Preclinical Study Design for CRISPR-based Therapeutics

This white paper discusses the preclinical study design necessary for CRISPR-based therapeutics, emphasizing the importance of safety and appropriate dosage levels during drug development. It outlines the differences between preclinical studies and earlier discovery-phase studies, highlighting that preclinical studies are conducted using animal models or organoids to predict clinical trial outcomes. The document details essential safety studies required by regulatory agencies prior to Investigational New Drug (IND) submission, which support clinical trial design and dosing recommendations. A strategic approach to preclinical development is presented, stressing the need for early planning and unique assessments for cell and gene therapy (CGT) products. The paper explains the significance of selecting appropriate animal models for efficacy and safety testing, conducting pilot studies, and using clinical-grade materials throughout the development process. It also addresses pharmacology studies, proof of concept studies, and the importance of biodistribution analysis to ensure safety and efficacy in clinical applications.