Wolfgang Miesbach on AAV5 Antibodies in Hemophilia B Gene Therapy
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“New study on AAV5 antibodies in haemophilia B gene therapy. We just published in Molecular Therapy – Methods and Clinical Development on how AAV5 antibodies behave over time in adults with haemophilia B.
This analysis is based on the HOPE-B phase 3 trial with 67 patients in the US and Europe.
Importantly, for etranocogene dezaparvovec, AAV5 NAb positivity is not a strict contraindication. But very high titers may reduce transduction efficiency. That’s why NAb screening is an essential part of assessing eligibility for commercial gene therapy.
Key findings:
- 48% of patients had neutralizing antibodies (NAbs) at screening (median titer 58; range 9–3440)
- Levels remained stable over 8 months ( median variation 25%; range 2–154%)
- Strong correlation between NAbs and IgG (r=0.96)
- Fewer than 5% switched from negative to positive
- ~10% seroreverted from positive to negative
- NAb positivity was more frequent in patients ≥50 years (p=0.0065)
Why this matters?
NAb test results were consistent over months. Screening can be done up to 8 months before gene therapy infusion, giving more flexibility for clinical planning.
Thanks to Robert Klamroth and Sandra Le Quellec for leading this work, and to CSL for supporting the study.”
Read the full article here.
Article: Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
Authors: Robert Klamroth, Michael Recht, Nigel S. Key, Wolfgang Miesbach, Steven W. Pipe, Radoslaw Kaczmarek, Douglass Drelich, Blanca Salazar, Sandra Le Quellec, Paul E. Monahan, Nicholas Galante, Paul van der Valk, Jacqueline Tarrant
Stay updated on all scientific advances in the field of Hemophilia with Hemostasis Today.
-
Aug 25, 2025, 09:26Caitlin Raymond on Platelet Refractoriness: New Series Alert!
-
Aug 23, 2025, 16:06Hemophilia Doesn’t Define Me—Skyler Tulley’s 100-Mile Challenge
-
Aug 23, 2025, 08:34Mamas A. Mamas Highlights UK Registry Data on STEMI Mortality and Infarct Size Trends
-
Aug 23, 2025, 08:28Len Valentino Named Researcher of the Year at BDC 2025
-
Aug 23, 2025, 08:26Thomas Reiser Highlights ISTH Pulse Podcast: Geoffrey Barnes Interviews Fionnuala Ní Áinle on Guideline Development
-
Aug 25, 2025, 10:40Maria Filomena Ruberto on The Hypercoagulable State
-
Aug 25, 2025, 08:28The Galeno Study: Doris Barcellona’s Insights on Oral Anticoagulants-related Bleeding
-
Aug 25, 2025, 08:16Wolfgang Miesbach on AAV5 Antibodies in Hemophilia B Gene Therapy
-
Aug 23, 2025, 16:30Benjamin Terrier: RCT Shows Rituximab Outperforms Conventional Therapy for Remission Induction in EGPA Annals of Int Med
-
Aug 23, 2025, 08:33British Journal of Haematology – Ferritin and Haemoglobin Recovery Patterns
-
Aug 18, 2025, 23:56Dr. Arun V. J Invites You to His Lecture on ‘AI and Data Sciences in Transfusion Medicine’ at the AI in Healthcare Conclave. Register now!
-
Aug 18, 2025, 23:50Safe and Efficient Thrombectomy with Artix System: Clinical Insights from Neil Desai and Colleagues
-
Aug 16, 2025, 14:42Mark Crowther Honors Davide Matino for Leading Promising Hemophilia Trial at McMaster University
-
Aug 14, 2025, 10:28Impressive 95% Clot Removal in a VTE Case: Symphony16F and Symphony24F Catheters
-
Jul 28, 2025, 14:55HORIBA Decoding High Efficiency in Hematology Lab: Free Online Workshop
-
Aug 23, 2025, 07:48Protecting Heart and Brain Health: Stroke Prevention in Women with Bleeding Disorders: NBDF
-
Aug 21, 2025, 15:08Global Variations in Blood Collection: What Explains the Differences?
-
Aug 20, 2025, 13:33Askat's Life with Hemophilia A after Access to Prophylactic Treatment Through the WFH Humanitarian Aid Program
-
Aug 19, 2025, 17:22Louise Bannon Encourages You to Support World Thrombosis Day!
-
Aug 17, 2025, 07:31Melissa Korn's story: Recognizing Pulmonary Embolism Symptoms Can Save Lives, NBCA