Gene therapies, such as CRISPR, antisense oligonucleotides (ASOs), and small interfering RNAs (siRNAs), are revolutionizing the treatment of genetic disorders. Despite their potential, optimizing these therapies for maximum efficacy and safety remains challenging. Systems modelling offers a robust approach to address these challenges by providing insights into the pharmacokinetics (PK) and pharmacodynamics (PD) of gene therapies. Expected Outcomes • Detailed Systems Models: Produce comprehensive models for CRISPR, ASOs, and siRNA therapies that can be used for further research and development. • Enhanced Understanding: Gain deeper insights into the factors affecting the efficacy and safety of gene therapies, facilitating more effective treatments. • Optimized Dosing Regimens: Develop personalized dosing strategies that minimize adverse effects and maximize therapeutic benefits. • Improved Therapeutic Outcomes: Reduce the risk of adverse effects and improve the overall outcomes for patients undergoing gene therapy.
Learning Objectives:
Develop Comprehensive Systems Models for CRISPR, ASOs, and siRNA therapies to understand their mechanisms and effects.
Identify critical factors that influence the efficacy and safety of these therapies.
Understanding Dose-Exposure-Response relationships for gene based therapies
