Gene Therapy Provides Hope to Hemophilia B Patients

In 2020, CSL Behring entered into a global license and commercialization agreement with UniQure for the company’s experimental hemophilia B gene therapy. The expected, one-and-done treatment carries the potential to free people with the genetic bleeding disease from reliance on regular infusions of clotting factor IX for which they are deficient. We spoke to Steve Pascoe, senior vice president and head of therapeutic areas and development strategy for R&D at CSL about hemophilia B, how the gene therapy fits into CSL’s broader therapeutic offerings, and the encouraging results from the recent pivotal study.