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Gene- and Cell-based therapies (GCT) offer promising approaches to treating previously incurable diseases and can support regenerative processes. Gene-based therapies use nucleic acids, i.e. DNA or RNA, which can be used to regulate or add biological functions. Cell therapies generate and/or transfer cells into the organism for therapeutic purposes. These two approaches can also be combined, e.g. using genetically modified cells.

Gene and cell-based therapies include the following procedures (not definitive):
 

  • Therapeutic approaches with drugs for novel therapies (Advanced Therapy Medicinal Products, ATMPs):
    • Somatic cell therapeutics ( for example in the form of stem cells, cells of the immune system or mesenchymal stromal cells )
    • Gene therapeutics in the form of substitution, addition or suppression therapies using viral and non-viral vectors or genome editing
    • Tissue engineering products such as the fabrication of tissue for surgical use, including the use of innovative biomaterials
       
  • Therapeutic approaches with novel biological products, such as mRNA- and other nucleic acid-based methods, extracellular vesicles or exosomes, which are used in the context of a gene- and cell-based therapeutic procedure
     
  • Further approaches of this kind in the context of gene and cell-based therapies
     

The following procedures, however, are not included:
 

  • Approaches that are being developed for different objectives then gene and cell-based therapies (e.g. mRNA vaccination against infectious diseases)
     
  • Approaches based exclusively on small molecules and/or recombinant proteins (including antibodies)

GCTs have an effect that goes beyond purely symptomatic treatment, as they target the causes of disease specifically - with the prospect of cure and regeneration. Their range of applications extends from rare genetic diseases to more common and complex diseases, some of which still have no effective treatments. Despite existing research questions regarding safety and efficacy, millions of patients in Germany alone could benefit from GCT.