Feb 22, 2023 · Here, we report the safety and efficacy of etranacogene dezaparvovec from Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B), a phase 3 study of gene therapy for the treatment...

Background: Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic factor IX in terms of bleeding protection 18 months after gene therapy in a phase 3 trial.

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Mar 1, 2024 · The HOPE-B trial shows that etranacogene dezaparvovec can provide durable disease correction throughout 24 months after gene therapy in men with severe or moderately severe haemophilia B, including those with pre-existing AAV5 neutralising antibodies.

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Nov 15, 2022 · The Phase 3 HOPE-B clinical trial (NCT03569891) of etranacogene dezaparvovec met its primary efficacy endpoint, providing hemostatic protection superior to standard of care FIX prophylaxis over 52 weeks of follow-up after stable FIX Padua expression (defined as Months 7 …

Nov 5, 2024 · The present analysis explores the impact of sustained FIX Padua expression on the joint health of participants of the HOPE-B trial during the initial 36 months following etranacogene dezaparvovec administration.

HOPE-B is the first phase III trial for a gene therapy in hemophilia B, with the largest gene therapy cohort to date, Dr. Pipe noted. He presented data from 54 patients who had received etranacogene dezaparvovec and completed at least 26 weeks of follow-up.

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