Project overall strategic goals

The overall objective of ARDAT is to develop and provide data and tools to address knowledge gaps in the field of gene therapy. We have a scientific focus on the areas of immunology and vector metabolism and also, we aim to identify areas for regulatory harmonization. Ultimately we do all this to accelerate the research and development of gene therapies for people living with rare genetic conditions who often have no or very limited treatment options.

Short description of the main outputs of the project/coalition

  • Develop improved and sustainable model systems, bioanalytical methods and novel reagents for predicting immunogenicity in humans.
  • Establish a central and sustainable biobanking infrastructure for patients receiving approved or experimental advanced therapy medicinal products for supporting immunogenicity assessments.
  • Explore the clinical factors around pre-existing immunity that can limitsing patient access to advanced therapies.
  • Enhance our understanding of AAV gene therapy metabolism inside a range of cell types and explore strategies to mitigate this loss.
  • Engage with regulatory authorities and stakeholders, including patient advocacy groups, charities and sponsors to identify areas for regulatory harmonisation.

How does the project/coalition address the specificities of the white spots?

The ARDAT project is disease agnostic, yet, by the genetic nature of many of the white spots, our research and the deployment of the outcomes will be applicable to white spots.

How is the project/coalition a public-private partnership? 

The consortium is a collaboration between academic institutions, micro, small and medium-sized enterprises, and pharmaceutical companies.

How being a PPP helps to address the white spots?

PPPs are a great way to speed up medical innovation because of the complementary expertise in academia and biopharma industry, These partnerships bring together expertise across a number of different areas including basic science, discovery, drug development and regulatory affairs. Ultimately this facilitates the translation of scientific knowledge into tangible advances in patient care.