Gene therapy medicinal products (GTMPs)
Gene therapy medicinal products contain recombinant nucleic acid, which is administered to human beings in order to reach a therapeutic, prophylactic or diagnostic effect. The intended effect of the product is directly related to the sequence of the nucleic acid.
The full definition according to the European guidelines is as follows:
Gene therapy medicinal product means a biological medicinal product which has the following characteristics:
(a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence;
(b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence.
Gene therapy medicinal products shall not include vaccines against infectious diseases.
More information can be found in Directive 2009/120/EC, Annex 1 part IV Directive 2001/83/EC and amendment Directive 2003/63/EC part IV.
Examples of GTMPs include plasmids of biological origin, viral vectors and genetically modified human cells (e.g. CAR-T cells).
More information on legislation and regulations for research with genetically modified organisms (GMOs) can be found on the website of the Gene Therapy Office.