Gene therapy products study design

The clinical indication and specific development plan should drive the design of the safety evaluation programme for gene therapy products. For example, ex vivo treatment of somatic or stem cells with a gene therapy vector followed by re-introduction of these cells into the host is not very amenable to risk assessment with animal studies. The persistence of the gene or the vector in the treated cells will, in many cases, be all that can be assessed in these instances animal toxicology studies will yield little useful information. 

As with most biotechnology products, the assessment of geno-toxic potential using conventional screening assays is of little use for gene therapy products. It may be necessary, however, to conduct specific studies to address this possibility this issue will be considered further below. The need for, and design of, these studies should be carefully considered and based on scientific rationale. 

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