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 PMID: 40188458, 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 Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy
Reiss U, Davidoff A, Tuddenham E, Chowdary P, McIntosh J, Riddell A, Pie A, Batty P, Calvert J, Mangles S, Mahlangu J, Haley K, Recht M, Shen Y, Halka K, Zhou J, Kang G, Okhomina V, Morton C, Nathwani A. Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy. Blood 2023, 142: 1056. DOI: 10.1182/blood-2023-186891.Peer-Reviewed Original ResearchAnnualized bleeding rateSelf-complementary adeno-associated virusGene therapySevere hemophilia BFactor IXFactor IX activityAdverse eventsHemophilia BAdeno-associated virus gene therapyFollow-upTransient elevation of liver transaminasesLung adenocarcinoma in situMedian annualized bleeding rateElevation of liver transaminasesTreatment-related adverse eventsHigh vector dosesYears of follow-upSustained clinical benefitMedian Follow-UpAdenocarcinoma in situAdeno-associated virusFactor IX inhibitorsFirst-in-humanLiver-specific promoterDose-dependent increaseStable 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
2020
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Pipe S, Recht M, Key N, Leebeek F, Castaman G, Lattimore S, Van Der Valk P, Peerlinck K, Coppens M, O'Connell N, Pasi J, Kampmann P, Meijer K, von Drygalski A, Young G, Hermans C, Astermark J, Klamroth R, Lemons R, Visweshwar N, Crary S, Kazmi R, Symington E, Escobar M, Gomez E, Kruse-Jarres R, Kotowski A, Quon D, Wang M, Wheeler A, Sawyer E, Verweij S, Colletta V, Bajma N, Gut R, Miesbach W. First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies. Blood 2020, 136: lba-6. DOI: 10.1182/blood-2020-143560.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyCSL BehringHemophilia BAdverse eventsPersonal feesGene therapyFrequent treatment-related adverse eventsClinical studiesPre-existing neutralizing antibodiesTreatment-related adverse eventsLead-InGene therapy clinical studiesPre-existing NAbsAnnualized bleeding rateGene therapy trialsInfusion-related reactionsPhase 2b studyPhase 3 studyLead-in periodUse of corticosteroidsInfluenza-like illnessLiver-specific promoterEtranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
von Drygalski A, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gomez E, Gut R, Pipe S. Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial. Blood 2020, 136: 13. DOI: 10.1182/blood-2020-139295.Peer-Reviewed Original ResearchEntity's Board of DirectorsEtranacogene dezaparvovecInvestigational gene therapyLiver-specific promoterAMT-061Gene therapyHemophilia BCSL BehringAdverse eventsNormal rangeWeek 6FIX activity levelsRecords of bleedingYears of follow-upPhase 2b trialAmeliorate disease severityYears of follow-up dataWeeks post-dosingFollow-up dataMulti-center trialNational Hemophilia FoundationAdvisory CommitteeHemophilia B subjectsSustained FIX activityFixing activity
2019
One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B
Pipe S, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Gomez E, Long A, Gut R, von Drygalski A. One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B. Blood 2019, 134: 3348. DOI: 10.1182/blood-2019-128765.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Entity's Board of DirectorsAMT-061CSL BehringGene therapyAdverse eventsHemophilia BFollow-upWeek 6Weeks of follow-upFIX activity levelsInvestigational gene therapyRecords of bleedingYears of follow-upPhase 2b trialPhase 3 studyLiver-specific promoterMulti-center trialAdvisory CommitteeSpeakers bureauPatient-reported outcomesOpen-labelFixing activityLaboratory parametersPrimary endpointEtranacogene 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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