Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the co-Director for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NExTRAC advisory committee evaluating the emergence of new technologies.
Keith Yamamoto, UCSF
Patient-centric data – Real-World Evidence (RWE) and Real-World Data (RWD) - is becoming instrumental in the drug development process and for health care decisions in general. This data is not only informative for the process from discovery to new indications, clinical trial design, and drug development, it also can be of value to monitor post-marketing drug safety and for decision support in clinical practice. As the data becomes a decision driver, science companies and medical organizations are increasingly focused on leveraging RWD and RWE to not only better understand the patient populations using their drugs and the respective outcomes, but also to accelerate clinical decision support. This session will focus on the various aspects of integrating RWE and RWD to support drug development and clinical decision support