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

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Nathan Yozwiak, PhD, the head of research at the GCTI
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Related Content
© 2025 MJH Life Sciences

All rights reserved.