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 increase
2018
Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer
Nathwani A, Reiss U, Tuddenham E, Chowdary P, McIntosh J, Riddell A, Pie J, Mahlangu J, Recht M, Shen Y, Halka K, Meagher M, Nienhuis A, Davidoff A, Mangles S, Morton C, Junfang Z, Radulescu V. Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing “Empty Viral Particles” on Safety and Efficacy of Gene Transfer. Blood 2018, 132: 491. DOI: 10.1182/blood-2018-99-118334.Peer-Reviewed Original ResearchEntity's Board of DirectorsSelf-complementary adeno-associated virusVector preparationsFollow-upSevere HBDose cohortsHB patientsAAV-mediated transgene expressionEfficacy of gene transferGene transferLiver enzymesYear followHemophilia B (HBTransgene expressionLoss of transgene expressionFIX activity levelsPlasma FIX levelsSerotype 8 capsidAnnualized bleeding rateElevation of serum alanine aminotransferaseMedian Follow-UpAdeno-associated virusIncidence of hepatotoxicityB (HBRisk of hepatotoxicity
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