Descrizione del progetto
Vettori di adenovirus per il trasferimento genico clinico
La terapia genica è un approccio altamente promettente per il trattamento di determinate malattie nell’ambito della quale vengono sviluppati vettori virali privi della propria capacità di provocare patologie, utilizzati invece per somministrare nelle cellule materiale genetico con finalità terapeutiche. Gli adenovirus sono particolarmente interessanti in quanto sono in grado di somministrare in modo efficace il DNA sia nelle cellule in fase di divisione che in quelle quiescenti. Finanziato dal Consiglio europeo per l’innovazione, il progetto iAds si prefigge di superare le limitazioni dei vettori adenovirali, come le risposte immunitarie dell’ospite e il bersagliamento imperfetto. Il consorzio creerà una piattaforma in silico per la progettazione di vettori di adenovirus intelligenti concentrando l’attenzione sul bersagliamento specifico del cervello e del cuore e consentendo di affrontare esigenze non soddisfatte in vari campi medici.
Obiettivo
Advanced therapies, and in particular gene therapies, hold great potential for treating diseases for which few options exist. Efficient gene transfer is inherently and intransigently linked to vector efficacy. Partially due to the lack of suitable delivery systems for particular applications, the success of too many gene therapies is limited.
Over the last two decades, immense progress has been made in the development of viral vectors. Importantly, this progress has also identified vector characteristics and biological factors that decrease efficacy. While limited efficacy is relevant for all vector platforms, it is more pressing in the case of adenoviruses because they have so much potential. In some cases, host responses and imperfect targeting have stunted adenovirus vector development for therapies that require long-term transgene expression.
Our multi-faceted consortium proposes an innovative approach to overcome these limitations and to construct a pathway for developing improved vectors for clinical gene transfer. By synergising French, Dutch, British, Spanish and Swedish expertise in structural biology, receptor engagement, neurobiology, cardiobiology, and bioprocessing, we will create in silico designed intelligent adenovirus vectors (iAds). Our disruptive concept abandons the classical approach of developing vectors from naturally occurring adenoviruses. Instead, a proprietorial adenovirus type will be serially stripped of unwanted elements to create a bank of iAds, which will then be engineered for heart- and brain-specific targeting. Our consortium blends academic ingenuity and SME/pharma manufacturing that will allow seamless clinical translation. With the support of the EIC Programme, our ground-breaking approach should revolutionise gene transfer and generate solutions in areas of unmet medical need via a platform that exploits the full potential of viral vectors.
Campo scientifico
Parole chiave
Programma(i)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Invito a presentare proposte
HORIZON-EIC-2022-PATHFINDEROPEN-01
Vedi altri progetti per questo bandoMeccanismo di finanziamento
HORIZON-EIC - HORIZON EIC GrantsCoordinatore
75794 Paris
Francia