Preclinical considerations for assessment of cellular & gene therapy products: FDA perspective

The conduct of a clinical trial for an investigational cellular or gene therapy (CGT) product is governed by the Code of Federal Regulations (CFR) Title 21, Part 312 to ensure the safety and rights of human subjects in all phases of a clinical investigation. Preclinical evaluation of CGT products in vitro and in vivo contributes significantly to characterization of the product benefit:risk profile throughout clinical development. Major objectives of the preclinical program for CGT products are to establish the scientific rationale to support the planned clinical investigation and to assess any toxicities associated with administration of the investigational CGT product. This article will discuss: 1) potential safety concerns associated with CGT products; 2) preclinical testing approaches to support an Investigational New Drug (IND) submission for a first-in-human (FIH) clinical trial and a Biologics License Application (BLA); and 3) early communications with the Food and Drug Administration (FDA).

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