Cell and Gene Therapies and Regulation

The ATMP Regulatory Frame, applicable in Europe

Advanced Therapy Medicinal Products (ATMPs) are innovative, regenerative therapies combining aspects of medicine, cell biology, science and engineering for the purpose of regenerating, repairing or replacing damaged tissue/cells. ATMPs are regulated through Regulation (EC) No 1394/2007 and an amendment to Directive 2001/83/EC on medicinal products for human use. The main aspects of the advanced therapy regulation are the centralised marketing authorisation, the establishment of the Committee for Advanced Therapies (CAT), the hospital exemption ruling, specific GMP regulations, and post-authorisation vigilance systems.

ATMPs include any of the following medicinal products for human use:

(overflow those words in order to have their definitions)

(1) a gene therapy medicinal productGene therapy medicinal products means biological medicinal products that are used to treat genetic diseases. Many of these products work by inserting genes (DNA) into cells. When the new gene is integrated into a cell’s chromosomes, the cell produces, or stops the production of a protein, which may help slow down or cure a disease.,

(2) a somatic cell Somatic cell therapy medicinal product means a biological medicinal product which has the following characteristics: a) contains or consists of cells or tissues that have been subject to substantial manipulation so that biological characteristics, physiological functions or structural properties relevant for the intended clinical use have been altered, or of cells or tissues that are not intended to be used for the same essential function(s) in the recipient and the donor; b) is presented as having properties for, or is used in or administered to human beings, with a view to treating, preventing or diagnosing a disease through the pharmacological, immunological or metabolic action of its cells or tissues. therapy medicinal product, or

(3) a tissue engineered productTissue engineered products (TEPs) are medicinal products intended for the structural regeneration, repair or replacement of human tissue or tissue defects. TEPs contain ‘engineered’ cells or tissue, meaning they have been subjected to substantial manipulation and/or that they are not intended for the same essential function in the recipient as in the donor. as defined in the Regulation (EC) No. 1394/2007, or

(4) a combined advanced therapyCombined advanced therapy medicinal product comprise viable cells and, as an integral part, a medical device or active implantable device. medicinal product where the cells are in combination with a medical device.

There are two general approaches to cell therapy: autologous and allogeneic.

Autologous: cells are harvested from a patient, treated or expanded, and then introduced back into the same patient. This is a patient-specific approach.
Allogeneic: cells are harvested from one or more donors,treated or expanded and then introduced back into a patient who is different from the donor(s).