Project description DEENESFRITPL Innovative gene therapy for the treatment of heart failure and myocardial ischemia According to WHO estimates, 17.7 million people die each year from cardiovascular disease (CVD), which constitutes more than 30 % of all deaths worldwide. Recent advances in biomedical research improved our understanding of the molecular and cellular mechanisms of the disease and introduced efficient and safer gene therapy vectors for the development of next-generation advanced therapy medicinal products (ATMPs) for CVD. The EU-funded CardioReGenix project focuses on technological innovations for the treatment of heart failure and myocardial ischemia. The goal is to overcome obstacles in CVD gene therapy, capitalising on robust preliminary data and multi-disciplinary consortium know-how in vector development, preclinical and clinical cardiology, business development and clinical translation. Show the project objective Hide the project objective Objective WHO estimates 17.7 million people die each year from cardiovascular disease (CVD), an estimated 31% of all deaths worldwide. While progress with conventional treatments is making incremental gains, there remains a need to develop innovative therapeutic approaches. Gene therapy has gained significant momentum, mainly for treatment of rare monogenetic diseases. Marketing authorization for gene therapy products has not impacted diseases such as CVD. Recent, deeper understanding of the molecular/cellular mechanisms of CVD and technology associated with more efficient and safer gene therapy vectors has allowed new opportunities for development of next-generation ATMPs for CVD. CardioReGenix focuses on technological innovations for the treatment of CVD, in particular heart failure and myocardial ischemia. We aim to overcome bottlenecks in gene therapy for CVD by (i) maximizing cardiac-specific gene expression; (ii) maximizing cardiac-specific gene delivery; (iii) reducing adverse immune responses; (iv) optimizing manufacturing for research and clinical-grade ATMPs ; (v) validate new promising targets using in vitro and preclinical models; (vi) undertaking first-in-man studies; (vii) developing and implementing an exploitation and valorization strategy with strong EU SME involvement. CardioReGenix is a high risk/high gain project supported by robust preliminary data and multi-disciplinary consortium harnessing know-how in vector development, GMP manufacturing, preclinical and clinical cardiology, regulation of ATMP and liaising with EMA, business development and clinical translation. CardioReGenix will (i) strengthen Europe's competitive position in gene therapy development for CVD; (ii) improve the prospect of successfully treating patients suffering from CVD by gene therapy and (iii) develop and refine the latest gene therapy platforms that go significantly beyond the state of the art. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesbasic medicineimmunologymedical and health sciencesclinical medicinecardiologycardiovascular diseases Keywords Cardiovascular disease ncRNA gene therapy 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-BHC-09-2018 - Innovation platforms for advanced therapies of the future Call for proposal H2020-SC1-BHC-2018-2020 See other projects for this call Sub call H2020-SC1-2018-Single-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator VRIJE UNIVERSITEIT BRUSSEL Net EU contribution € 2 314 025,49 Address Pleinlaan 2 1050 Bruxelles / brussel Belgium See on map Region Région de Bruxelles-Capitale/Brussels Hoofdstedelijk Gewest Région de Bruxelles-Capitale/ Brussels Hoofdstedelijk Gewest Arr. de Bruxelles-Capitale/Arr. Brussel-Hoofdstad 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 (12) Sort alphabetically Sort by Net EU contribution Expand all Collapse all ITA-SUOMEN YLIOPISTO Finland Net EU contribution € 2 566 115,25 Address Yliopistonranta 1 e 70211 Kuopio See on map Region Manner-Suomi Pohjois- ja Itä-Suomi Pohjois-Savo 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 A2F ASSOCIATES LIMITED United Kingdom Net EU contribution € 718 295,00 Address 7 laureate paddocks CB8 0AP Newmarket 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 East of England East Anglia Suffolk 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 MEDIZINISCHE HOCHSCHULE HANNOVER Germany Net EU contribution € 1 193 622,50 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 KING'S COLLEGE LONDON United Kingdom Net EU contribution € 1 013 675,00 Address Strand WC2R 2LS London See on map Region London Inner London — West Westminster 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 THE UNIVERSITY OF EDINBURGH United Kingdom Net EU contribution € 1 424 113,75 Address Old college, south bridge EH8 9YL Edinburgh See on map Region Scotland Eastern Scotland Edinburgh 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 STICHTING VUMC Netherlands Net EU contribution € 1 106 750,75 Address De boelelaan 1117 1081 HV Amsterdam See on map Region West-Nederland Noord-Holland Groot-Amsterdam 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 BATAVIA BIOSCIENCES BV Netherlands Net EU contribution € 955 907,50 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 € 0,00 FINVECTOR OY Participation ended Finland Net EU contribution € 239 707,51 Address Microkatu 1 s 70210 Kuopio See on map Region Manner-Suomi Pohjois- ja Itä-Suomi Pohjois-Savo 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 CARDIOR PHARMACEUTICALS GMBH Germany Net EU contribution € 908 249,50 Address Feodor lynen str 15 30625 Hannover 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 Niedersachsen Hannover Region Hannover 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 ASPHALION SL Spain Net EU contribution € 310 035,00 Address Calle tarragona, numeros 151-157, planta 10, bloqu 08014 Barcelona 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 Este Cataluña Barcelona 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 € 0,00 POHJOIS-SAVON SAIRAANHOITOPIIRIN KUNTAYHTYMA Finland Net EU contribution € 677 750,00 Address Pl 100 70029 Kys See on map Region Manner-Suomi Pohjois- ja Itä-Suomi Pohjois-Savo 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 CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL Germany Net EU contribution € 1 411 526,25 Address Olshausenstrasse 40 24118 Kiel See on map Region Schleswig-Holstein Schleswig-Holstein Kiel, 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