Wolfgang Miesbach Shares Newly Released Gene Therapy Qualification Criteria Paper
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on X:
“Exciting News! Our opinion paper on gene therapy qualification criteria has just been published! It outlines comprehensive standards for hub-and-spoke centers delivering gene therapy for haemophilia, helping ensure safety and efficacy across Europe and beyond:
Timely infrastructure guidance: The paper establishes practical qualification criteria for haemophilia centres delivering gene therapy, helping address new organizational, technical, and clinical requirements as gene therapies move into real-world practice.
Expert consensus: A comprehensive survey achieved 100% response among interdisciplinary specialists, ensuring robust recommendations grounded in broad clinical experience.
Hub-and-spoke model: The publication details a scalable and flexible framework—hub centres manage dosing and complexity while spoke centres provide local follow-up—making advanced therapies feasible and accessible across diverse care settings.
Unprecedented multidisciplinary team requirements including gene therapy liaison nurses, specialized pharmacists, administrative coordinators, and mandatory access to hepatologists.
Clear operational standards: The authors provide actionable criteria (e.g., bioisolator for ATMPs, 24-hour on-call service, rapid laboratory turnaround) and delineate staff roles, allowing centres to objectively assess readiness.
Flexible and adaptive: The model accommodates regional differences and supports spoke centre capacity-building, allowing responsibility to shift as local experience increases—a practical approach for evolving health systems.
Focus on safety and coordination: Emphasizes standardized protocols, scenario preparedness (“dry run” exercises), and robust SOPs for adverse event management, underpinning a safety-first transition to gene therapy.
Globally relevant: While based on European experience, the findings and framework are adaptable to international settings, with comparisons to other models such as the US health system.
Congratulations and a BIG THANK YOU to the EAHAD Gene Therapy Working Group!”
For more information follow the link.
Stay informed 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