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.

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

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

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

: Characterizing potential immune responses to the viral vector or the new protein produced by the therapy.

: Determining where the therapeutic gene goes in the body and how long it persists in both target and non-target cells.

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