Table 4 Important considerations during gene editing-based drug development
From: From fiction to science: clinical potentials and regulatory considerations of gene editing
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 |