Webinar Details
This webinar was hosted by Genetic Engineering & Biotechnology News and recorded on April 3, 2024.
Cell and gene therapies are exciting new modalities that have the potential to treat, and even cure, a range of diseases that may not be possible with small molecules or monoclonal antibodies. However, there are many unique challenges for biotech and pharma companies developing these therapies, such as understanding the risks of developability, the balance of safety vs. efficacy, and determining if these therapies will be cost effective or better than the standard of care.
Quantitative systems pharmacology (QSP) can be a valuable resource for elucidating the interactions between potential therapies, biological systems, and disease mechanisms. In this GEN webinar, our expert speakers will discuss how QSP modeling improves early feasibility assessment, rational therapy design, and clinical development of cell and gene therapies. Attendees will learn about three case studies that illustrate some exciting applications of QSP modeling:
- mRNA delivery through LNP for the treatment of Crigler-Najjar Syndrome Type 1 (CN1)
- CAR-T therapy for B-cell non-Hodgkin Lymphoma
- Stem cell gene therapy for sickle cell disease