Shots:

• The P-III (HOPE-B) trial evaluates the safety & efficacy of etranacogene dezaparvovec in 54 male patients with hemophilia B
• The results showed that patients experienced a stable & durable increase in mean FIX activity & hemostatic protection @18mos. at the single infusion of etranacogene dezaparvovec, 64% reduction in ABR for all bleeds & 77% in FIX-treated bleeds, 98% discontinued use of prophylaxis, 97% reduction in mean unadjusted annualized FIX consumption & was well-tolerated
• Etranacogene Dezaparvovec is an AAV5-based gene therapy. Additionally, preclinical/clinical data showed that the therapy may be clinically effective in ~95% of patients with pre-existing Abs to AAV vectors

Ref: PR Newswire | Image: CSL Behring