什么是cell and gene therapy？
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Definition of Gene Therapy in the EU
In the EU, the definition of a Gene Therapy Medicinal Product (GTMP) is provided
in Directive 2009/120/EC amending Directive 2001/83/EC, part IV of Annex I.
A GTMP means a biological medicinal product that has the following
characteristics:
(a) it contains an active substance that 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 the
genetic expression of this sequence.
GTMPs do not include vaccines against infectious diseases.
Hazel Aranha, Humberto Vega-Mercado - Handbook of Cell and Gene Therapy_ From Proof-of-Concept through Manufacturing to Commercialization-CRC Press (2023).pdf