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Recombinant adeno-associated virus (rAAV) is generally considered a non-integrating vector. Most vector DNA remains episomal, but rare integration into host DNA does happen. The safety concern is not simply whether integration occurs, but where it occurs and whether it can affect genes involved in cancer. This question has received particular attention in the liver. Earlier studies in neonatal mice treated with high-dose, liver-directed rAAV found an association between integ

The FDA's 2023 draft guidance, Potency Assurance for Cellular and Gene Therapy Products, represents an important evolution in how cell and gene therapy developers should approach potency throughout product development. The earlier 2011 guidance focused primarily on developing appropriate potency tests. The newer draft expands this concept into a broader potency assurance strategy that connects product understanding, manufacturing controls, material controls, analytical charac