Objective
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.
Fields of science
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesbasic medicineimmunology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- medical and health sciencesclinical medicineemergency medicinegraft versus host disease
- medical and health sciencesclinical medicinetransplantation
Keywords
Programme(s)
Funding Scheme
RIA - Research and Innovation actionCoordinator
2333 ZA Leiden
Netherlands
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Participants (16)
75654 Paris
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WC1E 6BT London
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WC1N 3JH London
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20132 Milano
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30625 Hannover
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89081 Ulm
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50 367 Wroclaw
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NE1 7RU Newcastle Upon Tyne
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PL11 3LE Torpoint
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2333 CL Leiden
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
08035 Barcelona
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00165 Roma
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75012 Paris
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52621 Ramat Gan
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80539 MUNCHEN
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69120 Heidelberg
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.