‘The Best Decision I Ever Made’: Wolfgang Miesbach Marks 10 Years Since First Haemophilia B Gene Therapy Trial at Frankfurt
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on X:
“The Best Decision I Ever Made – Celebrating 10 Years of Gene Therapy at Frankfurt University Hospital. 10 years ago today, on July 22, 2015, Frankfurt University Hospital set a groundbreaking milestone by enrolling the first participant in the AMT-060 gene therapy trial for haemophilia B. This trial introduced an AAV5 vector carrying the wild-type Factor IX gene.
This would not have been possible without the outstanding expertise and close cooperation with the DRK Blood Donor Services and the Institute for Transfusion Medicine in Frankfurt.
AMT-060 was initiated during an early stage marked by scientific uncertainty. Long-term efficacy, safety, and side effect management were still largely unknown. Progress became possible through remarkable collaboration among researchers across the Netherlands, Denmark, Germany, and Italy. This multinational team—including leading hepatologists—leveraged a hub-and-spoke model to ensure treatment of patients from diverse centers and maintain seamless communication.
Over the past decade, scientific progress has been remarkable. The successor therapy, Etranacogene dezaparvovec (AMT-061), which utilizes the same AAV5 capsid combined with the hyperactive Padua variant of Factor IX, has received regulatory approval—a milestone that underscores the transition from early uncertainty to the availability of established gene therapy options for haemophilia.
Today, those early trial participants continue to benefit from sustained improvements, demonstrating the real-world impact of clinical innovation. During a recent follow-up, one patient expressed: ‘This was the best decision I ever made.’
Deepest gratitude goes to all the investigators, patients, and industry partners—uniQure and CSL Behring—whose dedication made this development possible.”
Wolfgang Miesbach reflects on the 10-year journey of gene therapy for haemophilia B at Frankfurt University Hospital, celebrating the groundbreaking AMT-060 trial, its collaborative success, and its lasting impact on patients.
Stay updated with Hemostasis Today.
-
Sep 7, 2025, 16:14Paddy Barret on Thin Cap Fibroatheroma's Outcomes
-
Sep 6, 2025, 14:04Chokri Ben Lamine – VTE Prophylaxis in Pregnancy (ASH 2018 Guidelines)
-
Sep 6, 2025, 13:48The Body Explained on Blood Clotting Pathways
-
Sep 5, 2025, 14:06Thrombosis Update: VTE Death Rate 3X Higher in Los Angeles Jails Than General Population
-
Sep 5, 2025, 10:59New Study Featured by Rare Disease Advisor Reveals Quality-of-Life Challenges in Hemophilia
-
Sep 6, 2025, 16:43Khaled Musallam: de-LIGHT Study on Use of Lifetime Anemia and Iron Control Index (LAICI) in Thalassemia Treatment
-
Sep 6, 2025, 15:06Managing Gynecologic Bleeding in Anticoagulated Patients: Toolkit Co-Authored by Divyaswathi Citla Sridhar
-
Sep 6, 2025, 14:30Mattia Galli on Whether Reduction in MACE Really Depicts Situation in Spontaneous MI or Death post-PCI
-
Sep 6, 2025, 13:55Patrick Cieran Gallogly and Colleagues on Atypical Symptoms in TIA and Minor Stroke
-
Sep 6, 2025, 13:37Unfractionated Heparin Dosing in ECMO: A Systematic Review and Meta-Analysis
-
Sep 2, 2025, 13:53Cost-Effective Strategies to Reduce Anaemia Highlighted in The Lancet Haematology
-
Sep 2, 2025, 13:04Ischemic Stroke or TIA Linked to Higher Femoral Fracture Risk
-
Sep 2, 2025, 12:51Treatment of Deep Brain AVMs Tied to Increased Hemorrhagic Stroke Risk
-
Aug 29, 2025, 09:03Vasculearn Network: New 2025 High Blood Pressure Guidelines Are Here
-
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!
-
Sep 7, 2025, 16:01Congratulations to Bart Loeys on Receiving Doctor Honoris Causa!
-
Sep 6, 2025, 14:09Erin VanDyke: Shifting Attention Upstream in VTE Care Through NBCA Centers of Excellence
-
Sep 6, 2025, 12:02Caitlin Raymond on Kairos in Medicine: How Curiosity Sustains Physicians and Prevents Burnout
-
Sep 5, 2025, 14:12EAHAD Podcast: Reducing Pain and Distress in Children Receiving Emicizumab
-
Sep 5, 2025, 10:58Côme Bommier Recommends CRTH for Clinical Trial Training and Career Growth in Hematology