Objective Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for two forms of SCID (SCID-X1 and ADA SCID) has shown significant safety and efficacy in correcting the immunodeficiency and allowing children to live normal lives. Proof of concept of gene therapy for 3 other SCID forms has also been shown by members of the proposed SCIDNET consortium and is ready for translation into clinical trials. We are therefore in a position whereby, over the next 4 years, we can offer gene therapy as a curative option for over 80% of all forms of SCID in Europe. Importantly for 1 of these conditions (ADA SCID) we will undertake clinical trials that will lead to marketing authorisation of the gene therapy product as a licensed medicine. In addition, we will investigate the future technologies that will improve the safety and efficacy of gene therapy for SCID.Our proposal addresses an unmet clinical need in SCID, which is classified as a rare disease according to EU criteria (EC regulation No. 141/2000). The proposal also addresses the need to develop an innovative treatment such as gene therapy from early clinical trials though to a licensed medicinal product through involvement with regulatory agencies and is in keeping with the ambitions of the IRDiRC. The lead ADA SCID programme has Orphan Drug Designation and clinical trial design is assisted by engagement with the European medicines Agency. The ADA SCID trial will act as a paradigm for the development of the technologies and processes that will allow gene therapy for not only SCID, but also other bone marrow disorders, to become authorised genetic medicines in the future. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesbasic medicineimmunologymedical and health sciencesmedical biotechnologycells technologiesstem cellsmedical 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) PHC-14-2015 - New therapies for rare diseases Call for proposal H2020-PHC-2014-2015 See other projects for this call Sub call H2020-PHC-2015-two-stage Funding Scheme RIA - Research and Innovation action Coordinator UNIVERSITY COLLEGE LONDON Net EU contribution € 1 549 174,00 Address Gower street WC1E 6BT London United Kingdom 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 € 0,75 Participants (11) Sort alphabetically Sort by Net EU contribution Expand all Collapse all ACADEMISCH ZIEKENHUIS LEIDEN Netherlands Net EU contribution € 799 877,00 Address Albinusdreef 2 2333 ZA Leiden 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 € 0,50 INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE France Net EU contribution € 850 002,00 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 € 0,50 PROTAGENE CGT GMBH Germany Net EU contribution € 699 750,00 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 OSPEDALE SAN RAFFAELE SRL Italy Net EU contribution € 474 250,00 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 UNIVERSITAETSKLINIKUM FREIBURG Germany Net EU contribution € 499 300,00 Address Hugstetter strasse 49 79106 Freiburg See on map Region Baden-Württemberg Freiburg Freiburg im Breisgau, 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 ASSOCIATION GENETHON France Net EU contribution € 800 000,00 Address Rue de l internationale 1 bis 91002 Evry See on map Region Ile-de-France Ile-de-France Essonne 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 MEDIZINISCHE HOCHSCHULE HANNOVER Germany Net EU contribution € 509 562,00 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 € 0,50 GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST United Kingdom Net EU contribution € 423 198,00 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 € 0,75 MILTENYI BIOTEC GMBH Germany Net EU contribution € 296 200,00 Address Friedrich ebert strasse 68 51429 Bergish gladbach See on map Region Nordrhein-Westfalen Köln Rheinisch-Bergischer Kreis 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 CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS Switzerland Net EU contribution € 0,00 Address Rue du bugnon 21 1011 Lausanne See on map Region Schweiz/Suisse/Svizzera Région lémanique Vaud Activity type Public bodies (excluding Research Organisations and Secondary or Higher 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 € 548 000,00 INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES United Kingdom Net EU contribution € 25 000,00 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
