Nathan Connell: A Groundbreaking Study Has Just Been Published in Blood, From Vincent Muczynski and Colleagues
Nathan Connell, Clinical Chief of Hematology at Brigham and Women’s Faulkner Hospital, shared on LinkedIn:
”Exciting news in the field of hemophilia A gene therapy!
A groundbreaking study, “Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody,” has just been published in Blood, from Vincent Muczynski and colleagues (including Olivier Christophe, Caterina Casari, Gavin Ling, Peter Lenting, and Amit Nathwani).
This research addresses significant hurdles in current adeno-associated virus (AAV)-based treatments, specifically the challenge of packaging the large factor VIII (FVIII) transgene and concerns over long-term durability.
Key Scientific Takeaways:
- Novel Approach: Researchers developed AAV8-Bi8, a liver-directed gene therapy encoding a highly compact, 54.5 kDa single-chain FVIII-mimetic antibody, Bi8. This smaller cassette (4.4 kb) is much easier to package into the AAV vector.
- Efficacy: In FVIII-deficient mice, a single administration of AAV8-Bi8 resulted in dose-dependent, durable expression and complete phenotypic correction of bleeding.
- Clinical Equivalence: The therapeutic effect was comparable to emicizumab, the current market standard, but delivered via a gene therapy platform.
- Future Potential: This strategy offers a more flexible and efficient platform for long-term haemostatic control, including for patients who have developed inhibitors to FVIII.
A highly promising development that could significantly advance treatment options for hemophilia patients. Read the full article to dive into the data!”
Read the full article in Blood.
Article: Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody
Authors: Vincent Muczynski Corresponding Author , Olivier D. Christophe, Lewis Tanner, Charlotte Vayssiere, Alice Guérin, Caterina Casari, Jenny Hazel McIntosh, Doyoung Lee, Gavin Ling, Satyen Harish Gohil, Peter J Lenting, Edward G Tuddenham, Amit C Nathwani
Stay updated on all scientific advances in the field of hemophilia with Hemostasis Today.
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