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[Therapeutic approaches using genetically modified cells].

Brigitte Anliker1, Matthias Renner1, Matthias Schweizer2

  • 1Paul-Ehrlich-Institut, Fachgebiet 6/2, Paul-Ehrlich-Straße 51-59, 63225, Langen, Deutschland.

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Genetically modified cell therapies show promise for treating inherited diseases and cancers. While not yet licensed in Europe, these advanced gene and cell therapies are progressing through clinical trials.

Keywords:
Cell therapyChimeric antigen receptor/T-cell receptorGene therapyGenetically modified cellsInherited immunodeficiency

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Area of Science:

  • Biotechnology
  • Genetics
  • Immunology

Context:

  • Medicinal products with genetically modified cells are typically gene and cell therapy products.
  • No genetically modified cell products are licensed in Europe currently.
  • Numerous therapeutic strategies using genetically modified cells are in clinical development.

Purpose:

  • To present promising therapeutic strategies using genetically modified cells.
  • To highlight gene therapy for inherited immunodeficiencies.
  • To discuss tumor immunotherapy using genetically modified T-cells.

Summary:

  • Focuses on gene therapy for inherited immunodeficiencies.
  • Explores tumor immunotherapy with genetically modified T-cells, including CAR-T and TCR therapies.
  • Discusses the clinical development of various genetically modified cell therapies.

Impact:

  • Provides an overview of advanced cell and gene therapies.
  • Highlights potential treatments for acquired and inherited diseases.
  • Informs about the current landscape and future prospects of genetically modified cell medicinal products.