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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

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

A.R. Sternberg1,2, R.J Davidson1,2, B.J Samelson-Jones1,2,3, L.A George1,2,3

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

S.W Pipe1, F.W. Leebeek2, M. Recht3, N.S Key4, S. Lattimore3, G. Castaman5, E.K Sawyer6,7, S. Verweij6,7, V. Colletta6,7, D. Cooper6,7, R. Dolmetsch6,7, W. Miesbach8, HOPE-B Investigators

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

C.W. Rementer1, C. Li1, C.-Y. Chen1, C.H. Miao1

Abstract Number: PB0663

A Savvy Approach to Clinical Trial Recruitment for the SAAVY (Seroprevalence of AAV AntibodY) Study in the Era of COVID-19: Designing for a Prospective, Observational Study in the United States during a Global Pandemic

L.A. Valentino1, M. Vaghela2, M. Lauw2, G. Dela Cerda2, M. Jones2, D. Hinds2, V. Newman2, F. Leal-Pardinas2, D. Rotellini1, K. Schafer3, G. Pierce4, S. Pipe5,6

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

G. Nguyen1, E. Merricks2, T. Long2, K. Ponder3, T. Nichols2, D. Sabatino1

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

F.W. Leebeek1, W. Miesbach2, M. Recht3, N.S Key4, S. Lattimore3, G. Castaman5, E.K Sawyer6,7, D. Cooper6,7, V. Ferriera6,7, S.W Pipe8, HOPE-B Investigators

Abstract Number: PB0654

Clinical Translatable Preconditioning for Platelet Gene Therapy in Murine Hemophilia A with Inhibitors

Y. Chen1,2, J. Schroeder1, J. Li1,2, W. Jing1, J. Hu2, Q. Shi1

Abstract Number: PB0655

Development of Ordinary Differential Equation-based aPTT and Thrombin Generation Models for Characterizing FIX-Padua Gain of Function

T. Orfeo1, M. Gissel1, S.J. Everse1, E. Shehu2, R. Corbau2, J.H. Foley2

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

M.C Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, A.D Leavitt5, M. Laffan6, E. Symington7, D.V Quon8, J.-D. Wang9, K. Peerlinck10, S. Pipe11, B. Madan12, N.S Key13, G.F Pierce14, B. O’Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, W.Y. Wong19, B. Kim19, GENEr8-1 Trial Group

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

E. Gomez1, A. Giermasz2, G. Castaman3, N.S Key4, S.U Lattimore5, F.W. Leebeek6, W. Miesbach7, M. Recht5, A. von Drygalski8, E.K Sawyer9, S.W. Pipe10

Abstract Number: PB0652

Evolution of AAV Vector Gene Therapy is Ongoing In Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?

S.W. Pipe1, C. Hay2, J. Sheehan3, T. Lissitchkov4, M. Coppens5, H. Eichler6, S. Weigmann7, F. Ferrante8

Abstract Number: PB0660

Exigency: The Real-world Impact of Gene Therapy on the Lives of People with Haemophilia and their Families. An Interim Report

S. Fletcher1, S. Chaplin2, M. Holland2, K. Jenner2, K. Khair2

Abstract Number: LPB0019

Expression of Adenovirus-mediated Human Coagulation Factor IX Gene in SD Rat Adipose Mesenchymal Stem cells

Z. Yan1, Q. Sun1, Y. Xie1, L. Wang1, S. Chen1, W. Wu1, Y. Yan1, J. Yang1

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

W. Miesbach1, K. Meijer2, M. Coppens3, P. Kampmann4, R. Klamroth5, R. Schutgens6, G. Castaman7, E.K Sawyer8, F.WG Leebeek9

Abstract Number: LPB0022

Global Seroprevalence of Pre-existing Immunity against AAV Serotypes in People with Hemophilia A

R. Klamroth1, G. Hayes2, T. Andreeva3, T. Suzuki4, B. Hardesty5, M. Shima6, T. Pollock7, P. Slev7, J. Oldenburg8, M.C Ozelo9, S.-M. Castet10, J. Mahlangu11, F. Peyvandi12, R. Kazmi13, A.D Leavitt14, M. Callaghan15, B. Pan-Petesch16, D. Quon17, M. Li2, W.Y. Wong2

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

KJ. Pasi1, S. Rangarajan2, T.M Robinson3, W. Lester4, E. Symington5, B. Madan6, M. Laffan7, M. Li3, B. Kim3, G.F Pierce8, W.Y. Wong3

Abstract Number: OC 75.2

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

P. Batty1, B. Yates2, D. Hurlbut1, S. Siso-Llonch2, A. Menard3, H. Akeefe2, T. Christianson2, P. Colosi2, L. Harpell1, S. Liu2, A. Mo1, A. Pender1, G. Veres2, C. Vitelli2, A. Winterborn4, S. Bunting2, S. Fong2, D. Lillicrap1

Abstract Number: OC 67.4

Liver Safety Case Report from the Phase 3 HOPE-B Gene Therapy Trial in Adults with Hemophilia B

M. Schmidt1, G.R Foster2, M. Coppens3, H. Thomsen1, D. Cooper4, R. Dolmetsch4, E.K Sawyer4, L. Heijink5, S.W Pipe6

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

M. Recht1, F.W. Leebeek2, W. Miesbach3, N.S Key4, S. Lattimore1, G. Castaman5, E.K. Sawyer6, D. Cooper6, V. Colletta6, S.W. Pipe7, HOPE-B Investigators

Abstract Number: PB0657

Novel Hyperactive FIX Variants and their Potential for Hemophilia B Therapy

A.-K. Urbanowitz1, P. Milanov1, P. Quade-Lyssy1, D. Abriss1, E. Seifried1, J. Schüttrumpf2, J. Schwäble1

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