ASH2020注目テーマ Late-Breaking Abstracts Session #6

中和抗体が存在しても
AAVベクターによる血友病の遺伝子治療に成功 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 (#LBA-6)

Steven W. Pipe（University of Michigan, Ann Arbor, MO）