125459 May 2026

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

: Assessing the risk of "insertional mutagenesis"—where the therapy might accidentally interfere with the patient's existing DNA. 125459

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. : Determining where the therapeutic gene goes in