Date
Time
Speakers: Lindsey George, MD; John Morris, PhD; Bill Maurits
This session of the Research Journal Club will focus on gene therapy. Gene therapy for hemophilia A is now commercially available in some countries. While early patient data show a modest reduction in disease severity, current FVIII gene therapies using viral delivery have not yet maintained consistent desired results year after year. This session will explore current work in Dr. Lindsey George’s laboratory at the Children’s Hospital of Philadelphia to use mechanisms of FVIIIa inactivation to engineer enhanced transgenes for hemophilia A gene therapy.
John Morris, PhD
Dr. John Morris is a NHLBI/NIH T32 trainee in hemostasis and thrombosis at the University of Pennsylvania and a postdoctoral fellow at the Children’s Hospital of Philadelphia in Dr. Lindsey George’s lab. His research focuses on defining the biochemistry of activated factor VIII inactivation and exploiting this understanding to engineer gain-of-function clotting factors resistant to inactivation to improve translational applications in hemophilia gene therapy. Additionally, his work aims to develop comprehensive animal models of hemostasis to adequately assess the prothrombotic risk associated with enhanced transgenes that bypass regulatory pathways.
Lindsey George, MD
Dr. George is an assistant professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania and director of clinical in vivo gene therapy at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation with a focus on mechanisms that regulate factor VIII/VIIIa cofactor function to improve understanding of associated disease states of deficiency, hemophilia A, or excess function with associated thrombosis. Ongoing studies in her group aim to exploit their biochemical insights of FVIII function to develop novel gene-based therapies for hemophilia A. Dr. George previously led early-phase, adeno-associated virus (AAV) mediated gene transfer trials for hemophilia A and B. Dr. George is a member of the board of directors of the American Society of Gene and Cell Therapy and participates in multiple national and international professional societies for hemostasis and gene therapy research.
Bill Maurits
Bill Maurits is a 53-year-old man with hemophilia B who’s lived through the entire evolution of hemophilia treatment, including a gene therapy trial that changed his life. He’s a devoted husband and proud dad who loves hitting the road with his family, rocking out at concerts with his wife, getting his hands dirty restoring classic cars, and trying to keep up with his 13-year-old son in backyard sports and Taekwondo (though his son is starting to give him a run for his money). Bill brings humor, resilience, and real-life perspective to every audience he meets.