Table 4.

Important considerations during gene editing-based drug development

Product dossier section	Common challenges	Solutions
Quality	Inconsistent manufacture Insufficient product characterization Deficiencies in potency assays Lack of comparability after manufacturing changes	Strategic manufacturing and control strategy for the drug product as well as critical materials Integrated development and regulatory plan Consider related benchmark cases
Nonclinical	Lack of sensitive and relevant models Difficulty to assess safety and pharmacodynamic properties Unsuitable alternative strategies and justification thereof	Smart selection of the lead and back-up candidate Tailored nonclinical program Integrated on-target/off-target assessment report
Clinical	Lack of valid therapeutic endpoints Selection of comparator Lack of proper statistical analysis	Comprehensive benefit/risk assessment Diligent plan towards the first in human study Involvement of regulatory Agencies