Home > Cardiology > ISTH 2020 > Haemophilia and Rare Bleeding Disorders > Novel gene therapy leads to normal FIX activity levels in severe haemophilia B

Novel gene therapy leads to normal FIX activity levels in severe haemophilia B

Presented By
Dr Pratima Chowdary, Royal Free London NHS Foundation Trust, United Kingdom
Conference
ISTH 2020
Trial
Phase 1/2, B-AMAZE
The B-AMAZE study showed that the novel gene therapy FLT180a achieved normal factor IX (FIX) activity levels in patients with severe haemophilia B. In addition, transaminitis could be prevented with a prophylactic, immunosuppressive regime [1]. The multicentre, open-label, phase 1/2 B-AMAZE study was presented by Dr Pratima Chowdary (Royal Free London NHS Foundation Trust, United Kingdom). Dr Chowdary explained, “The novel synthetic adeno-associated virus (AAV) capsid that we used in our study [AAVS3] has a 4 times higher liver transduction efficiency compared with a conventional one. The advantage is that with the same amount of dose or a lower dose you can potentially achieve factor IX in the normal range.” The study aimed to restore normal coagulation, thus eliminating the ...


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