2023
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
Pipe S, Leebeek F, Recht M, Key N, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O’Connell N, Pasi K, Hart D, Kazmi R, Astermark J, Hermans C, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary S, Escobar M, Gomez E, Kruse-Jarres R, Quon D, Symington E, Wang M, Wheeler A, Gut R, Liu Y, Dolmetsch R, Cooper D, Li Y, Goldstein B, Monahan P. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. New England Journal Of Medicine 2023, 388: 706-718. PMID: 36812434, DOI: 10.1056/nejmoa2211644.Peer-Reviewed Original ResearchConceptsAnnualized bleeding rateLead-in periodAdeno-associated virus 5Factor IX prophylaxisGene therapyHemophilia BBleeding rateEtranacogene dezaparvovecFactor IX activityMonth 7Factor IX replacementPhase 3 studyIX activityNeutralizing antibody titersFactor IX concentrateOpen-labelRate ratiosSafety profileEfficacy measuresAdverse eventsPrevent bleedingNeutralizing antibodiesNoninferiority marginNoninferiority analysisNoninferiority
2014
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani A, Reiss U, Tuddenham E, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen Y, Halka K, Basner-Tschakarjan E, Mingozzi F, High K, Allay J, Kay M, Ng C, Zhou J, Cancio M, Morton C, Gray J, Srivastava D, Nienhuis A, Davidoff A. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B. New England Journal Of Medicine 2014, 371: 1994-2004. PMID: 25409372, PMCID: PMC4278802, DOI: 10.1056/nejmoa1407309.Peer-Reviewed Original ResearchConceptsSevere hemophilia BAdeno-associated virus serotype 8Factor IX levelsHigh-dose groupHemophilia BLong-term safetyGene therapyIX levelsSelf-complementary adeno-associated virus serotype 8Phase 1 dose-escalation trialDurability of transgene expressionHigh dosesTransgene expressionInfusion of vectorLate toxic effectsAlanine aminotransferase levelsDose-dependent increaseFollow-up periodDose-response relationshipCirculating factor IXFactor IX concentrateLate toxicityAAV8 vectorsBleeding episodesMedian period
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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