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
Keywords
Programme(s)
Funding Scheme
RIA - Research and Innovation actionCoordinator
2333 ZA Leiden
Netherlands
See on map
Participants (17)
75654 Paris
See on map
WC1E 6BT London
See on map
WC1N 3JH London
See on map
20132 Milano
See on map
30625 Hannover
See on map
89081 Ulm
See on map
50 367 Wroclaw
See on map
NE1 7RU Newcastle Upon Tyne
See on map
Participation ended
PL11 3LE Torpoint
See on map
2333 CL Leiden
See on map
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
08035 Barcelona
See on map
00165 Roma
See on map
75012 Paris
See on map
52621 Ramat Gan
See on map
80539 MUNCHEN
See on map
69120 Heidelberg
See on map
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
1050 Bruxelles / Brussel
See on map