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Focus on Regulation

Scientific progress enables EMA to improve guidance on medicinal products containing genetically modified cells

The European Medicines Agency (EMA) has released a concept paper concerning a proposed revision of the current guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells. The proposed revision will update the previous Guideline of 2012.

EMA’s concept paper is intended to address the issue that the current guideline does not reflect scientific progress that has been made since its entry into force. The current guideline focuses on genetic modifications by traditional methods, based on the use of vectors carrying recombinant nucleic acids. However, five years after the entry into force of the current guideline newer genome-editing tools are available. The use of these tools to genetically modify cells ex vivo for clinical applications has already begun and is expected to increase rapidly. These tools may use different starting materials and are able to allow more precise gene modifications. The current guideline does not address these specific issues as it offers only “very general recommendations” on genome-editing tools. Furthermore, EMA outlines that genome editing techniques raise new concerns such as off-target genomic modifications for which guidance is necessary.

The recently released concept paper also makes reference to the dramatic increase that has occurred in the use of genetically-modified cells for cancer immunotherapy. According to EMA, increasing CAR-T cells or recombinant TCR T cells require that the current guideline be revised. Additionally, a number of medicinal products based on CAR-T cells are already authorised. As a result, the current guideline needs to be further revised in order to provide experience specific to CAR-T cells.

The concept paper has been released for three months external consultation. Bio-pharmaceutical industries, academia or other developers of gene and cell therapy medicinal products can provide their comments until 31 October 2017.