In a significant development reported by HCP Live (2025), a gene therapy known as etranacogene dezaparvovec (HEMGENIX) has shown promising long-term results for patients with hemophilia B. The Phase 3 HOPE-B study revealed that over a period of four years, more than 90% of the patients treated with this one-time infusion were able to stop their regular prophylactic treatments. This gene therapy has been effective in maintaining factor IX activity, which is crucial for blood clotting, and has provided better protection against bleeding compared to previous treatments.
Furthermore, the safety profile of etranacogene dezaparvovec remained favorable throughout the study duration. These findings, presented at the 18th Annual Congress of the European Association for Hemophilia and Allied Disorders, mark a significant milestone in the treatment of hemophilia B, offering hope for sustained management of the condition with a single treatment.
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