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Conference Briefings

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults With Severe Hemophilia

Barbara A. Konkle, MD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Steven W. Pipe, MD 

Frequency, Location and Nature of AAV Vector Insertions After Long-term Follow-up of FVIII Transgene Delivery in a Hemophilia A Dog Model

Paul Batty, MBBS, PhD 

First Data From the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults With Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing

Steven W. Pipe, MD

A Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieves Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)

Pratima Chowdary, MRCP, FRCPath

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

Wolfgang A. Miesbach, MD

Phase I/II Trial of SPK-8011: Stable and Durable FVIII Expression for > 2 Years With Significant ABR Improvements in Initial Dose Cohorts Following AAV-Mediated FVIII Gene Transfer for Hemophilia A

Presented by Lindsey A. George, MD

  • Long-term Vector Genome Outcomes and Immunogenicity of AAV FVIII Gene Transfer in the Hemophilia A Dog Model
  • Recent Progress in the Development of AMT-061 (Etranacogene Dezaparvovec) for Persons With Severe or Moderately Severe Hemophilia B
  • First-in-human Four-year Follow-up Study of Durable Therapeutic Efficacy and Safety: AAV Gene Therapy With Valoctocogene Roxaparvovec for Severe Haemophilia A
  • First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results
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Supported by educational grants from BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc

Click here to access the enduring symposium:
“Key Considerations: Advances in Gene Therapy for Hemophilia”

Featuring Glenn Pierce, MD, PhD, Lindsey George, MD, Alfonso Iorio, MD and Barbara Konkle, MD

© 2021 International Society on Thrombosis and Haemostasis, Inc. All rights reserved.
  • Home
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    • Train-the-Trainer Toolkit
    • 2020 Virtual Learning Sessions
    • Past ISTH Congress Education
    • Roadmap
  • News
  • Resources
  • Subscribe
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