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.
: Assessing the risk of "insertional mutagenesis"—where the therapy might accidentally interfere with the patient's existing DNA. 125459
: Checking if the patient might "shed" or spread the virus/vector through secretions. This document outlines the essential safety and efficacy
: Defining scientific principles for non-clinical studies specifically for gene therapies, which differ significantly from conventional pharmaceuticals. Key Safety Requirements : 125459
: This guideline is a cornerstone for developers of Advanced Therapy Medicinal Products (ATMPs) in the European Union.