Descrizione del progetto
Una terapia genica innovativa per il trattamento dell’insufficienza cardiaca e dell’ischemia miocardica
Secondo le stime dell’OMS, ogni anno muoiono 17,7 milioni di persone in tutto il mondo a causa delle malattie cardiovascolari, che costituiscono oltre il 30 % di tutti i decessi a livello globale. Progressi recenti della ricerca biomedica hanno migliorato la nostra comprensione in merito ai meccanismi molecolari e cellulari della malattia e hanno introdotto vettori di terapia genica efficienti e più sicuri per lo sviluppo di medicinali per terapie avanzate di nuova generazione destinati a combattere le malattie cardiovascolari. Il progetto CardioReGenix, finanziato dall’UE, è incentrato sulle innovazioni tecnologiche per il trattamento dell’arresto cardiaco e dell’ischemia miocardica. L’obiettivo è quello di superare gli ostacoli della terapia genica per le malattie cardiovascolari, traendo vantaggio dai solidi dati preliminari e dal know-how multidisciplinare del consorzio nello sviluppo di vettori, nella cardiologia preclinica e clinica, nello sviluppo aziendale e nella traslazione clinica.
Obiettivo
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.
Campo scientifico
Parole chiave
Programma(i)
Invito a presentare proposte
Vedi altri progetti per questo bandoBando secondario
H2020-SC1-2018-Single-Stage-RTD
Meccanismo di finanziamento
RIA - Research and Innovation actionCoordinatore
1050 Bruxelles / Brussel
Belgio