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

: Defining scientific principles for non-clinical studies specifically for gene therapies, which differ significantly from conventional pharmaceuticals. Key Safety Requirements : 125459

: Checking if the patient might "shed" or spread the virus/vector through secretions. The guideline, officially titled , provides a framework

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

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