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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

The number most commonly refers to a specific scientific guideline established by the European Medicines Agency (EMA) regarding the development of Gene Therapy Medicinal Products (GTMPs) . This document outlines the essential safety and efficacy

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

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

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