Summary
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.
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More information & hyperlinks
Web resources: | https://cordis.europa.eu/project/id/825670 |
Start date: | 01-01-2019 |
End date: | 30-06-2024 |
Total budget - Public funding: | 14 839 773,00 Euro - 14 839 773,00 Euro |
Cordis data
Original description
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.Status
SIGNEDCall topic
SC1-BHC-09-2018Update Date
26-10-2022
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EU-Programme-Call
Horizon 2020
H2020-EU.3. SOCIETAL CHALLENGES
H2020-EU.3.1. SOCIETAL CHALLENGES - Health, demographic change and well-being
H2020-EU.3.1.3. Treating and managing disease
H2020-EU.3.1.3.0. Cross-cutting call topics
H2020-SC1-2018-Single-Stage-RTD
SC1-BHC-09-2018 Innovation platforms for advanced therapies of the future