In the vast landscape of CRISPR-based therapeutic avenues, two primary paradigms emerge ex-vivo and in-vivo. Today, we shall channel our focus on the latter, unravelling the intricate tapestry of its biodistribution. Using Transthyretin Amyloidosis as our vantage point, we will be showcasing some preliminary findings from our sophisticated translational mPBPK (minimal Physiologically-Based Pharmacokinetic) model. Our analysis will shed light on how varying dosages of in-vivo CRISPR gene therapy modulate exposure.
Learning Objectives:
Understanding Dose-Exposure Relationship for CRISPR-Cas
Understanding the Biodistribution of CRISPR-Cas
Model Informed Drug Development approaches for CRISPR-Cas