No More Injections After One-Off Gene Therapy in Hemophilia B

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No More Injections After One-Off Gene Therapy in Hemophilia B
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  • 📰 Medscape
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Patients with hemophilia B face a lifelong need for regular factor IX injections. But in 9 of 10 patients treated with a novel gene therapy, this need was eliminated.

Following the FLT180a infusion, all patients showed dose-dependent increases in factor IX levels. After a median follow-up of 27.2 months , nearly all the patients continued to show sustained factor IX activity.

The exception was one patient who required a return to factor IX prophylaxis. He had experienced a failure in the immunosuppression regimen due to a delay in the recognition of an immune response at approximately 22 weeks after treatment, the authors report. Otherwise, 24% of adverse events were determined to be related to the immunosuppression, and were consistent with the known safety profiles of glucocorticoids and tacrolimus.

The current findings, along with data from another recent study involving gene therapy for patients with, emphasize that"immune responses can occur later than previously expected and may coincide with the withdrawal of immunosuppression," the authors caution.

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