GUIDANCE DOCUMENT

Final

This guidance is intended to provide industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during the development of cellular and gene therapy (CGT) products and is intended to help facilitate the development of safe, effective, and high-quality CGT products.  The FAQs represent common questions directed to the Agency and span multiple disciplines, including regulatory review, chemistry, manufacturing, and controls (CMC), pharmacology/toxicology (PT), clinical, and clinical pharmacology.

In general, FDA’s guidance documents, including this guidance, do not establish legally enforceable responsibilities.  Instead, guidances describe the FDA’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited.  The use of the word should in FDA’s guidances means that something is suggested or recommended, but not required.