GESU

Italy

EU Contribution

€ 539 995

MEDIZINISCHE HOCHSCHULE HANNOVER

Germany

EU Contribution

€ 1 346 634

MOLECULAR MEDICINE SPA

Italy

EU Contribution

€ 678 270

MILTENYI BIOTEC GMBH

Germany

EU Contribution

€ 395 720

UNIVERSITAT BASEL

Switzerland

EU Contribution

€ 557 538

Eurofins Genomics Europe Sequencing GmbH

Germany

EU Contribution

€ 289 400

FINOVATIS

France

EU Contribution

€ 276 900

BIONTECH INNOVATIVE MANUFACTURING SERVICES GMBH

Germany

EU Contribution

€ 49 998

HACETTEPE UNIVERSITESI

Turkey

EU Contribution

€ 355 974,36

UNIVERSITAT ZURICH

Switzerland

EU Contribution

€ 49 998

EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION

Netherlands

EU Contribution

€ 25 000

EUROPESE VERENIGING VOOR GEN- EN CELTHERAPIE IVZW

Belgium

EU Contribution

€ 27 002

Fondazione Centro San Raffaele

Italy

EU Contribution

€ 1 146 169,92

Project information

Grant agreement ID: 261387

Status

Closed project

  • Start date

    1 November 2010

  • End date

    30 April 2016

Funded under:

FP7-HEALTH

  • Overall budget:

    € 15 799 071

  • EU contribution

    € 11 887 735

Coordinated by:

UNIVERSITA VITA-SALUTE SAN RAFFAELE

Italy

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