Project description
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.
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.
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Funding Scheme
RIA - Research and Innovation actionCoordinator
1050 Bruxelles / Brussel
Belgium