ABSTRACT
Administration of the cellular and gene therapy (CGT) product in a clinical trial does not necessarily signify the completion of nonclinical evaluation. Preclinical studies to assess the safety of the CGT product prior to administration in a clinical trial should employ designs that identify, characterize, and quantify local and systemic toxicities, even if the CGT product is administered to a presumed local site. The regulatory oversight of advanced therapies varies, depending on the modality in question. Cellular therapies can be simply defined as any modality that employs the use of an intact cell as the primary means to impart an effect. In many ways, the therapeutic strategy of genome editing is similar to standard viral-based gene therapies. Two main safety concerns have arisen in the evolution of T-cell immunotherapy platform—significant cytokine release and the potential for normal tissue targeting by the modified.
