ATMPs, or Advanced Therapy Medicinal Products, represent a highly innovative class of biologics that are fundamentally based on genes, tissues, or cells, signifying a paradigm shift in modern medicine. These therapeutic agents are highly complex, often targeting diseases at the molecular or cellular level to repair, replace, regenerate, or modify human tissues and biological function. They are specifically categorized into three main types: gene therapy, somatic cell therapy, and tissue-engineered products, offering curative potential for severe diseases.
Origin
The term ATMP is a regulatory classification established by the European Commission under Regulation (EC) No 1394/2007, reflecting the unique scientific and technical complexity of these novel products. This designation arose from the need for a harmonized regulatory framework to ensure the quality, safety, and efficacy of these highly innovative, biologically sourced medicines across the European Union. Their development is closely tied to the emergence of regenerative medicine and advanced biotechnology in the late 20th and early 21st centuries.
Mechanism
The mechanism of ATMPs involves introducing a modified biological substance into the body to achieve a profound therapeutic effect, rather than relying on a traditional small molecule drug to bind a single target. Gene therapies operate by inserting a recombinant nucleic acid to correct a genetic defect, while somatic cell therapies use substantially manipulated cells to restore or change physiological function. Tissue-engineered products work by containing or consisting of engineered cells or tissues intended to regenerate, repair, or replace damaged human tissue structures.
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