Addressing AAV Antibodies in Hemophilia B Gene Therapy: Wolfgang Miesbach, MD, PhD

Video

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study.

“We can conclude, and this was a really surprising finding of the study, that in addition to patients with prior hepatitis B, prior hepatitis C and HIV infection, patients with preexisting antibodies to AAV, up to a certain titer, can be include in the study.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) reduced annualized bleeding rate (ABR) in patients with hemophilia B, both with and without neutralizing adeno-associated virus vector antibodies, compared to superior to Factor IX (FIX) prophylactic therapy.

These findings, from the final analysis of the phase 3 HOPE-B study (NCT03569891), were presented by investigator Wolfgang Miesbach, MD, PhD, at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach serves as professor and head of coagulation disorders at the Comprehensive Care Centre at University Hospital of Frankfurt, Germany.

Miesbach and colleagues found that, after a 6-month lead-in period, adjusted ABR was reduced by 64% (P = .0002) and FIX-treated bleeds reduced by 77% (P <.0001) over months 7 to 18. Almost all participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

CGTLive spoke with Miesbach to learn more about the demographics and findings from the HOPE-B study. He discussed the surprising finding that treatment was effective in participants with prior hepatitis B or C, HIV infection, or AAV antibodies.

REFERENCE
Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogenedezaparvovec gene therapy in hemophilia B – Data presented at EAHAD 2022. News release. CSL Behring. February 4, 2022. https://www.cslbehring.com/newsroom/2022/hope-b-data-presented-at-eahad2022
Recent Videos
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Jamie Jacobs, PhD, the program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences
Brian Kim, MBA, the chief executive officer of Mission Bio
Peter Cook, PhD, a senior research scientist at Seattle Children’s Research Institute
Nicholas Giovannone, PhD, a senior principal scientist at Regeneron
Related Content
© 2025 MJH Life Sciences

All rights reserved.