2020
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
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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