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
: Determining where the therapeutic gene goes in the body and how long it persists in both target and non-target cells. The guideline, officially titled , provides a framework
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. This document outlines the essential safety and efficacy
: Checking if the patient might "shed" or spread the virus/vector through secretions.
: Evaluating whether the viral vector or therapeutic gene could unintentionally be passed on to future generations.
: Defining scientific principles for non-clinical studies specifically for gene therapies, which differ significantly from conventional pharmaceuticals. Key Safety Requirements :