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 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
2005
A New Formulation of Recombinant Human Factor IX Is Bioequivalent to BeneFIX: A Double-Blind, Randomized, Crossover Pharmacokinetic and Open-Label Safety and Efficacy Study.
Powell J, Lambert T, Navarro F, Recht M, Sullivan S, Udata C, Lilly L, Roth D. A New Formulation of Recombinant Human Factor IX Is Bioequivalent to BeneFIX: A Double-Blind, Randomized, Crossover Pharmacokinetic and Open-Label Safety and Efficacy Study. Blood 2005, 106: 4076. DOI: 10.1182/blood.v106.11.4076.4076.Peer-Reviewed Original ResearchAdverse eventsDouble-blindLarger dosesOpen-label treatment periodClinically significant adverse eventsFIX inhibitor developmentAdministration of large dosesRecombinant factor IX concentrateSignificant adverse eventsOpen-label safetyRBC agglutinationRecombinant human factorPK parameter estimatesFactor IX concentrateOpen-labelRoutine prophylaxisSurgical prophylaxisBioequivalence intervalFactor IX activityThrombotic complicationsBolus infusionEfficacy profileHemophilia BBeneFIXPK characteristics
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