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Wolfgang Miesbach: From Uncertainty to Breakthrough – A Decade of Gene Therapy for Haemophilia B

Wolfgang Miesbach/LinkedIn

Jul 22, 2025, 20:31

Wolfgang Miesbach: From Uncertainty to Breakthrough – A Decade of Gene Therapy for Haemophilia B

Wolfgang Miesbach, Head of the Department of Haemostaseology and the Haemophilia Center of Medical Clinic 2 and Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:

” ‘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: From Uncertainty to Breakthrough - A Decade of Gene Therapy for Haemophilia B