Design modifications of gene therapy medicinal products during development - Scientific guideline

This document presents regulatory considerations given for specific gene therapy medicinal products where the characteristics have been changed at various stages during clinical development. It gives some insight into the types of studies that are likely to be required in an application dossier to support the modification in the product design introduced during development.

Keywords: Gene therapy, modifications to product design

Current version

Reflection paper on design modifications of gene therapy medicinal products during development

Adopted Reference Number: EMA/CAT/GTWP/44236/2009 Summary:

This paper focuses only on changes made to the design of the product with a purpose to improve the efficacy and/or safety of the product.