125459 Apr 2026

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

: This guideline is a cornerstone for developers of Advanced Therapy Medicinal Products (ATMPs) in the European Union. 125459

The number most commonly refers to a specific scientific guideline established by the European Medicines Agency (EMA) regarding the development of Gene Therapy Medicinal Products (GTMPs) . Guideline on the Non-Clinical Studies Required before First

For more detailed technical specifications, you can access the full Guideline on Non-Clinical Studies for GTMPs via the official . For more detailed technical specifications, you can access

: Defining scientific principles for non-clinical studies specifically for gene therapies, which differ significantly from conventional pharmaceuticals. Key Safety Requirements :

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

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