Advanced therapy medicinal products are based on manufacturing processes focussed on various gene transfer-produced bio-molecules, and/or biologically advanced therapeutic modified cells as active substances or part of active substances. They include:
Gene therapy medicinal product: a product obtained through a set of manufacturing processes aimed at the transfer, to be performed either in vivo or ex vivo, of a prophylactic, diagnostic or therapeutic gene (i.e. a piece of nucleic acid), to human/animal cells and its subsequent expression in vivo. The gene transfer involves an expression system contained in a delivery system known as a vector, which can be of viral, as well as non-viral origin. The vector can also be included in a human or animal cell.
Somatic cell therapy medicinal product: it means the use in humans of autologous (emanating from the patient himself), allogeneic (coming from another human being) or xenogeneic (coming from animals) somatic living cells, the biological characteristics of which have been substantially altered as a result of their manipulation to obtain a therapeutic, diagnostic or preventive effect through metabolic, pharmacological and immunological means.
Somatic cell therapy medicinal products include:
- Cells manipulated to modify their immunological, metabolic or other functional properties in qualitative or quantitative aspects;
- Cells sorted, selected and manipulated and subsequently undergoing a manufacturing process in order to obtain the finished medicinal product;
- Cells manipulated and combined with non-cellular components (e.g. biological or inert matrixes or medical devices) and exerting the principle intended action in the finished product;
- Autologous cell derivatives expressed in vitro under specific culture conditions;
- Cells genetically modified or otherwise manipulated to express previously unexpressed homologous or non-homologous functional properties.
Tissue engineered product: it is a product that contains or consists of engineered cells or tissues, and is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue.
A tissue engineered product may contain cells or tissues of human or animal origin, or both. The cells or tissues may be viable or non-viable. It may also contain additional substances, such as cellular products, bio-molecules, biomaterials, chemical substances, scaffolds or matrices.
Products containing or consisting exclusively of non-viable human or animal cells and/or tissues, which do not contain any viable cells or tissues and which do not act principally by pharmacological, immunological or metabolic action, shall be excluded from this definition.
Last update: 7 June 2023
Source: Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001