Explore how Quantitative Systems Pharmacology (QSP) and Virtual Twin® technology are reshaping clinical development strategies in rare diseases. With patient populations often too small for traditional trial designs, Virtual Twin modeling offers a transformative approach to improve therapeutic development and regulatory interactions.
In this on-demand session, our partners from Sanofi showcase real-world applications, including case studies in ASMD and Pompe disease. Learn how QSP-based Virtual Twin models can address unique challenges in rare disease trials and gene therapies. Whether you’re a translational scientist, clinical developer, or regulatory strategist, you’ll gain practical insights into transformative modeling approaches advancing rare disease drug development.
Learning Objectives
By viewing this on-demand session, you will:
- Understand the role and value of QSP in clinical development for rare diseases.
- Learn how Virtual Twin models are developed and used to support therapeutic decisions.
- Explore real-world case studies demonstrating regulatory applications of QSP-based Virtual Twin platforms.
- Identify challenges and opportunities in applying these models to rare disease and gene therapy trials.
Intended Audience
- QSP Scientists and modelers
- Clinical pharmacologists and modelers
- Translational scientists and pharmacometricians
- R&D professionals in drug development
- Regulatory affairs specialists
- Anyone interested in mechanistic modeling or rare disease research
