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FDA advises caution for gene therapy trials

At a recent meeting, the FDA advised a cautious approach for gene therapy trials. The two day CTGTAC meeting discussed the toxicity risks, in particular of AAV based gene therapy. The panel of advisers made a number of recommendations to enhance safety profile testing of gene therapy vectors including increasing the duration of testing in animals, comprehensive screening of patients in clinical trials and increased monitoring of patients in follow up assessments. There was much debate on whether gene therapy can cause cancer as a side effect. With inconclusive and oversensitive animal models being used, there is a need to be employ larger animal or human models to relate the toxicity risk to patients in the clinic. This shows the benefit of TestAVec's hInGeTox platform, employing the world's first and patented in vitro human platform for testing genotoxicity.


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