Intelligent design of adenovirus vectors (iAds)

Introduction

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.

Progress in Viral Vectors

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.

Challenges in Adenovirus Vector Development

In some cases, host responses and imperfect targeting have stunted adenovirus vector development for therapies that require long-term transgene expression.

Proposed Innovative Approach

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 various fields, we will create in silico designed intelligent adenovirus vectors (iAds).

Methodology

1. Abandon Classical Approaches: Our disruptive concept abandons the classical approach of developing vectors from naturally occurring adenoviruses.
2. Creation of iAds: A proprietorial adenovirus type will be serially stripped of unwanted elements to create a bank of iAds.
3. Targeting: These iAds will then be engineered for heart- and brain-specific targeting.

Collaboration and Translation

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.