125459 May 2026

The guideline, officially titled , provides a framework for non-clinical studies required before a gene therapy can be used in human clinical trials. Guide to EMA Guideline 125459

Guideline on the Non-Clinical Studies Required before First ... - EMA 125459

For more detailed technical specifications, you can access the full Guideline on Non-Clinical Studies for GTMPs via the official . The guideline, officially titled , provides a framework

: Evaluating whether the viral vector or therapeutic gene could unintentionally be passed on to future generations. 125459

This document outlines the essential safety and efficacy data that developers must generate during the "non-clinical" (animal or lab) phase to justify moving into human subjects.