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

Current effective version

Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Revision 1

AdoptedLegal effective date: 01/06/2021Reference Number: EMA/CAT/GTWP/671639/2008 Rev. 1 Corr.

Revision 1 (effective from 1/06/2021)

Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Revision 1

AdoptedLegal effective date: 01/06/2021Reference Number: EMA/CAT/GTWP/671639/2008 Rev. 1 Corr.

Overview of comments received on 'Draft guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Revision 1'

Reference Number: EMA/34765/2019

Draft guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Revision 1

Consultation dates: 31/07/2018 to 31/07/2019Draft: consultation closedReference Number: EMA/CAT/GTWP/671639/2008 Rev. 1Summary:

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans and presented for marketing authorisation. Its focus is on the quality, nonclinical aspects and safety and efficacy requirements of genetically modified cells developed as medicinal products.

Concept paper on the revision of the guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - Superseeding document

Consultation dates: 24/07/2017 to 31/10/2017Draft: consultation closedReference Number: EMA/CAT/424191/2017Summary:

The revised guideline referred to in this concept paper will replace guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells (EMA/CAT/GTWP/671639/2008).

First version

Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - First version

AdoptedLegal effective date: 01/11/2012Reference Number: EMA/CAT/GTWP/671639/2008Summary:

This guideline defines scientific principles and provides guidance for the development and evaluation of

medicinal products containing genetically modified cells intended for use in humans and presented for

marketing authorisation. Its focus is on the quality, safety and efficacy requirements of genetically

modified cells developed as medicinal products.

Overview of comments received on 'guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - First version

Reference Number: EMA/CAT/GTWP/155212/2011

Draft guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells - First version

Consultation dates: 20/05/2010 to 30/11/2010Draft: consultation closedReference Number: EMA/CHMP/GTWP/671639/2008

Concept paper on the development of a guideline on the quality,Preclinical and clinical aspects of medicinal products containing genetically modified cells - First version

Reference Number: EMEA/CHMP/GTWP/405681/2006