Questions and answers on gene therapy - Scientific guideline

This document addresses questions on matters related to the development of gene therapy medicinal products. It provides harmonized position on issues that can be subject to different interpretation or require clarification, typically arising from discussions during briefing meetings with stakeholders.

Keywords: Gene therapy medicinal products, European Pharmacopoeia, comparability, characterisation, host cell DNA, selection marker, potency, viral shedding, GMO requirements

Current version

Questions and answers on gene therapy

Adopted Reference Number: EMA/CHMP/GTWP/212377/2008