Descripción del proyecto
Vectores adenovíricos para la transferencia clínica de genes
La genoterapia es un método muy prometedor para tratar ciertas enfermedades. Los vectores víricos se han creado despojados de patogenicidad y se utilizan para introducir material genético en las células con fines terapéuticos. Los adenovirus son especialmente atractivos porque pueden liberar ADN de forma eficaz tanto en las células que se dividen como en las que no. El objetivo del proyecto iAds, financiado por el Consejo Europeo de Innovación, es superar las limitaciones de los vectores adenovíricos, como la respuesta inmunitaria del hospedador y la especificidad imperfecta. El consorcio creará una plataforma «in silico» para diseñar vectores adenovíricos inteligentes centrados en el corazón y el encéfalo, con el fin de abordar áreas de necesidad médica no cubierta.
Objetivo
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.
Ámbito científico
Palabras clave
Programa(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Convocatoria de propuestas
HORIZON-EIC-2022-PATHFINDEROPEN-01
Consulte otros proyectos de esta convocatoriaRégimen de financiación
HORIZON-EIC - HORIZON EIC GrantsCoordinador
75794 Paris
Francia