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TableĀ 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