Gene therapeutics


Advanced Therapy Medicinal Products (ATMPs) are drugs produced by genetic engineering methods and used to treat serious or life-threatening diseases. Here are some examples of genetically engineered ATMPs that are in clinical use:

 

  1. Gene therapy
    Gene therapeutics are used to treat genetic diseases. For example, CAR-T cell therapy is used to treat cancers such as leukaemia and lymphoma.
     
  2. Genome editing
    Targeted editing of DNA makes it possible to remove, add or change specific genes in the genome. CRISPR/Cas systems, the targeted cutting of the DNA starng, are used for genome editing and have the potential to treat many diseases, including genetic diseases.
     
  3. Cell therapy
    Cell therapeutics are produced by taking the body's own cells, genetically modifying them and putting them back into the body. An example of cell therapy is CAR-T cell therapy, in which T cells are taken from the patient's body, genetically modified and then returned to the patient's body.
     

As with all ATMPs, the list of possible therapies is not complete. Because ATMPs are still in their infancy, a great deal of research and study is taking place at the individual levels. The research and development of ATMPs has the potential to revolutionise the treatment of many severe and previously incurable diseases.