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Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (CELL-PID) 


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. PID diseases 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. The aim of this project is to develop a safe therapy for PID using genetically modified HSC and their descendants as immunotherapeutic cells to build a healthy immune system. The consortium will establish a technology platform to implement, harmonize and run controlled, standardized multi-centre preclinical studies using state-of-the-art advanced therapy. 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.

GATC Biotech’s task:

GATC Biotech performs high-throughput sequencing and analysis of the genomic integration sites of gene therapy vectors. Insertion site analysis in the human and mouse genomes will be implemented to provide information about the genomic integration locus and aid risk assesment of gene therapy vectors.


Participating partners:

Chemotherapeutisches Forschungsinstitut Georg-Speyer-Haus Stiftung, Germany
Erasmus Universitair Medisch Centrum Rotterdam, Netherlands
Europaeisches Institut Fuer Forschung Und Entwicklung Von Transplantationsstrategien GmbH, Germany
European Group For Blood And Marrow Transplantation, Netherlands
Europese Vereniging Voor Gen- En Celtherapie Ivzw, Belgium
Finovatis, France
Hacettepe Universitesi, Turkey
Institut National De La Sante Et De La Recherche Medicale (Inserm), France
Medizinische Hochschule Hannover, Germany
Miltenyi Biotec GmbH, Germany
Molecular Medicine Spa, Italy
Ospedale Pediatrico Bambino Gesu, Italy
Technische Universitaet Dresden, Germany
Universitaet Basel, Switzerland
Universitaet Zuerich, Switzerland
University College London, United Kingdom
Weizmann Institute Of Science, Israel

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