Systems Modelling for CRISPR Cas9 and RNAi Therapeutics

1. Using organ-on-a-chip technology to model CAR-T cell therapies and bi-specific antibodies (15 minutes)
o Speaker: Rodrigo Cristofoletti, PhD, Assistant Professor & Associate Director (University of Florida)
o Holistic organ-on-a-chip model to assess recruitment, target-specific efficacy and fitness of CAR-T cell therapies
o Modeling cytokine release syndrome in vitro
2. Systems modelling for CRISPR Cas9 and RNAi therapeutics (20 minutes)
o Speaker: Devam A. Desai, PhD Student, University of Florida
o Translational QSP model for CRISPR – Cas9 gene therapies
o Translational Whole body Physiologically based Pharmacokinetic model for RNAi (ASOs and siRNA) – accessing an impact of molecular descriptors on the PK of RNAi
o Mechanistic PK-PD model for a priori prediction of first-in-human dose for LNP-mRNA encoding mAbs
3. Development and Application of Quantitative Systems Pharmacology Models for Cell Therapies in Oncology and Autoimmune Disorders (20 minutes)
o Speaker: Aman P. Singh, PhD, Director – Quantitative Clinical Pharmacology, Data Science Institute, Takeda Pharmaceuticals
o Develop multi-scale PK-PD relationships to evaluate the impact of Product and manufacturing on Cell therapy clinical pharmacology.
o Development of QSP model to investigate the PK-PD of cell therapies in Autoimmune conditions
4. Mechanistic Pharmacokinetics and Pharmacodynamics of GalNAc-siRNA: Translational Model Involving Competitive Receptor-Mediated Disposition and RISC-Dependent Gene Silencing (20 minutes)
o Speaker: Vivaswath Ayyar, PhD, Associate Scientific Director at Johnson & Johnson
o Quantitative and Clinical Pharmacology Principles for Efficient RNAi Therapeutic Development
o PK translation across two preclinical species (rat and monkey) with subsequent prediction of human plasma PK