125459 ✦ Top-Rated & Extended

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

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

: 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

: Checking if the patient might "shed" or spread the virus/vector through secretions. Key Safety Requirements : : Checking if the

: Assessing the risk of "insertional mutagenesis"—where the therapy might accidentally interfere with the patient's existing DNA.

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.

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