As we enter the gene therapy era for hemophilia, a critical limitation is our knowledge and understanding of the approach. The promise of gene therapy is a single treatment (“one and done”) that leads to steady-state expression of endogenous factor VIII or factor IX sufficient to achieve a functional cure (free of recurrent hemophilic bleeding), if not normalized hemostasis. In yesterday’s session “Medical plenary: gene therapy”, moderated by Glenn Pierce, MD, PhD, WFH Vice-President, Medical, this exciting research avenue for people with hemophilia (PWH) was discussed in detail.
Pierce explained that gene therapy has the potential to offer optimized health and well-being at a level unimaginable several decades ago. But delivering on this promise will require broad-based multi-stakeholder preparation with a focused emphasis on education, approval of safe and effective therapies, and the removal of barriers to access and excellence in clinical delivery. Steven Pipe went into more detail about delivering on the promise of gene therapy for hemophilia, and Luigi Naldini talked about lentiviral therapy in hemophilia.
To watch sessions from the WFH Virtual Summit – Connecting the Global Bleeding Disorders Community, please click here.