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. Sternberg^1,2, R.J Davidson^1,2, B.J Samelson-Jones^1,2,3, L.A George^1,2,3

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 Sawyer^6,7, S. Verweij^6,7, V. Colletta^6,7, D. Cooper^6,7, R. Dolmetsch^6,7, 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. Pipe^5,6

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 Sawyer^6,7, D. Cooper^6,7, V. Ferriera^6,7, S.W Pipe⁸, HOPE-B Investigators

Abstract Number: PB0654

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

Y. Chen^1,2, J. Schroeder¹, J. Li^1,2, 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’Mahony^15,16, R. Kaczmarek^17,18, 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