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 therapymedical and health sciencesbasic medicineimmunologymedical and health sciencesmedical biotechnologycells technologiesstem cellsmedical and health sciencesclinical medicineemergency medicinegraft versus host diseasemedical and health sciencesclinical medicinetransplantation Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-PM-08-2017 - New therapies for rare diseases Call for proposal H2020-SC1-2016-2017 See other projects for this call Sub call H2020-SC1-2017-Two-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator ACADEMISCH ZIEKENHUIS LEIDEN Net EU contribution € 2 170 625,00 Address Albinusdreef 2 2333 ZA Leiden Netherlands See on map Region West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Participants (16) Sort alphabetically Sort by Net EU contribution Expand all Collapse all INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE France Net EU contribution € 437 500,00 Address Rue de tolbiac 101 75654 Paris See on map Region Ile-de-France Ile-de-France Paris Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITY COLLEGE LONDON United Kingdom Net EU contribution € 20 000,00 Address Gower street WC1E 6BT London See on map Region London Inner London — West Camden and City of London Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST United Kingdom Net EU contribution € 62 500,00 Address Great ormond street WC1N 3JH London See on map Region London Inner London — West Camden and City of London Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 OSPEDALE SAN RAFFAELE SRL Italy Net EU contribution € 532 500,00 Address Via olgettina 60 20132 Milano See on map Region Nord-Ovest Lombardia Milano Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 MEDIZINISCHE HOCHSCHULE HANNOVER Germany Net EU contribution € 558 960,00 Address Carl-neuberg-strasse 1 30625 Hannover See on map Region Niedersachsen Hannover Region Hannover Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITAET ULM Germany Net EU contribution € 62 500,00 Address Helmholtzstrasse 16 89081 Ulm See on map Region Baden-Württemberg Tübingen Ulm, Stadtkreis Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU Poland Net EU contribution € 62 500,00 Address Ul pasteura 1 50 367 Wroclaw See on map Region Makroregion południowo-zachodni Dolnośląskie Miasto Wrocław Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITY OF NEWCASTLE UPON TYNE United Kingdom Net EU contribution € 62 500,00 Address Kings gate NE1 7RU Newcastle upon tyne See on map Region North East (England) Northumberland and Tyne and Wear Tyneside Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES United Kingdom Net EU contribution € 182 500,00 Address Main road firside PL11 3LE Torpoint See on map Region South West (England) Cornwall and Isles of Scilly Cornwall and Isles of Scilly Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 BATAVIA BIOSCIENCES BV Netherlands Net EU contribution € 982 125,00 Address Zernikedreef 16 2333 CL Leiden See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,75 FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA Spain Net EU contribution € 62 500,00 Address Passeig vall d hebron 119-129 edificio de recerca 08035 Barcelona See on map Region Este Cataluña Barcelona Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 OSPEDALE PEDIATRICO BAMBINO GESU Italy Net EU contribution € 57 500,00 Address Piazza sant onofrio 4 00165 Roma See on map Region Centro (IT) Lazio Roma Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 ASSISTANCE PUBLIQUE HOPITAUX DE PARIS France Net EU contribution € 25 000,00 Address 55 boulevard diderot 75012 Paris See on map Region Ile-de-France Ile-de-France Val-de-Marne Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER Israel Net EU contribution € 57 500,00 Address Tel hashomer sheba medical center 52621 Ramat gan See on map Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Germany Net EU contribution € 57 500,00 Address Geschwister scholl platz 1 80539 Muenchen See on map Region Bayern Oberbayern München, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 PROTAGENE CGT GMBH Germany Net EU contribution € 596 250,00 Address Im neuenheimer feld 582 69120 Heidelberg See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Baden-Württemberg Karlsruhe Heidelberg, Stadtkreis Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
