These are medicinal products based on modified human genes, tissue, or cells. They are known as ATMP (Advanced Therapy Medicinal Products).
The term "advanced therapies" is a regulatory definition that includes three main types of medicinal product:
Gene therapy medicinal products: they are based on laboratory-created recombinant nucleic acids and are used to repair, control, add or eliminate gene sequences in order to treat diseases.
Some applications include blindness caused by genetic problems in the retina or spinal muscular atrophy.
Gene therapy medicinal products also include CAR-T (Chimeric Antigen Receptor T-cell), which use modified T-lymphocytes of patients.
The two CAR-T authorised at present attack the cells expressing the CD19 membrane antigen that is distinctive of B-lymphocytes, and are used in the treatment of acute lymphoblastic leukaemia in children and some B-cell lymphomas in adults.
Other CAR are currently being developed that target other antigens. This opens up new therapeutic possibilities for treating other types of tumour, such as solid tumours, or other disorders such as infectious and auto-immune diseases.
Somatic cell therapy medicinal products: they contain cells or tissue that have been manipulated to modify their biological characteristics for use in therapy.
One example of such is the treatment of anal fistulae caused by Crohn's Disease using stem cells obtained from fat.
Tissue-engineered medicinal products: they contain cells or tissue manipulated by engineering for use in repairing, regenerating or replacing human tissue.
PeriCord, for example, is a bioimplant capable of repairing cardiac tissue damaged by a heart attack. Banc de Sang i Teixits was involved in the project (link).
Where the advanced therapy medicinal product includes one or more health products, it is known as a combined Advanced Therapy Medicinal Product.