Three-year efficacy and safety results from a phase 1/2 clinical study of AAV5-hFVIII-SQ gene therapy (valoctocogene roxaparvovec) for severe hemophilia A (BMN 270-201 study)
Objective: Hemophilia A (HA) is an X-linked disorder caused by mutations in the gene encoding Factor VIII protein (FVIII). Gene therapy is increasingly being viewed as a viable treatment option for hemophilia. Herein, long-term…
Gene Therapy Licensing Agreement Between uniQure and CSL Now in Effect
uniQure recently announced that their commercialization and license agreement with CSL Behring  for their investigational hemophilia B gene therapy was finally closed on May 5, 2021 – the agreement had been pending the completion of a series of…
Nancy Messina
Peter Senior
Gary Pennington
Bruce Weiner
Mike Miller
Ashley Castello
Brittani Spencer
Factor VIII deficiency is associated with abnormal brain volumes