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.
: Characterizing potential immune responses to the viral vector or the new protein produced by the therapy. 125459
: This guideline is a cornerstone for developers of Advanced Therapy Medicinal Products (ATMPs) in the European Union. This document outlines the essential safety and efficacy
: Evaluating whether the viral vector or therapeutic gene could unintentionally be passed on to future generations. 125459
: Checking if the patient might "shed" or spread the virus/vector through secretions.