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The BEQVEZ gene therapy inserts a working copy of the Factor IX (FIX) gene that codes for a high-activity FIX variant. Patients with moderate to severe hemophilia B can now take Pfizer’s BEQVEZ (fidanacogene elaparvovec-dzkt) with the approval of the FDA. Hemophilia B is an inherited bleeding disorder that affects more than 38,000 people globally and is caused by impaired blood clotting due to a factor IX (FIX) deficiency. The current gold standard for FIX replacement therapy prevention is infusions given once weekly or monthly. These infusions supplement or temporarily replace low blood clotting factor levels. Despite receiving prophylaxis and regular intravenous infusions, people with moderate to severe hemophilia B may still experience spontaneous bleeding episodes. Additionally, the current standard of care places a burden on healthcare systems’ budgets and resource allocation...
