Projects

GTRP-DIAT

R&D

En cours

The goal of this gene therapy research partnership is to discover and apply innovative analytical technologies to advance the characterisation of recombinant adeno-associated viral vectors (rAAVs), the safest and most effective vehicles for delivering genes into cells and driving their long-term expression. Investigation of the vectors’ quality and efficacy is critical to treating genetic diseases affecting millions of people worldwide: we need to progress our fundamental understanding of the impact of complex vector structures on its properties, to establish label-free non-invasive analytical approaches to determine bio- and virological activity of the vectors, to invent novel methods to reveal their structural complexity, genome integrity and impurity, and finally to understand the impact of impurities on the final drug structure and its function.

ThermoFisher Scientific (represented by Henogen S.R.L.) and UCB have the ambition to discover novel analytical technologies to address these challenges and to translate rAAV-based gene therapy research into safe and effective therapeutic solutions for millions of patients worldwide. In addition, a digital data platform, supported by statistical and advanced data analytics, will be put in place to discover the link between pre-clinical and clinical outcomes and drug quality attributes, with that feedback loop enabling more informed decision-making and overall higher success rates for future projects.

The project will further strengthen the ATMP ecosystem in Wallonia by deepening the knowledge and expertise of the region’s scientific talent and reinforce Wallonia’s and Belgium’s position as the ‘Health and Biotech Valley of Tomorrow’.

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