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• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2026

Efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer

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• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2026

Impact of gene therapy on the hemophilia-free mind: Outcomes before and after valoctocogene roxaparvovec in GENEr8-1 trial participants

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2026

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

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2026

Characterization of ALT elevations during the 5-year GENEr8-1 trial of valoctocogene roxaparvovec gene transfer for severe hemophilia A

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• Hemophilia A
• Bari International Congress (BIC)
• 2025
• Safety and Efficacy Profile

Safety and efficacy of valoctocogene roxaparvovec in participants with active or prior FVIII inhibitors: Results from a phase 1/2 trial

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2025

Steady Musculoskeletal Health After Gene Therapy in Severe Hemophilia A: 5-Year Results

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2025

Mechanisms of AAV Vector Persistence Following Infant Gene Therapy in the Severe Hemophilia A Dog Model

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• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2025

Final GENEr8-1 results confirm enduring efficacy, safety, and quality of life improvements 5 years after valoctocogene roxaparvovec gene transfer

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2025
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2025
• Patient Reported Outcomes
• Safety and Efficacy Profile

Impact of gene therapy on the hemophilia-free mind: Outcomes before and after valoctocogene roxaparvovec in GENEr8-1 trial participants

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia Comprehensive Care Summit (WFH-CCS)
• 2025
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2025
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2025
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2025
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2025
• Safety and Efficacy Profile

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

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2025
• Safety and Efficacy Profile

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

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• Hemophilia A
• American Society of Hematology (ASH)
• 2024
• Safety and Efficacy Profile

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

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• Hemophilia A
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2024
• Patient Reported Outcomes

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

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• Hemophilia A
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2024
• Patient Reported Outcomes

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

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Presentation

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• Hemophilia A
• Societa Italiana per lo Studio dell’Emostasi e della Trombosi (SISET)
• 2024
• Safety and Efficacy Profile

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

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Presentation

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• Hemophilia A
• Societa Italiana per lo Studio dell’Emostasi e della Trombosi (SISET)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• Societa Italiana per lo Studio dell’Emostasi e della Trombosi (SISET)
• 2024
• Safety and Efficacy Profile

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

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• Hemophilia A
• Blood
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• Blood
• 2024
• Safety and Efficacy Profile

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

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Poster

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• 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

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Poster

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• Congresses
• Hemophilia A
• Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular (HEMO)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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• Hemophilia A
• Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular (HEMO)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Hemophilia A
• Congresso Brasileiro de Hematologia, Hemoterapia e Terapia Celular (HEMO)
• 2024
• Safety and Efficacy Profile

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

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• Hemophilia A
• National Bleeding Disorders Foundation (NBDF)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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• Hemophilia A
• National Bleeding Disorders Foundation (NBDF)
• 2024
• Safety and Efficacy Profile

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

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• Hemophilia A
• National Bleeding Disorders Foundation (NBDF)
• 2024
• Safety and Efficacy Profile

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

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Poster

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• Hemophilia A
• Congrès Français d’Hémostase (CFH)
• 2024
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• Congrès Français d’Hémostase (CFH)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2024

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

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Presentation

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• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2024
• Patient Reported Outcomes

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

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2024

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2024
• Patient Reported Outcomes

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

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Poster

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

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

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Presentation

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• Congresses
• Hemophilia A
• Thrombosis and Hemostasis Summit of North America (THSNA)
• 2024
• Safety and Efficacy Profile

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

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Poster

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

Characterization of pre-existing immunity to AAV5

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Presentation

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

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

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Presentation

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

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

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Presentation

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

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

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Poster

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

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

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Poster

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2024
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024

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

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2024
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• Blood
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Hematology (ASH)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Hematology (ASH)
• 2023

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

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Poster

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• 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

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Presentation

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• Congresses
• Hemophilia A
• Nordic Coagulation Meeting (NordCoag)
• 2023
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• Nordic Coagulation Meeting (NordCoag)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• Nordic Coagulation Meeting (NordCoag)
• 2023

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

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Poster

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• Congresses
• Hemophilia A
• Academy of Managed Care Pharmacy Nexus (AMCP)
• 2023

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

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Poster

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• Congresses
• Hemophilia A
• National Hemophilia Foundation (NHF)
• 2023
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• National Hemophilia Foundation (NHF)
• 2023
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• National Hemophilia Foundation (NHF)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2023

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2023
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2023
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2023

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2023

The longitudinal kinetics of AAV5 vector integration profiles in mice

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2023

Characterization of pre-existing immunity to AAV5

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia Comprehensive Care Summit (WFH-CCS)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia Comprehensive Care Summit (WFH-CCS)
• 2023
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• Congrès Français d’Hémostase (CFH)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• Gesellschaft für Thrombose- und Hämostaseforschung e.V. (GTH)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2022
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• American Thrombosis & Hemostasis Network Data Summit (ATHN)
• 2022

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

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Presentation

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

Molecular Characterization of Recombinant AAV5 encoding FVIII after Human administration

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Presentation

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

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

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Poster

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

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

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Poster

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

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

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Poster

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

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

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Poster

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

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

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Poster

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

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

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Poster

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• Congresses
• Hemophilia A
• Academy of Managed Care Pharmacy Nexus (AMCP)
• 2022

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

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Presentation

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• 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.

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022

Immune Suppression following Gene Therapy in Hemophilia

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• World Federation of Hemophilia Comprehensive Care Summit (WFH-CCS)
• 2022

Immune Suppression following Gene Therapy in Hemophilia

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2022

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2022

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2022

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

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Poster

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• Congresses
• Hemophilia A
• American Society of Gene & Cell Therapy (ASGCT)
• 2022

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2022
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2022
• Safety and Efficacy Profile

Valoctocogene Roxaparvovec Gene Transfer in Participants with HIV

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2022

Qualitative Research Evaluating Patient Preference for Haemophilia Therapy

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Poster

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• Congresses
• Hemophilia A
• European Association for Haemophilia & Allied Disorders (EAHAD)
• 2022
• Patient Reported Outcomes
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• Congrès Français d’Hémostase (CFH)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Hemophilia A
• Congrès Français d’Hémostase (CFH)
• 2023

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Poster

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• Congresses
• Hemophilia A
• International Society on Thrombosis & Haemostasis (ISTH)
• 2021
• Patient Reported Outcomes

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

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Presentation

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• Congresses
• Hemophilia A
• World Federation of Hemophilia (WFH)
• 2022
• Safety and Efficacy Profile

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

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