“Future technologies for curing hemophilia – Part 1” was held yesterday at the WFH Virtual Summit. The session was moderated by Glenn Pierce, MD, PhD, WFH Vice-President, Medical. Looking at new technologies and how they will impact hemophilia treatment, the event showed how a number of promising new developments are supporting the search for a cure.
Speaker Antonia Follenzi talked about combined gene and cell therapy for the treatment of hemophilia A using an implantable device. Thomas Harrington gave an update on the follow-up of the high-dose cohort in the Alta Study, a phase 1/2 study of SB-525 gene therapy in adults with severe hemophilia A. Shielded cell therapy was shown to be a novel approach for rare bleeding disorders by speaker Devyn Smith. Annemieke Willemze discussed BIVV001 (rFVIIIFc-VWF-XTEN): a new class of factor VIII (FVIII) replacement for hemophilia A that uncouples FVIII from endogenous von Willebrand factor (VWF).
To watch sessions from the WFH Virtual Summit – Connecting the Global Bleeding Disorders Community, please click here.