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 endpoint
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