Wolfgang Miesbach: Adenoassociated Virus-Based Gene Therapy for Haemophilia
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn about a recent article he and his colleagues co-authored, adding:
“Gene Therapy for Haemophilia: From Experimental Concept to Clinical Reality. Written by young investigators (haematologists and hepatologists), for young investigators. Delighted to share the new educational review published in the Journal of Thrombosis and Haemostasis (JTH), providing a comprehensive overview of adenoassociated virus (AAV)-based gene therapy for haemophilia:
- Clinical Efficacy Demonstrated:
Phase III trials of valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (haemophilia B) have shown durable factor expression for up to 5 years, with persons with haemophilia achieving factor levels in the normal or mild haemophilia range and significant reductions in bleeding rates. The FIX-Padua variant has proven particularly transformative, enhancing treatment efficacy 5- to 8-fold.
- Hepatological Considerations:
Approximately 20% to 90% of persons with haemophilia experience transaminase elevation postinfusion, requiring immunosuppressive management and close hepatologic monitoring. This underscores the essential role of hepatologists within the multidisciplinary care team and the importance of comprehensive liver health assessment prior to treatment.
- Future Directions:
Whilst AAV-based therapies represent a remarkable advance, several developments promise to further enhance outcomes: alternative vectors to address neutralising antibody limitations (expanding access for seropositive patients), enhanced factor variants for improved efficacy, and gene editing technologies including CRISPR-Cas9 for precise genetic correction.
- Critical Challenges Remain:
Long-term durability beyond 5 years, immune response management, standardised care pathways, and equitable global access require continued attention and collaborative research efforts.
Congratulations to Pascal Hoffmann, Niccolò Bitto, and Vincenzo La Mura.”
Title: Overview of gene therapy for hemophilia: questions and answers to navigate the innovation
Authors: Pascal Hoffmann, Niccolò Bitto, Vincenzo La Mura, Wolfgang Miesbach
Read the Full Article on Journal of Thrombosis and Haemostasis.
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