Toxicology

The most common approach for toxicology evaluation corresponds to single-dose administrations in relevant species, with subsequent long-term follow-up (e.g., up to 6 or 9 months), depending on the anticipated persistence of the lesion or tumor cell behavior, to cover the potential effects of the cell drug product (DP) to which the patient will be chronically exposed after the single dose of the cell drug product (DP). If repeated administrations in humans are anticipated, the animal studies will also be of the repeated-dose type such that the administration schedule mimics the human one, and the duration of the effect (biodistribution, vector persistence, and gene product persistence) will be in line with that intended in humans. Similar to small molecules compound, biodistribution is a fundamental to identifying target organs and anticipating safety and efficacy, and may eventually be anticipated based on the specificities for research dimensions and constructs according to different experiment models.