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BioWorld - Tuesday, March 3, 2026
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Home » ASH 2022: One-time infusion CSL’s Hemgenix gene therapy shows durable response for hemophilia B
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ASH 2022: One-time infusion CSL’s Hemgenix gene therapy shows durable response for hemophilia B

Dec. 13, 2022
By Tamra Sami
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding.
BioWorld Asia Clinical Conferences American Society of Hematology Hematologic Gene therapy

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