Adults with severe or moderately severe haemophilia B receiving etranacogene dezaparvovec in the HOPE-B phase 3 trial experience a stable increase in mean Factor IX activity levels and durable haemostatic protection after 24 months’ follow-up

Introduction Etranacogene dezaparvovec (formerly AMT-061) is an investigational gene therapy for haemophilia B comprising an adeno-associated virus serotype 5 (AAV5) vector and a codon-optimised factor IX (FIX) Padua R338L transgene with a liver-specific promoter. The Phase 3 HOPE-B trial (NCT03569891) aims to assess the efficacy and safety of etranacogene dezaparvovec.