Since the project started in 2020, our primary achievement has been the release of 13 publications with several more under review and a level of collaboration that will stand us in great stead for the future.
More specifically, the ARDAT project has:
- Taken significant steps towards better understanding of immune responses by developing 3D modelling for liver and CNS and validating immunogenicity assays.
- Progressed a new gene therapy for hereditary spastic paraplegia 47 to Investigational New Drug (IND) status with the Food and Drug Administration (FDA). We expect a clinical trial to start soon.
- Established the ARDAT biobank with NHS Research Ethics Commitee (REC) approval and this collection of bio samples gathered during the ARDAT project will facilitate future analysis of patient responses to ATMPs, quantify risk and better predict outcomes.
- Engaged effectively with key regulators, published a paper on biodistribution and immunogenicity regulation and conducted workshops with pharma companies and regulators.
- Listened to those with lived experience in order to embed the patient voice at the heart of everything we do.