Gene therapy and genome editing have revolutionized our approach to the development of new therapeutics for previously untreatable medical conditions. Much of this progress has been due to designer nucleases (most notably CRISPR-Cas9) that have enabled targeted genomic modifications. The delivery mechanism(s) of these genome-modifying tools is equally important and can be accomplished in variety of ways, including ex vivo and in vivo approaches. As more investigational agents are progressing through the pre-clinical pipeline and arriving to clinical trials, it will become critically important to be mindful of how we regulate such products and ensure that there is equitable and affordable access to them.