Scientific progress in the field of cellular and molecular biotechnology has allowed the development of numerous Advanced Therapy Medicinal Products (ATMPs), offering new and innovative treatment opportunities. ATMPs are biological medicines which are classified into four main groups:
- Gene therapy medicinal products: they contain or consist of a recombinant nucleic acid capable of inducing a therapeutic, prophylactic or diagnostic effect. Gene therapy drugs permit to adjust, repair, replace, add or delete a genetic sequence. In the case of genetic diseases in which a gene is defective or absent, gene therapy allows to transfer the working copy of the gene in question.
- Somatic cell therapy medicinal products: they contain or consist of cells or tissues which have been significantly manipulated to modify their biological characteristics, physiological functions or structural properties or which are not intended to be used for the same original functions in the body. The purpose of somatic cell therapy is to treat, prevent or diagnose diseases. The cells or tissues can be of autologous origin (derived from the patient himself), allogeneic (obtained from a donor) or xenogenic (derived from a donor of an animal species other than man).
- Tissue engineered medicinal products: they contain cells or tissues that have undergone significant manipulation or are not intended to be used for the same original functions in the body, with the aim of repairing, regenerating or replacing human tissues.
- Medicinal products for combined advanced therapies: they contain one or more medical devices as an integral part of the medicinal product containing cells or tissues.