Human Gene Therapy Products
Incorporating Human Genome Editing: Draft Guidance for Industry
"For in vivo genome modification, GE components may be delivered by viral
vectors or nanoparticles. When choosing an in vivo delivery method, it is
important to consider the ability of the delivery vector to target the cells/tissue of
interest and minimize distribution to non-targeted tissue. Consideration should
also be given to the ability to control expression of vector delivered GE
components (e.g., using tissue-specific promoters, small molecule inhibitors), if
appropriate. Viral vectors may support sustained expression of GE component
transgenes, and nanoparticles may allow the temporal delivery of GE components as messenger RNAs or proteins. The potential for vector-mediated toxicity as well as pre-existing immunity to the GE component and vector should also be considered. The sponsor should select the appropriate delivery method based on the intended use. "
Well, there it is:
Human Gene Therapy Products Incorporating Human Genome Editing: Draft Guidance for Industry
"For in vivo genome modification, GE components may be delivered by viral vectors or nanoparticles. When choosing an in vivo delivery method, it is important to consider the ability of the delivery vector to target the cells/tissue of interest and minimize distribution to non-targeted tissue. Consideration should also be given to the ability to control expression of vector delivered GE components (e.g., using tissue-specific promoters, small molecule inhibitors), if appropriate. Viral vectors may support sustained expression of GE component transgenes, and nanoparticles may allow the temporal delivery of GE components as messenger RNAs or proteins. The potential for vector-mediated toxicity as well as pre-existing immunity to the GE component and vector should also be considered. The sponsor should select the appropriate delivery method based on the intended use. "
https://www.fda.gov/media/156894/download