The FDA has issued its guidance for chemistry, manufacturing, and control information that is included in investigational new drug applications (INDs) for gene therapies, called “Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).” The guidance finalizes the draft guidance that was released for comment in November 2004. As the FDA explains in the introduction to the guidance, 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 (i.e., autologous or allogeneic), 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.
The guidance addresses these concerns, as well as issues related to the distribution of gene therapy products, which can also be a challenge because of stability issues and the frequently short dating period of many ex vivo genetically modified cell products, which may necessitate release of the final product for administration to a patient before certain test results are available.
Guidance (PDF)
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