11:15 AM

Session I: Gene and Cell Therapy –

Drug Target Discovery for Genetic Disorders Leveraging Multi-Omics Data and Comparability Assessment for Manufacturing Changes in CGT, presented by Dr. Lira Pi, Pharmalex;  

Challenges of Underdosing Control in Dose-Escalation Studies for DNA-Based Gene Therapies, presented by Quang Nguyen, Regenereon

1:00 PM

Session 2: Gene Therapy in Rare Disease, presented by Chenkun Wang, Vertex;  

Assessing the Impact of Crossover on the Power for Overall Survival in Randomized Clinical Trials: A Simulation-Based Method, presented by Zhi Yang, BMS

2:00 PM

Statistical Techniques and Learnings from GVHD Treatment Trials, presented by Dr. Laura Morrison, Emmes

9:00 AM

A Case Study: The Adventure of the Missing Data, presented by Dr. Ryan Butterfield and Dr. Viral Panchal, Siemens Healthineers

10:00 AM

Up-Front Matching: A Recruitment Method for Prospective Observational Studies That Mimics Randomization, presented by Ibrahim Turkoz, Janssen

11:15 AM

Optimizing Drug Development Designs for Precision Medicine Considerations, presented by Dr. Gary Cline, Cline Consulting LLC