We, FDA, are providing you, investigational new drug application (IND) sponsors, with recommendations concerning IND submissions for microbial vectors used for gene therapy (MVGTs) in early-phase clinical trials. MVGTs meet the definition of “biological product” in section 351(i) of the Public Health Service (PHS) Act (42 U.S.C. 262), when such products are applicable to the prevention, treatment, or cure of a disease or condition of human beings. This guidance focuses on the chemistry, manufacturing, and control (CMC) information that you should submit in an IND for MVGTs and provides an overview of preclinical and clinical considerations for these products.
This guidance finalizes the draft guidance of the same title dated October 2015 and supplements the guidance entitled, “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),” dated April 2008.
Posted on the FDA website on 16 September 2016