Advanced Therapy Medicinal Products (ATMP)
The current development of ATMP is limited by their intrinsic complexity (from recombinant nucleic acids to artificial organs), their complex interactions (cell to cell and to the organism), and by the multiplicity of mode of action, making the definition of identity, potency and dose a major interdisciplinary challenge.
The participating EATRIS centres have already accumulated a vast experience in various aspect of the development of ATMP as demonstrated by the large number of different products already in clinical trials:
- Cancer Immunotherapies based on autologous dendritic, antigen-specific T-cells/NK cells;
- Gene therapies for immune-deficient patients;
- Control of Graft-versus-host disease (GVHD) in recipients of allogenic hematopoietic stem cell transplantation by infusion of mesenchymal cells or donor engineered T cells;
- Restoration of articular cartilage or bone structures using autologous chondrocytes /mesenchymal cells
- Ex vivo expanded antigen specific T-cells for the control of infectious diseases or as anti-tumour treatment
- Decellularised tissues as substrate for tissue engineering product.
The academic institutions which are the backbone of this Product Group have special facilities required for the GMP production and GLP toxicity testing of these products. They have developed innovative analytical tools, such as:
a.) Identity testing of the final product including the developing of methodology for tissue engineering constructs replicating organ functions
b.) Potency assays for the definition of dosage and for comparability studies including the definition of validated biomarkers for clinical studies
c.) Innovative animal models required for a meaningful efficacy and safety assessment in vivo
d.) Gene Therapy vectors for efficient and specific gene transfer to target cells.