Table 3.

Comparison of regulatory stance for genome-editing products for gene therapy

	US FDA	EU EMA	Japan MHLW/PMDA
Relevant guidelines, etc.	Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (2020.1).	Guideline on the quality, nonclinical and clinical aspects of gene therapy medicinal products (2018.3)	Guidelines for Gene Therapy Clinical Research (2019, 2)
	Long-Term Follow-Up After Administration of Human Gene Therapy Products (Draft, 2020.1)	Guideline on quality, nonclinical and clinical aspects of medicinal products containing genetically modified cells (Draft, 2018.7)	Science Board: Reflection paper for genome-editing products (2020, 2)
Genome editing	No description on tools for genome editing	Products consisting of recombinant nucleic acid as an active ingredient and other components, and causing control, repair, replacement, insertion, or deletion of DNA sequences in humans	Modification of human genes of specific target DNA sequences and administration of genetically modified cells
Products including genome editing and raw materials	Gene therapy includes viral vectors and plasmids, as well as genome editing using mRNA	Vectors (including mRNA) coding for enzymes used for gene modification, enzyme proteins for gene modification, nucleic acid for genome editing, nucleic acid templates to be knocked in, and cells to be modified	Viral vectors, plasmids, mRNA, or proteins that modify the specific DNA sequences, nucleic acid, etc.