ISTH 2021 Congress
Hemophilia and Rare Bleeding Disorders » Hemophilia Gene Therapy
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- ISTH 2021 Congress
- Hemophilia and Rare Bleeding Disorders
- Hemophilia Gene Therapy
- Hemophilia and Rare Bleeding Disorders
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Abstract Number: OC 75.3
In vitro and In vivo Models to Understand One-stage and Chromogenic Factor VIII Activity Assay Discrepancy of Hepatocyte-derived Factor VIII
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Abstract Number: PB0653
52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderate-severe Hemophilia B: Data from the Phase 3 HOPE-B Gene Therapy Trial
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Abstract Number: PB0650
A Novel Strategy for Platelet-specific Gene Therapy for the Treatment of Hemophilia A via Intranasal Delivery Lentiviral Vectors Containing Factor VIII
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Abstract Number: OC 75.4
AAV Delivery of a Novel Human Factor VIII Variant with Improved Secretion Results in Higher FVIII Expression in Hemophilia A Dogs
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Abstract Number: OC 67.3
Clinical Outcomes in Adults with Hemophilia B with and without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial
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Abstract Number: PB0654
Clinical Translatable Preconditioning for Platelet Gene Therapy in Murine Hemophilia A with Inhibitors
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Abstract Number: PB0655
Development of Ordinary Differential Equation-based aPTT and Thrombin Generation Models for Characterizing FIX-Padua Gain of Function
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Abstract Number: OC 26.1
Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial
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Abstract Number: LPB0020
Etranacogene Dezaparvovec (AAV5-Padua hFIX Variant, AMT-061), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-severe Hemophilia B: 2.5 Year Data from a Phase 2b Trial
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Abstract Number: PB0652
Evolution of AAV Vector Gene Therapy is Ongoing In Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?
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Abstract Number: PB0660
Exigency: The Real-world Impact of Gene Therapy on the Lives of People with Haemophilia and their Families. An Interim Report
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Abstract Number: LPB0019
Expression of Adenovirus-mediated Human Coagulation Factor IX Gene in SD Rat Adipose Mesenchymal Stem cells
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Abstract Number: OC 26.3
Five Year Data Confirms Stable FIX Expression and Sustained Reductions in Bleeding and Factor IX Use Following AMT-060 Gene Therapy in Adults with Severe or Moderate-severe Hemophilia B
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Abstract Number: LPB0022
Global Seroprevalence of Pre-existing Immunity against AAV Serotypes in People with Hemophilia A
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Abstract Number: OC 67.1
Hemostatic Response is Maintained for up to 5 Years Following Treatment with Valoctocogene Roxaparvovec, an AAV5-hFVIII-SQ Gene Therapy for Severe Hemophilia A
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Abstract Number: OC 75.2
Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model
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Abstract Number: OC 67.4
Liver Safety Case Report from the Phase 3 HOPE-B Gene Therapy Trial in Adults with Hemophilia B
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Abstract Number: PB0659
Management of Infusion Reactions: Lessons from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec in Adults with Hemophilia B
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Abstract Number: PB0657
Novel Hyperactive FIX Variants and their Potential for Hemophilia B Therapy