FDA Guidance on Gene Therapy Clinical Trials

4 October 2005

Recent draft guidance on the design of clinical trials involving gene transfer technology issued by the US Food and Drugs Agency (FDA) has called for long-term follow-up of trial participants, in order to properly monitor for long-term adverse effects of gene therapy treatments and mitigate their potential impact on participants. Gene transfer technology is defined as gene therapy products or cells or tissue that has been transduced with gene therapy products ex vivo. The document also provides a framework for risk-assessment to determine whether the trial involves high risks that merit long-term follow-up. Factors considered likely to increase the risk of delayed adverse events include persistence of the viral vector, integration of genetic material into the host genome, prolonged expression of the transgene, and altered expression of the host

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