Advanced therapy medicinal products (ATMPs) such as cell and gene therapies have the potential to revolutionise treatments for many serious genetic diseases as well as some cancers. However, developing ATMPs is extremely challenging; in particular it is currently very difficult to know which patients will respond well to an advanced therapy and which will experience serious side effects.
The aim of ARDAT is to deliver the knowledge, tools and standards needed to speed up the development of ATMPs.
Among other things, the project will deliver tools and knowledge that will allow researchers to study how treatments are metabolised (i.e. broken down by the body); predict which treatments are likely to trigger a harmful immune response; and identify how best to deliver the therapy (e.g. via a drip / injection).
They also aim to deliver tools to assess the safety and efficacy of ATMPs, and to determine how many doses a patient will need to obtain a lasting improvement in their health.
Finally, ARDAT plans to work closely with regulators on regulatory harmonisation to accelerate ATMP product development.
ARDAT will focus on rare diseases caused by a single gene mutation. However, many of the project’s findings will be applicable to other diseases.