CMC information for gene therapy INDs addressed

FDA issued a final guideline on April 9 specifying the type of CMC information to be included in investigational new drug applications (INDs) for gene therapy products. The guidance specifies the necessary sterility testing and microbiological testing to support INDs, and replaces a November 2004 draft. The guidance notes that "in order to deliver a safe and effective product, human gene therapies present many manufacturing challenges. Some of these challenges include the variability and complexity inherent in the components used to generate the final product, such as the source of cells, the potential for adventitious agent contamination, the need for aseptic processing, and in the case of ex vivo genetically modified cell therapies the inability to 'sterilize' the final product because it contains living cells.