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Hemophilia A

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Congress Posters and Presentations

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia Comprehensive Care Summit
  • 2025

Efficacy and safety of valoctocogene roxaparvovec for severe hemophilia A 5 years after gene transfer in GENEr8-1

Presenting Author: Johnny Mahlangu

A. D. Leavitt, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar, S. W. Pipe, S. Chou, R. Klamroth, J. Mason, H. Chambost, F. Peyvandi, E. Majerus, D. Pepperell, K. M. Chavele, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2025

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

A. D. Leavitt, J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar,, S. W. Pipe, B. Madan, S. Chou, R. Klamroth, J. Mason, H. Chambost, F. Peyvandi, H. Yu, T. M. Robinson, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2025

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

B. Madan, M. C. Ozelo, G. Kenet, S. Chou, S. W. Pipe, A. D. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Kordsachia, C. Rivat, J. Mahlangu

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2025

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

S. Santos, T. M. Robinson, S. Harris, D. Trueman

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2025

Final analysis of the phase 1/2 trial of valoctocogene roxaparvovec for severe hemophilia A

P. Raheja, S. Rangarajan, W. Lester, B. Madan, G. F. Pierce, E. Symington, C. Millar, D. Osmond, M. Li, K. Chavele

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2025

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

S. Santos, T. M. Robinson, S. Harris, D. Trueman

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Hematology
  • 2024

Valoctocogene roxaparvovec estimated long-term durability of treatment effect: An extrapolation of the most recent clinical data

S. Santos, T. M. Robinson, D. Trueman

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Poster

  • Congresses
  • Hemophilia A
  • The Professional Society for Health Economics and Outcomes Research Europe
  • 2024

Comparative EQ-5D utilities for valoctocogene roxaparvovec gene therapy and emicizumab in people with severe hemophilia A using a matching-adjusted indirect comparison

K. Taylor, T. Douglas, A. J. Hatswell, A. Clark, S. Santos, M. Karimi

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Poster

  • Congresses
  • Hemophilia A
  • The Professional Society for Health Economics and Outcomes Research Europe
  • 2024

Clinical and humanistic burden of people with severe haemophilia A treated with prophylaxis in Europe: a longitudinal analysis from the CHESS studies

E. Ferri Grazzi E, D. Hinds, S. Santos, Y. Zhang, T. Burke, T. Blenkiron

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Presentation

  • Congresses
  • Hemophilia A
  • Societa Italiana per lo Studio dell'Emostasi e della Trombosi
  • 2024

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Presenting Author: Flora Peyvandi

A. D. Leavitt, J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar, S. W. Pipe, B. Madan, S. Chou, R. Klamroth, J. Mason, H. Chambost, H. Yu, T. M. Robinson, M. C. Ozelo

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Presentation

  • Congresses
  • Hemophilia A
  • Societa Italiana per lo Studio dell'Emostasi e della Trombosi
  • 2024

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

Presenting Author: Giovanni di Minno

B. Madan, M. C. Ozelo, G. Kenet, S. C. Chou, S. W. Pipe, A. D. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu

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Presentation

  • Congresses
  • Hemophilia A
  • Societa Italiana per lo Studio dell'Emostasi e della Trombosi
  • 2024

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

Presenting Author: Giancarlo Castaman

B. Madan, E. Symington, S. Rangarajan, W. Lester, G. Pierce, P. Raheja, C. M. Millar, D. Osmond, M. Li, T. M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • Blood
  • 2024

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

D. Pepperell, B. Madan, M. Ozelo, G. Kenet, S-C. Chou, S. Pipe, A. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu

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Poster

  • Congresses
  • Hemophilia A
  • Blood
  • 2024

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

C. W. Tan, A. D. Leavitt, J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. M. Millar, S. W. Pipe, B. Madan, S-C. Chou, R. Klamroth, J. Mason, H. Chambost, F. Peyvandi, H. Yu, T. M. Robinson, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Blood
  • 2024

Human liver biopsy analysis shows decline in FVIII levels following AAV5-hFVIII-SQ gene therapy may be due to low RNA transcription levels despite persistence of full-length episomal vector genomes

J. Mason, B. Yates, M. I. Ashrafali, A. Mattis, A. Gonzalez, B. Kim, K. Jayaram, G. Kenet, J. Mahlangu, A. Dunn, S. Shapiro, M. Wang, F. Peyvandi, A Giermasz, R. Kazmi, N. Key, M. Ozelo,  T. Robinson, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular
  • 2024

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

F. P. Careta, B. Madan, M. C. Ozelo, G. Kenet, S. Chou, S. W. Pipe, A. D. Leavitt, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu

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Poster

  • Congresses
  • Hemophilia A
  • Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular
  • 2024

Bleeding rate, FVIII utilization, corticosteroid use, quality of life and safety 4 years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort: Results from GENEr8-1

G. G. Yamaguti-Hayakawa, M. Ozelo, A. Prezotti, M. Cerqueira, P. Villaça, C. Lorenzato, T. Robinson 

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Poster

  • Congresses
  • Hemophilia A
  • Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular
  • 2024

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

M. C. Ozelo, A. D. Leavitt, J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar, S. W. Pipe, B. Madan, S. Chou, R. Klamroth, J. Mason, H. Chambost, F. Peyvandi, H. Yu, T. M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • National Bleeding Disorders Foundation
  • 2024

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

B. Madan, M. C. Ozelo, G. Kenet, S-C. Chou, S. W. Pipe, A. D. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu

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Poster

  • Congresses
  • Hemophilia A
  • National Bleeding Disorders Foundation
  • 2024

How long do the effects of valoctocogene roxaparvovec last? A plain language summary of a model estimating how long patients with hemophilia A may experience benefit

S. Santos, T. M. Robinson, F. Rico, D. Trueman, S. Jeffrey

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Poster

  • Congresses
  • Hemophilia A
  • National Bleeding Disorders Foundation
  • 2024

Plain language summary of 4-year efficacy and safety data in the GENEr8-1 trial of valoctocogene roxaparvovec, a gene therapy for severe hemophilia A

A. D. Leavitt, J. Mahlangu, E. Symington, D. V. Quon, A. Giermasz, N. S. Key, S. W. Pipe, F. P. Careta, P. J. Lynch, A. Ruiz, H. Yu, C. Rivat, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Congrès Français d’Hémostase
  • 2024

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

A. Rauch, H. Chambost, A. D. Leavitt, J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar, S. W. Pipe,
B. Madan, S-C. Chou, R. Klamroth, J. Mason, F. Peyvandi, H. Yu, T. M. Robinson, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Congrès Français d’Hémostase
  • 2024

Health-related quality-of-life outcomes 4 years after treatment with valoctocogene roxaparvovec

A. Rauch, B. Madan, G. Kenet, S. C. Chou, S. W.  Pipe, A. D. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Efficacy, safety and quality of life three years after gene transfer with valoctocogene roxaparvovec in a Brazilian cohort

M. C. Ozelo, G. G. Yamaguti-Hayakawa, A. N. L. Prezotti, M. H. Cerqueira, P. R. Villaça, C. Lorenzatto, T. M. Robinson

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Health-related quality of life outcomes 4 years after treatment with valoctocogene roxaparvovec

Presenting Author: Bella Madan

M. Ozelo, G. Kenet, S. C. Chou, S. Pipe, A. Leavitt, F. P. Careta, P. J. Lynch, S. Fletcher, D. Goshen, A. Ruiz, E. Dashiell-Aje, C. Rivat, J. Mahlangu

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Efficacy and safety of valoctocogene roxaparvovec 4 years after gene transfer in GENEr8-1

Presenting Author: Andrew D. Leavitt

J. Mahlangu, P. Raheja, E. Symington, D. V. Quon, A. Giermasz, G. Kenet, G. Lowe, N. S. Key, C. M. Millar, S. W. Pipe, B. Madan, S. C. Chou, R. Klamroth, J. Mason, H. Chambost, F. Peyvandi, H. Yu, T. M. Robinson, M. C. Ozelo

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for hemophilia A

Presenting Author: Bella Madan

E. Symington, S. Rangarjan, W. Lester, G. F. Pierce, P. Raheja, C. Millar, D. Osmond, M. Li, T. M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Results from the Taiwanese cohort in GENEr8-1: bleeding rates, factor VIII usage, quality of life and safety in severe hemophilia A patients after gene therapy

S. C. Chou, S. S. Chiou, Y. C. Chen, S. Santos, T. M. Robison, M. C. Shen

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Persistent and stable therapeutic levels of transgenic FVIII expression following AAV delivery to adult and infant hemophilic dogs

P. Batty, A. M. Ismail, B. Yates, L. Swystun, A. Mo, L. Harpell, A. Pender, A. Menard, A. Winterborn, S. Salcido, A. Gonzalez, C. K. Kim, S. Brennon, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2024

Kinetic Profiles of anti-AAV5 Antibody Generation and Clearance in Neonatal, Infant, and Adult Hemophilia A Dogs

Presenting Author: Paul Batty

B. Handyside, L. Swystun, A. Mo, L. Harpell, A. Pender, A. Winterborn, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2024

Perceptions, needs and expectations of patients with hemophilia toward gene therapy

Presenting Author: Laurent Frenzel

C. Chenuel, A. Celli, N. Grinda, R. Varin, N. Giraud, G. Pietu, T. Backus, C. Schepperle, A. Lupi, K. Legendre, S. Njegic

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2024

Human liver biopsy analysis shows decline in FVIII levels following AAV5-hFVIII-SQ gene therapy may be due to low RNA transcription levels despite persistence of full-length episomal vector genomes

Presenting Author: Bridget Yates

A. M. Ismail, A. N. Mattis, A. Gonzalez, C. K. Kim, K. Jayaram, G. Kenet, J. Mason, J. Mahlangu, A. L. Dunn, S. Shapiro, M. Wang, F. Peyvandi, A. Giermasz, R. Kazmi, N. S. Key, M. Ozelo, T. M. Robinson, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2024

Gene Therapy for Hemophilia Clearing House: a single repository of resources for hemophilia

B. O’Mahony, T. Becker, C. Betsch, A. Buzzi, D. Coffin, E. F. Grazzi, D. García Diego, N. Giraud, A. G. Cavallini, D. Goshen, B. Hayes, D. Page, D. Rotellini, C. Smith, J. Liu, S. Njegic

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2024

Health-related quality of life improvements following valoctocogene roxaparvovec gene therapy in people without bleeds or target joints at baseline: a post hoc analysis from GENEr8-1

M. Karimi, S. Bourke, M. Little, I. A. Soare, F. Becker, S. Santos

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2024

Patients’ personal insights following agene therapy clinical trial for hemophilia A

S. Galili, S. Lalezari, G. Kenet

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Poster

  • Congresses
  • Hemophilia A
  • Thrombosis and Hemostasis Summit of North America
  • 2024

Seroprevalence and seroconversion among people with Hemophilia A in the United States: Observations from the SAAVY (Seroprevalence of AAV Antibody) study

A. Shapiro, L. A. Valentino, D. Hinds, M. Vaghela, E. Goodman, K. Iskander, G. D. Cerda, K. Schafer, S. Pipe

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Presentation

  • Congresses
  • Hemophilia A
  • Thrombosis and Hemostasis Summit of North America
  • 2024

Safety and efficacy of valoctocogene roxaparvovec gene transfer for severe hemophilia A: an update from 4 years after treatment

Presenting Author: Doris V. Quon
A. D. Leavitt, J. Mahlangu, E. Symington, A. Giermasz, N. S. Key, S. W. Pipe, B. Madan, S. C. Chou, R. Klamroth, J. Mason, F. Peyvandi, H. Yu, T. M. Robinson, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

Characterization of pre-existing immunity to AAV5

K. Lau, C. Vettermann, G. de Hart, M. B. Hock, S. Frazer-Abel, B. Long, S. Gupta

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Presentation

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

Early changes in liver transcriptomic profiles following adeno-associated viral gene therapy in the severe hemophilia A dog model

Presenting Author: Simon Harris

A.M. Ismail, P. Batty, A. Mo, B. Yates, L. Harpell, A. Menard, A. Pender, N.A. Khattak, L. Razon, A. Winterborn, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

Immunoadsorption Plasmapheresis for the Removal of Plasma Immunoglobulins to Enable Repeat Dose Administration with an AAV5 Gene Therapy Vector

Presenting Author: Brian R Long

M. B. Hock, C. A. O’Neill, J. Arens, T. Seitel, L. Crockett, F. Relouzat, C. Fovet, N. Dereuddre Bosquet, P. Maisonnasse, H. Letscher, C. Bouillie, R. Le Grand, C. Vettermann, S. Gupta

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Presentation

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

Exploring actionable strategies to improve AAV5-hFVIII-SQ durability and optimize gene expression

Presenting Author: Simon Harris

B. Yates, D. J. Groeneveld, S, Scheeler, B. Handyside, I. Villalpando, S. Agarwal, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

A simulation study to provide guidance for individuals transitioning from emicizumab to valoctocogene roxaparvovec

S. Agarwal, C. Hermans, W. Miesbach, S. Pipe, R. Sidonio Jr, D. Osmond, V. Newman, J. Henshaw, F. Peyvandi

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Poster

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2024

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/dL Post-Gene Transfer in GENEr8-1

J. Mahlangu, M. Ozelo, C. W. Tan, A. Giermasz, J. D. Wang, S. Harris, D. Osmond, H. Yu, T. M. Robinson

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Presentation

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Safety and efficacy of valoctocogene roxaparvovec with prophylactic corticosteroids: 1-year GENEr8-3 results

Presenting Author: Margareth C. Ozelo

J. Mason, A. L. Dunn, P. R. Villaça, M. Shen, S. K. Agarwal, U. Imtiaz, H. Liu, T. M. Robinson

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Presentation

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Safety and efficacy of valoctocogene roxaparvovec in participants with active and prior FVIII inhibitors: Preliminary results from GENEr8-INH, a phase 1/2 study

Presenting Author: Guy Young

Y. Park, M. C. Ozelo, S. Chou, M. Li, U. Imtiaz, K. Chavele

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Presentation

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Human liver biopsy analysis reveals lower RNA transcription may contribute to a decline in FVIII levels following AAV5- hFVIII-SQ gene therapy

Presenting Author: Ashrafali M. Ismail, PhD

B. Yates, K. Jayaram, G. Kenet, J. Mason, J. Mahlangu, A. L. Dunn, S. Shapiro, M. Wang, F. Peyvandi, A. Giermasz, R. Kazmi, N. S. Key, T. M. Robinson, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Treatment preferences in people with severe haemophilia A: a discrete choice experiment in the United States

W. Miesbach, L. A. Valentino, B. O’Mahony, K. Forsyth, M. Bullinger, J. M. Gonzalez, E. Dashiell-Aje, S. Santos, H. B. Lewis, D. Latibeaudiere-Gardner, T. Tervonen, M. Karimi

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Health-related quality of life and long-term joint damage in people with severe haemophilia A in Brazil

J. Evans, T. Burke

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2024

Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A

E. Symington, S. Rangarajan, W. Lester, B. Madan, G. F. Pierce, P. Raheja, C. Millar, D. Osmond, M. Li, T. M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • Blood
  • 2023

Bleeding, FVIII activity, and safety 3 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

C. W. Tan, J. Mahlangu, A. von Drygalski, S. Shapiro, S. Chou, M. C. Ozelo, G. Kenet, F. Peyvandi, B. Madan, M. Laffan, A. L. Dunn, J. Mason, D. V. Quon, A. D. Leavitt, J. Oldenburg, H. Chambost, M. T. Reding, K. Jayaram, H. Yu, T. M. Robinson, S. W. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Hematology
  • 2023

Bleeding Outcomes in Participants With Factor VIII Activity <5 IU/dL Post-Gene Transfer in GENEr8-1

J. Mahlangu, M. C. Ozelo, C. W. Tan, A. Giermasz, J. Wang, D. Osmond, H. Yu, T. M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Hematology
  • 2023

Childhood treatment with Adeno-Associated Viral gene therapy results in stable FVIII expression and improved bleeding phenotype in adult severe hemophilia A dogs

P. Batty, A. Menard, A. Mo, C. Brown, A. M. Ismail, B. Yates, L. Swystun, L. Harpell, A. Pender, A. Winterborn, S. Fong, D. Lillicrap

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Poster

  • Congresses
  • Hemophilia A
  • Blood
  • 2023

Quantitative pharmacokinetic model to characterize and extrapolate long-term FVIII activity levels in patients with severe hemophilia A treated with valoctocogene roxaparvovec

S. Wong, S. Agarwal, A. Tiede, T.M. Robinson, J. Henshaw

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Presentation

  • Congresses
  • Hemophilia A
  • NordCoag
  • 2023

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

Presenting Author: Simon Harris

D. V. Quon, J. Wang, M. Wang, D. Pepperell, Y. S. Park, G. Kenet, J. Mahlangu, T. Khoo, T. M. Robinson, K. Chavele, S. W. Pipe

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Presentation

  • Congresses
  • Hemophilia A
  • NordCoag
  • 2023

A simulation study to provide guidance for individuals transitioning from emicizumab to valoctocogene roxaparvovec

Presenting Author: Simon Harris

S. Agarwal, C. Hermans, W. Miesbach, S. Pipe, R. Sidonio Jr., D. Osmond, V. Newman, J. Henshaw, F. Peyvandi

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Presentation

  • Congresses
  • Hemophilia A
  • NordCoag
  • 2023

Exploring actionable strategies to improve AAV5-hFVIII-SQ durability and optimize gene expression

Presenting Author: Bridget Yates

D. J. Groeneveld, S. Scheeler, B. Handyside, I. Villalpando, S. Agarwal, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • Academy of Managed Care Pharmacy Nexus
  • 2023

A novel analysis of hemophilia treatment administration on patient utility: Combining a discrete choice experiment (DCE) with time trade-off (TTO) estimation

M. Benton, D. Hong, M. W. Skinner, L. P. Garrison, M. Karimi, E. Chen, H. Mead, L. Shi

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Poster

  • Congresses
  • Hemophilia A
  • National Hemophilia Foundation
  • 2023

Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE

M. W. Skinner, E. Chen, A. Kucher, F. Germini, M. Karimi, E. Clearfield, B. O’Mahony, M. Jain

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Poster

  • Congresses
  • Hemophilia A
  • National Hemophilia Foundation
  • 2023

Gene therapy in hemophilia A: The impact of valoctocogene roxaparvovec on patient outcomes – initial results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 trial

M. W. Skinner, E. Chen, Q. Ibrahim, A. Kucher, F. Germini, M. Karimi, E. Clearfield, B. O’Mahony, M. Jain

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Poster

  • Congresses
  • Hemophilia A
  • National Hemophilia Foundation
  • 2023

Effects of valoctocogene roxaparvovec gene therapy in people with severe hemophilia A after 3 years

G. Lowe, P. J. Lynch, J. Mahlangu, M. C. Ozelo, B. O’Mahony, T. M. Robinson, H. Yu, L. A. Valentino

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2023

Understanding the requirement for additional Factor VIII infusion associated with novel haemophilia A treatments: An expert elicitation exercise

E. F. Grazzi, S. Santos, T. Burke

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2023

Quantitative pharmacokinetic model to characterize and extrapolate long-term FVIII activity levels in patients with severe hemophilia A treated with valactocogene roxaparvovec

S.K. Agarwal, P. Chityala, A. Tiede, T.M. Robinson, J. Henshaw

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2023

Stable Factor VIII Expression and Improvement in Bleeding Phenotype Following Early Childhood Treatment with Adeno-Associated Viral Gene Therapy in the Severe Hemophilia A Dog Model

P. Batty, A. Menard, A. Mo, Brown C., A.M. Ismail, B. Yates, L. Harpell, A.Pender, A. Winterborn, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2023

Bleeding, FVIII activity, and safety 3 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Presenting Author: Johnny Mahlangu

A. von Drygalski, S. Shapiro, S. Chou, M. C. Ozelo, G. Kenet, F. Peyvandi, B. Madan, M. Laffan, A. L. Dunn, J. Mason, D. V. Quon, A. D. Leavitt, J. Oldenburg, H. Chambost, M. T. Reding, K. Jayaram, H. Yu, T. M. Robinson, S. W. Pipe

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2023

Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial

M. W. Skinner, E. Chen, Q. Ibrahim, A. Kucher, F. Germini, M. Karimi, E. Clearfield, B. O’Mahony, M. Jain

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2023

Immunoadsorption Plasmapheresis for the Removal of Plasma Immunoglobulins to Enable Repeat Dose Administration with an AAV5 Gene Therapy Vector

B. R. Long, B. M. Hock, C. A. O’Neill, J. Arens, T. Seitel, L. Crockett, F. Relouzat, C. M. Fovet, N. Dereuddre-Bosquet, P. Maisonnasse, H. Letscher, R. Le Grand, C. Vettermann and S. Gupta

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2023

Early changes in liver transcriptomic profiles following adeno-associated viral gene therapy in the severe hemophilia A dog model

A. M. Ismail, P. Batty, A. Mo, B. Yates, L. Harpell, A. Menard, A. Pender, N. A. Khattak, L. Razon, A. Winterborn, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2023

The longitudinal kinetics of AAV5 vector integration profiles in mice

Presenting Author: A. M. Ismail
E. Witt, T. Bouwman, W. Clark, B. Yates, M. Franco, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2023

Characterization of pre-existing immunity to AAV5

Lau K, Vettermann C, de Hart G, Hock MB, Frazer-Abel S, Long B, Gupta S

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia Comprehensive Care Summit
  • 2023

Musculoskeletal health after gene therapy for Hemophilia A: a three-year follow-up study

Presenting Author: G. G. Yamaguti-Hayakawa

J. B. da S. Ricciardi, J. O. Frade-Guanaes, V. B. Faiotto, L. G. R. de Lima, G. Feldberg, E. T. I. Sakuma, D. M. de Paulo, A. L. A. Sambo, M. M. T. Hosokawa, V. Stahl, S. S. Medina, M. P. Colella, N. M. Foschi, C. Costa-Lima, M. C. Ozelo

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia Comprehensive Care Summit
  • 2023

Association of Work and Activity Impairment with Clinical Outcomes in Patients with Haemophilia A: A Multivariate Analysis of Data from the CHESS II Study

T. Blenkiron, C. Hawes, C. Camp, S. Santos, T. Burke, E. F. Grazzi

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Poster

  • Congresses
  • Hemophilia A
  • Congrès Français d’Hémostase
  • 2023

The effect of prophylactic corticosteroid treatment on adeno-associated virus mediated gene therapy and potential mechanisms of action

S. Harris, B. Handyside, L. Zhang, B. Yates, L. Xie, C. R. Sihn, R. Murphy, T. Bouwman, B. Baridon, C. Su, S. Bullens, A. M. Ismail, S. Bunting, S. Fong

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Presentation

  • Congresses
  • Hemophilia A
  • Gesellschaft für Thrombose- und Hämostaseforschung e.V.
  • 2023

Efficacy and safety of valoctocogene roxaparvovec gene transfer for severe hemophilia A: Results from the GENEr8 1 three year analysis

Presenting Author: Johnny Mahlangu

A. von Drygalski, S. Shapiro, S. Chieh Chou, M. C. Ozelo, G. Kenet, F. Peyvandi, B. Madan, M. Laffan, A. L. Dunn, J. Mason, D. V. Quon, A. D. Leavitt, J. Oldenburg, H. Chambost, M. T. Reding, K. Jayaram, H. Yu, T. M. Robinson, S. W. Pipe for the GENEr8 1 Trial Group

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2023

A Preliminary Analysis of Hemophilia Patient Utility Study of Treatment Administration Impact: A Discrete Choice Experiment (DCE) using Time Trade-Off (TTO) Methodology

M. Benton, M. W. Skinner, L. P. Garrison, E. Chen, H. Mead, L. Shi

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Presentation

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2023

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

Presenting Author: Doris V Quon, MD, PhD

 J. Wang, M. Wang, D. Pepperell, Y. S. Park, G. Kenet, J. Mahlangu, T. Khoo, T. M. Robinson, K. Chavele, S. W. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • The Professional Society for Health Economics and Outcomes Research Europe
  • 2022

Treatment Burden and Its Relationship with Health-Related Quality of Life, Work Productivity and Activity Impairment in Adults with Severe Non-Inhibitor Hemophilia A in the United States: Data Analysis from the CHESS US+ Study

E. F. Grazzi, T. Blenkiron, M. J. Aragon, J. O’Hara, E. Chen, D. Hinds, T. Burke

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Poster

  • Congresses
  • Hemophilia A
  • American Thrombosis and Hemostasis Network Data Summit
  • 2022

Range of Motion Data Collected in the ATHN dataset: Completeness, Variability, and Temporality

N. Daoud, C. Hawes, T. W. Buckner, S. Funk, D. Hinds, M. Recht

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Presentation

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Molecular Characterization of Recombinant AAV5 encoding FVIII after Human administration

Presenting Author: Kevin Eggan, PhD

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Presentation

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Vector genome loss and epigenetic modifications impact long-term transgene expression of AAV5 vectors produced in mammalian HEK293 and insect Sf cells

Presenting Author: B. Yates

A. M. Ismail, B. Handyside, L. Zhang, L. Xie, C. R. Sihn, R. Murphy, T. Bouwman, C. K. Kim, S. Bullens, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Methylation of rAAV vector DNA is not a mechanism for differential transgene RNA expression from rAAV gene therapy in mouse and NHP liver

K. O. Moss, M. Vora, C. Russell

 

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Poster

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Investigating mechanisms of variability of AAV5-hFVIII-SQ expression in vitro

B. Yates, W. Keenan, L. Razon, S. Scheeler, S. Liu, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Differential accessibility of chromatinised rAAV vector is a major regulator of variable expression in mouse and NHP liver

K. O. Moss, M. Vora, C. Russell

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Poster

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Prophylactic corticosteroid treatment in mice improved AAV5 transgene expression through multiple mechanisms

B. Handyside, B. Yates, L. Zhang, A. M. Ismail, L. Xie, C. R. Sihn, R. Murphy, T. Bouwman, S. Salcido, B. Keenan, S. Scheeler, B. Baridon, C. Su, S. Bullens, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • European Society of Gene & Cell Therapy
  • 2022

Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

B. Yates, L. Zhang, R. Murphy, S. Liu, L. Xie, B. Handyside, C. R. Sihn, D. Tan, J. Arens, J. Holcomb, J. Henshaw, C. Vettermann, C. Su, S. Bullens, S. Bunting, C. A. O’Neill, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • Academy of Managed Care Pharmacy Nexus
  • 2022

Impact of Gene Therapy on the Economic Burden of Adults with Severe Hemophilia A Managed with Prophylaxis in the United States

T. Wang, E. Chen, C. Regan, K. Cook, N. Kirson, E. Pezalla, M. W. Skinner

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Presentation

  • Congresses
  • Hemophilia A
  • Immunogenicity & Bioassay Summit
  • 2022

Non-Clinical Pharmacodynamic Effects and Immunogenicity Assessment ff Prophylactic Immune Modulation Prior to Gene Therapy Dose Administration in C57BL/6 Mice.

Presenting Author: Brian Long

B. Handyside, T. Seitel, R. Boyer, C. Wilson, K. O. Moss, A. Van Tuyl, J. Arens, K. Lau and S. Gupta.

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Comparability of bleeding outcomes by prophylactic FVIII replacement intensity: A post hoc analysis of a noninterventional study of men with severe hemophilia A

C. Camp, C. Hawes, D. Hinds, D. B. Reddy, V. Newman, E. Goodman

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring

H. Liu, C. Hawes, C. Hsu, P. You, X. Yang, V. Newman, T. M. Robinson, A. J. Hatswell, D. Hinds

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A Results from the Phase 3 GENEr8-1 Trial

S. Agarwal, K. Sandza, K. Obrochta, M. Vora, A. Bowen, B. Bunch, J. Holcomb, T. M. Robinson, K. Jayaram, C. B. Russell, C. Vettermann, J. Henshaw

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Innate and Adaptive Immune Responses to Adeno associated viral Gene Therapy in the Severe Hemophilia A Dog Model

P. Batty, A. Mo, A.M. Ismail, B. Yates, A. Gonzalez, B. Handyside, L. Harpell, D. Hurlbut, A. Menard, A. Pender, L. Razon, C.R. Sihn, A. Winterborn, S. Fong, D. Lillicrap

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5 hFVIII SQ gene therapy for severe hemophilia A

M. A. Laffan, S. Rangarajan, W. Lester, E. Symington, B. Madan, D. P. Hart, M. Li, T. M. Robinson, G. F. Pierce, W. Y. Wong

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2022

Relationship between transgene produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

J. Mahlangu, H. Chambost, S. C. Chou, A. Dunn, A. von Drygalski, R. Kaczmarek, G. Kenet, M. Laffan, A. D. Leavitt, B. Madan, J. Mason, J. Oldenburg, M. C. Ozelo, F. Peyvandi, D. V. Quon, M. T. Reding, S. Shapiro, H. Yu, T. M. Robinson, S. W. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

Interim 52-Week Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in GENEr8-1, a Phase 3 Clinical Study of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

B. Long, T. Robinson, J. Day, H. Yu, K. Lau, K. Patton, G. de Hart, J. Henshaw, S. Agarwal, C. Vettermann, S. Gupta

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

Use of immunosuppressives in patients with hemophilia receiving gene therapy: Evidence generation using a mixed-methods approach

W. Miesbach, D. Lillicrap, V. Newman, M. Jones, C. E. Parker, M. Ladouceur, G. Foster, S. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

Immune Suppression following Gene Therapy in Hemophilia

W. Miesbach

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

Health-Related Quality of Life Over 2 Years Following Valoctocogene Roxaparvovec Adeno-Associated Virus Gene Transfer For Severe Hemophilia A: Results From GENEr8-1

Presenting Author: Amy Dunn

A. Leavitt, F. Peyvandi, H. Chambost, E. Cockrell, R. Kazmi, R. Klamroth, G. Lowe, J. Mahlangu, E. Majerus, B. O’Mahony, M. C. Ozelo, M. Skinner, C. Tan, H. Tran, J. Wang, H. Yu, T. Robinson, J. Quinn, W.Y. Wong, S. Pipe

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • World Federation of Hemophilia Comprehensive Care Summit
  • 2022

Immune Suppression following Gene Therapy in Hemophilia

Presenting Author: W. Miesbach

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

Interim 52-Week Analysis of Immunogenicity to the Vector Capsid and Transgene-Expressed Human FVIII in GENEr8-1, a Phase 3 Clinical Study of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A

Presenting Author: Brian Long

T. Robinson, J. Day, H. Yu, K. Lau, K. Patton, G. de Hart, J. Henshaw, S. Agarwal, C. Vettermann, S. Gupta

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2022

Bioprocess Strategies to Mitigate the Impact of Proteases on Adeno-Associated Viral (AAV) Vector VP1 Capsid Content

J. Aponte-Ubillus, S. Pande, C. Lee, M. Hwu, J. Peltier

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2022

The Effect of Prophylactic Corticosteroid Treatment on Adeno-Associated Virus-Mediated Gene Therapy and Potential Mechanisms of Action

B. Handyside, L. Zhang, B. Yates, L. Xie, C.R. Sihn, R. Murphy, T. Bouwman, B. Baridon, K. Ngo, C. Su, P. Colosi, S. Bullens, A. M. Ismail, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2022

Impact of genome accessibility and long-term expression of adeno-associated virus 5 produced in mammalian (HEK293) and insect (Sf) cell lines

A. Ismail, B. Handyside, L. Zhang, B. Yates, L. Xie, C. Sihn, R. Murphy, T. Bouwman, K. Ngo, J. Woloszynek, P. Colosi, S. Bullens, S. Bunting, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2022

Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood

B. Yates, L. Zhang, R. Murphy, S. Liu, L. Xie, B. Handyside, C. R. Sihn, D. Tan, J. Arens, J. Holcomb, J. Henshaw, C. Vettermann, C. Su, S. Bullens, S. Bunting, C. A. O’Neill, S. Fong

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Poster

  • Congresses
  • Hemophilia A
  • American Society of Gene and Cell Therapy
  • 2022

Characterization of Residual DNA in rAAV Made in the Baculovirus/Sf9 Platform

D. Barajas, T. Cinek

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Presentation

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2022

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: Results from the GENEr8-1 year two analysis

Presenting Author: Johnny Mahlangu

M. C. Ozelo, F. Peyvandi, K. Jayaram, H. Yu,T.M. Robinson, W.Y. Wong, and S.W. Pipe on behalf of the GENEr8-1 Trial Group

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2022

Valoctocogene Roxaparvovec Gene Transfer in Participants with HIV

E. Majerus, S. Fong, B. Yates, S. Scheeler, L. Razon, S. Liu, H. Yu, D.B. Reddy, T.M. Robinson

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2022

Qualitative Research Evaluating Patient Preference for Haemophilia Therapy

Presenting Author: Wolfgang Miesbach

L.A. Valentino, D. Noone, K. Forsyth, M. Bullinger, E. Dashiell-Aje, V. Newman, C. Hawes, S. Hawley, H.B. Lewis, D. Latibeaudiere-Gardner, J. Quinn

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Poster

  • Congresses
  • Hemophilia A
  • European Association for Haemophilia and Allied Disorders
  • 2022

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Haemophilia A in the Phase 3 Trial GENEr8-1

Presenting Author: Brian O’Mahony

J. Mahlangu, K. Peerlinck, J.D. Wang, G. Lowe, C.W. Tan, H. Tran, T.L. Khoo, E. Cockrell, D. Pepperell, H. Chambost, M.F. López Fernández, R. Kazmi, E. Majerus, M.W. Skinner, R. Klamroth, J. Quinn, H. Yu,
W.Y. Wong, A. Lawal, T.M. Robinson, B. Kim

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Poster

  • Congresses
  • Hemophilia A
  • Congrès Français d’Hémostase
  • 2023

Endogenous FVIII activity and procedure-related FVIII use and bleeding: Post hoc analysis of GENEr8-1

A. Rauch, D. V. Quon, J. D. Wang, M. Wang, D. Pepperell, Y. S. Park, G. Kenet, J. Mahlangu, T. L. Khoo, T. M. Robinson, K. M. Chavele, S. W. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • Congrès Français d’Hémostase
  • 2023

Non-clinical pharmacodynamic effects and immunogenicity assessment of prophylactic immune modulation prior to gene therapy dose administration in C57BL/6 mice

S. Harris, B. Long, B. Handyside, T. Seitel, R. Boyer, C. Wilson, K. O. Moss, A. Van Tuyl, J. Arens, K. Lau, S. Gupta

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Efficacy and safety of valoctocogene roxaparvovec adeno-associated virus gene transfer for severe hemophilia A: Results from the Phase 3 GENEr8-1 Trial

Presenting Author: Margareth C. Ozelo

J. Mahlangu, K. J. Pasi, A. Giermasz, A. D. Leavitt, M. Laffan, E. Symington, D. V. Quon, J. D. Wang, K. Peerlinck, S. Pipe, B. Madan, N. S. Key, G. F. Pierce, B. O’Mahony, R. Kaczmarek, A. Lawal, M. Huang, W. Y. Wong, B. Kim

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Hemostatic response is maintained for up to 5 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

Presenting Author: Michael Laffan

K. J. Pasi, S. Rangarajan, T. M. Robinson, W. Lester, E. Symington, B. Madan, M. Li, B. Kim, G. F. Pierce, W. Y. Wong

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Presentation

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

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

Presenting Author: Paul Batty

B. Yates, D. Hurlbut, S. Siso-Llonch, A. Menard, H. Akeefe, T. Christianson, P. Colosi, L. Harpell, S. Liu, A. Mo, A. Pender, G. Veres, C. Vitelli, A. Winterborn, S. Bunting, S. Fong, D. Lillicrap

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Exploring the level of congruence between patient- and haematologist-reporting of anxiety and depression in people with haemophilia A

Presenting Author: Charles Hawes

T. Burke, A. Shaikh, G. Pedra, C. Hawes, C. Camp, J. O’Hara

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

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

Presenting Author: Manil Vaghela

L. A. Valentino, M. Vaghela, M. Lauw, G. Dela Cerda, M. Jones, D. Hinds, V. Newman, F. Leal-Pardinas, D. Rotellini, K. Schafer, S. Pipe

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Global seroprevalence of pre-existing immunity against AAV serotypes in people with hemophilia A

Presenting Author: Robert Klamroth

R. Klamroth, G. Hayes, T. Andreeva, T. Suzuki, B. Hardesty, M. Shima, T. Pollock, P. Slev, J. Oldenburg, M. C. Ozelo, S.-M. Castet, J. Mahlangu, F. Peyvandi, R. Kazmi, A. D. Leavitt, M. Callaghan, B. Pan-Petesch, D. Quon, M. Li, W. Y. Wong

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Examination and validation of a patient-centric joint metric, the “PROBLEM JOINT”: Empirical evidence from the CHESS Paediatrics dataset

Presenting Author: Idaira Rodriguez Santana

T. Burke, P. Chowdary, R. Curtis, K. Khair, M. Laffan, P. McLaughlin, D. Noone, B. O’Mahony, J. Pasi, M. W. Skinner, J. O’Hara

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Real-world clinical and patient-centric outcomes in people with haemophilia A in the United Kingdom: Findings from the CHESS II study

Presenting Author: Charlotte Camp

A. Shaikh, T. Burke, C. Hawes, W. McKeown, D. Morgan, J. O’Hara, C. Camp

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Real-world clinical and patient-centric outcomes in people with haemophilia A in Germany: Findings from the CHESS II study

Presenting Author: Anum Shaikh
A. Shaikh, T. Burke, C. Hawes, T. Becker, S. Brandt, J. O’Hara, C. Camp

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Real-world clinical and patient-centric outcomes in people with haemophilia A in France: Findings from the CHESS II study

Presenting Author: Charlotte Camp

A. Shaikh, T. Burke, C. Hawes, G. Duport, J. O’Hara, C. Camp

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Real-world clinical and patient-centric outcomes in people with haemophilia A in Spain: Findings from the CHESS II study

Presenting Author: Anum Shaikh

A. Shaikh, T. Burke, C. Hawes, G. Duport, J. O’Hara, C. Camp

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Poster

  • Congresses
  • Hemophilia A
  • International Society on Thrombosis and Haemostasis
  • 2021

Real-world clinical and patient-centric outcomes in people with haemophilia A in Italy: Findings from the CHESS II study

Presenting Author: Anum Shaikh

A. Shaikh, T. Burke, C. Hawes, A. Lupi, J. O’Hara, C. Camp

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Presentation

  • Congresses
  • Hemophilia A
  • World Federation of Hemophilia
  • 2022

LIVER BIOPSIES – MOLECULAR ANALYSES Analysis of Liver Biopsies Following AAV5-HFVIII-SQ Administration Revealed Inter-Individual Variability in Transgene mRNA and Protein Production

Presenting Author: Sylvia Fong

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