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EU-Förderung (5.990.460 €): Stem-cell based gene therapy for recombination deficient SCID (RECOMB) Hor01.01.2018 EU-Rahmenprogramm für Forschung und Innovation "Horizont"
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Stem-cell based gene therapy for recombination deficient SCID (RECOMB)
Gene therapy for rare inherited immune disorders has become a clinical reality. Especially for SCID, two major types of SCID (ADA-SCID, X-SCID) have been successfully treated by autologous stem cell based gene therapy. However, for the most common group of SCID, the SCID underlying recombination defects, this has not yet occurred due to the higher complexities of the affected genes involved. The aim of the current proposal is to fill the unmet medical need for the most common major category of SCID, recombination activating gene-1 (RAG-1) deficient SCID, by performing Stage I/II clinical trials using autologous hematopoietic stem cell based gene therapy. To this end we will develop novel safety assays, pre-GMP and GMP lentiviral batches and design and conduct multicenter, multinational clinical trials with input from regulatory authorities such as EMA and patient advocacy groups. The trial will be conducted with phenotypic, molecular (integration sites, therapeutic gene expression) and functional readouts and should lead to effective treatment for > 70% of all SCID patients in Europe. RECOMB forms the logical extension of highly successful previous EU consortia that have made the EU global leader in gene therapy for orphan immune diseases.
Geförderte Unternehmen:
| Firmenname | Förderungssumme |
| ACADEMISCH ZIEKENHUIS LEIDEN | 2.170.625 € |
| Assistance Publique Hopitaux de Paris | 25.000 € |
| BATAVIA BIOSCIENCES B.V. | 982.125 € |
| Fundacio Hospital Universitari Vall D'Hebron - Institut de Recerca | 62.500 € |
| Great Ormond Street Hospital for Children NHS Foundation Trust | 62.500 € |
| Institut National de la Sante et de la Recherche Medicale | 437.500 € |
| International Patient Organisation for Primary Immunodeficiencies | 0,00 € |
| Ipopi | 182.500 € |
| LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN | 57.500 € |
| MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER | 57.500 € |
| MEDIZINISCHE HOCHSCHULE HANNOVER | 558.960 € |
| Ospedale Pediatrico Bambino Gesu | 57.500 € |
| Ospedale SAN Raffaele Srl | 532.500 € |
| PROTAGENE CGT GmbH | 596.250 € |
| UNIVERSITAET ULM | 62.500 € |
| University College London | 20.000 € |
| University of Newcastle Upon Tyne | 62.500 € |
| Uniwersytet Medyczny im Piastow Slaskich WE Wroclawiu | 62.500 € |
Quelle: https://cordis.europa.eu/project/id/755170
Diese Bekanntmachung wurde von Englisch nach Deutsch übersetzt. Die Bekanntmachung bezieht sich auf einen vergangenen Zeitpunkt, und spiegelt nicht notwendigerweise den heutigen Stand wider.
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