2025
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Monahan P, Le Quellec S, Pipe S. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Advances 2025, 9: 3543-3552. PMID: 40188458, PMCID: PMC12275190, DOI: 10.1182/bloodadvances.2024015291.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Etranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5FIX inhibitor developmentLiver-specific promoterPost-administrationSelf-administered infusionsSevere hemophilia BMulti-center trialYears post-administrationBleeding episodesOpen-labelSingle-doseSecondary endpointsIntravenous doseSingle-armGene therapyThrombotic complicationsYears post-treatmentSafety profileClinically significant elevationsAdverse eventsBleeding frequencyNeutralizing antibodies
2023
Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gut R, Dolmetsch R, Monahan P, Le Quellec S, Pipe S. Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy. Blood Advances 2023, 7: 5671-5679. PMID: 36490302, PMCID: PMC10539871, DOI: 10.1182/bloodadvances.2022008886.Peer-Reviewed Original ResearchConceptsAdeno-associated virus serotype 5Adverse eventsEtranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5Self-limiting adverse eventsFIX inhibitor developmentSecondary end pointsFIX replacement therapyLiver-specific promoterHemophilia B patientsSelf-administered infusionsSevere hemophilia BElective surgical proceduresAMT-061Open-labelB patientsBleeding episodesSingle-doseIntravenous doseMulticenter trialReplacement therapySingle-armGene therapyClinically significant elevations
2019
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B
Von Drygalski A, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Long A, Gut R, Sawyer E, Pipe S. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Advances 2019, 3: 3241-3247. PMID: 31698454, PMCID: PMC6855101, DOI: 10.1182/bloodadvances.2019000811.Peer-Reviewed Original ResearchEtranacogene dezaparvovecFactor IX replacementFactor IX activityAMT-061Factor IXBleeding cessationHemophilia BFactor IX replacement therapyPhase 2b studySecondary end pointsLevels of liver enzymesCessation of bleedingClinically relevant increasePhase 3 trialLiver-specific promoterSevere hemophilia BSevere hemophilia B.AAV5 vectorReplacement therapyInflammatory markersSingle doseClinically significant elevationsBleeding frequencyNeutralizing antibodiesBefore treatment
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