The WFH 2018 World Congress will feature speakers who will discuss important—and cutting edge—topics facing the bleeding disorders community today. In the lead-up to Congress, Hemophilia World Online will be profiling several prominent speakers who will be presenting this May. Today, we look at Thierry VandenDriessche, PhD.
Name: Thierry VandenDriessche
Talk: Gene therapy: ready for prime-time?
Summary: Preclinical studies demonstrate the efficacy of and safety of gene therapy in hemophilic mice and dog models. It is encouraging that sustained therapeutic factor VIII and IX expression levels can now be attained after gene therapy in patients with severe hemophilia A or B, paving the way towards a bona fide cure. Nevertheless, there are still challenges that would need to be overcome to maximize the benefits of gene therapy for patients with hemophilia.
About the speaker: Thierry VandenDriessche obtained his PhD at the Free University of Brussels (Belgium) in 1992, and was a visiting fellow at the Weizmann Institute for Science (Israel) in the field of gene therapy for cancer. He continued his research as a post-doctoral fellow at the National Institutes of Health (USA) in the lab of gene therapy pioneers R. Michael Blaese, Rick Morgan and W. French Anderson, where he started his research on gene therapy for hereditary and infectious disease. He is now Director of the Department of Gene Therapy & Regenerative Medicine at the University of Leuven.
VandenDriessche has published over 130 peer-reviewed publications. He has received several awards for his research, including the European Society of Gene & Cell Therapy Outstanding Achievement Award, the Molecule of the Year Award and the Francqui Chair.
VandenDriessche’s main research focuses on gene therapy for hereditary diseases—particularly hemophilia—and the development of improved gene therapy and gene editing technologies.
His team has showed that hemophilia A can be cured by gene therapy in preclinical models. They have also demonstrated that the effectiveness of gene therapy can be substantially improved by using the hyper-functional factor IX Padua.
To find out more about other speakers who will be at the WFH 2018 World Congress, please visit the Congress program page.