Gene And Cell Therapy Products Community

Scope (1.3)

GT products within the scope of this guidance include products that mediate their effect by the expression (transcription or translation) of transferred genetic materials. Some examples of GT products can include purified nucleic acid (e.g., plasmids and RNA), microorganisms (e.g., viruses, bacteria, fungi) genetically modified to express transgenes (including products that edit the host genome), and ex vivo genetically modified human cells. Products that are intended to alter the host cell genome in vivo without specific transcription or translation (i.e., delivery of a nuclease and guide RNA by nonviral methods) are also covered in this guidance. Although not currently considered GT in certain regions, the principles outlined in this guidance are also applicable to oncolytic viruses that are not genetically modified to express a transgene.

This guidance does not apply to prophylactic vaccines. Chemically synthesized oligonucleotides or their analogues, which are not produced using a biotechnology-based manufacturing process, are also outside the scope of this guidance.

The release of a GT product outside the body via excreta and secreta (feces, urine, saliva, nasopharyngeal fluids, etc.), or through the skin (pustules, sores, wounds) is termed shedding. Evaluation of the nonclinical shedding profile of a GT product is outside the scope of this guidance. Assessment of genomic integration and germline integration of GT products is also outside the scope of this guidance. Considerations for these aspects of nonclinical data can be found in existing International Council for Harmonisation (ICH) consideration documents (1, 2).