We had the pleasure to talk to Steven Pipe (University of Michigan, Ann Arbor, MI, USA) to discuss the use of gene therapy for the treatment of haemophilia B. The abstract ‘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 Antibodies’ (ABSTRACT NUMBER: LBA-6) was presented at the Virtual 2020 ASH Congress, 2–10 December 2020.
- What are the major challenges and opportunities of gene therapy for haemophilia B? (0:18)
- What is etranacogene dezaparvovec and how does it differ from other gene therapy candidates? (3:13)
- What were the aims and design of the HOPE-B study? (4:59)
- What have we learned from the first data from this study and what other endpoints will be assessed at 52 weeks? (6:48)
Disclosures: Steven Pipe has served as a consultant to Apcintex, Bayer, Biomarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics and uniQure.
Support: Interview and filming supported by Touch Medical Media Ltd.
Filmed in coverage of the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition 2020.
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