Cel
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.
Dziedzina nauki
- 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
Słowa kluczowe
Program(-y)
Zaproszenie do składania wniosków
Zobacz inne projekty w ramach tego zaproszeniaSzczegółowe działanie
H2020-SC1-2017-Two-Stage-RTD
System finansowania
RIA - Research and Innovation actionKoordynator
2333 ZA Leiden
Niderlandy
Zobacz na mapie
Uczestnicy (16)
75654 Paris
Zobacz na mapie
WC1E 6BT London
Zobacz na mapie
WC1N 3JH London
Zobacz na mapie
20132 Milano
Zobacz na mapie
30625 Hannover
Zobacz na mapie
89081 Ulm
Zobacz na mapie
50 367 Wroclaw
Zobacz na mapie
NE1 7RU Newcastle Upon Tyne
Zobacz na mapie
PL11 3LE Torpoint
Zobacz na mapie
2333 CL Leiden
Zobacz na mapie
Organizacja określiła się jako MŚP (firma z sektora małych i średnich przedsiębiorstw) w czasie podpisania umowy o grant.
08035 Barcelona
Zobacz na mapie
00165 Roma
Zobacz na mapie
75012 Paris
Zobacz na mapie
52621 Ramat Gan
Zobacz na mapie
80539 MUNCHEN
Zobacz na mapie
69120 Heidelberg
Zobacz na mapie
Organizacja określiła się jako MŚP (firma z sektora małych i średnich przedsiębiorstw) w czasie podpisania umowy o grant.