The FDA's Final Guidance On Early-Phase Cell & Gene Therapy Trials

By Tim Sandle, Ph.D.

The cell and gene therapy development process involves carefully screening variants of a product type. To aid this initial process and to ensure that suitable evidence is gathered so that effective variants of the product are taken through to later-stage trials, the FDA has produced finalized guidance, Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial. Cell and gene therapy products are combined in this document, ostensibly because they represent overlapping fields of biopharmaceutical research and possess similar therapeutic goals, in targeting DNA or RNA inside or outside the human body.