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Conference Briefings

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

Pratima Chowdary, MRCP, FRCPath

AAV Integration Summary and Implications

Frederic D. Bushman, PhD

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B

Wolfgang Miesbach, MD

Relationship Between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Steven W. Pipe, MD

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis

Steven W. Pipe, MD

Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study

Steven W. Pipe, MD

Breaking Transgene Tolerance via Immunosuppression

Valder R. Arruda, MD, PhD

Targeting Sinusoial Endothelium

Valder R. Arruda, MD, PhD

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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