Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Scientific guideline

The original guideline was developed in 2010-2011, before the first gene therapy medicinal product based on genetically modified cells was authorised. The revision of the guideline reflects the experience gained since then with the approval of this type of gene therapy products. Additionally, science has moved on rapidly, and novel technologies that were not yet at the horizon in 2010 are now commonplace: these include CAR-T cells, induced pluripotent stem cells and genome editing. The revision does also incorporate guidance of genetically modified cells developed using these novel technologies.

Keywords: Genetically modified cell, advanced therapy, gene therapy, cell therapy, somatic cell, quality, non-clinical, clinical