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Congress Posters and Presentations

Access posters and presentations from recent congresses. All displayed posters and presentations are protected by copyright and are intended for individual use only.
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Presentation

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  • 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, S. Harris, T. M. Robinson, K. Chavele, S. W. Pipe

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Presentation

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  • 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., S. Harris, D. Osmond, V. Newman, J. Henshaw, F. Peyvandi

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Presentation

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  • Congresses
  • Hemophilia A
  • NordCoag
  • 2023

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

Presenting Author: Bridget Yates

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

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Poster

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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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Presentation

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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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Poster

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  • 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

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  • 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

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  • 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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Poster

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  • 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

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  • 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

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

Molecular Characterization of Recombinant AAV5 encoding FVIII after Human administration

Presenting Author: K. Eggan

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Presentation

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • Congresses
  • Hemophilia A
  • 2022

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

B. 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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

Immune Suppression following Gene Therapy in Hemophilia

W. Miesbach

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Presentation

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

Human Liver Biopsy Analysis showed interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

Presenting Author: Sylvia Fong

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Presentation

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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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Presentation

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  • 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

M.C Ozelo, J. Mahlangu, KJ. 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

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  • 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

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

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Presentation

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  • 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

P. 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

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  • 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

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  • 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, G. Pierce, S. Pipe

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Poster

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  • 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

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

Examination and Validation of a Patient-centric Joint Metric: “PROBLEM JOINT”; Empirical Evidence from the CHESS Paediatrics Dataset

Presenting Author: Idaira Rodriguez Santana

T. Burke, I. Rodriguez Santana, 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

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  • 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

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  • 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

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  • 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

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  • 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

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  • 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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