Description du projet
Vecteurs adénoviraux pour le transfert clinique de gènes
La thérapie génique est une approche très prometteuse pour le traitement de certaines maladies. Les vecteurs viraux ont été développés dépourvus de leur capacité à provoquer des maladies et sont au contraire utilisés pour introduire du matériel génétique dans les cellules à des fins thérapeutiques. Les adénovirus sont particulièrement intéressants parce qu’ils peuvent délivrer efficacement de l’ADN dans des cellules qui se divisent ou non. Financé par le Conseil européen de l’innovation, le projet iAds entend surmonter les limites des vecteurs adénoviraux, telles que les réponses immunitaires de l’hôte et le ciblage imparfait. Le consortium créera une plateforme in-silico pour la conception de vecteurs adénoviraux intelligents, en mettant l’accent sur le ciblage spécifique du cœur et du cerveau, dans des domaines où les besoins médicaux ne sont pas comblés.
Objectif
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.
Champ scientifique
Mots‑clés
Programme(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Régime de financement
HORIZON-EIC - HORIZON EIC GrantsCoordinateur
75794 Paris
France