Michael Makris: Early Clinical Results of AAV8-BDD FVIII Gene Therapy in Hemophilia A
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, shared on LinkedIn about a recent article by Wei Liu et al, published in Signal Transduction and Targeted Therapy:
”New Haemophilia A phase 1 gene therapy study from China.
Used AAV8 with BDD FVIII.
Two cohorts: 2e12 and 4e12.
Prophylactic steroids +/- tacrolimus.
FVIII:C at 144 weeks of 2e12 was 5.6 iu/dl.
FVIII:C at 104 weeks of 4e12 was 42.7 iu/dl.
As with other AAV FVIII gene therapies, FVIII:C falls with time, chromogenic > 1-stage FVIII, and there is significant variation in achieved FVIII levels between patients.”
Title: Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity
Authors: Wei Liu, Xiaolei Pei, Tenghui Yu, Bingqi Xu, Xinyue Dai, Feng Xue, Lingling Chen, Xiaolin Wang, Yuhua Wang, Yunfei Chen, Xiaofan Liu, Rongfeng Fu, Wentian Wang, Ying Chi, Hui yuan Li, Renchi Yang, Lei Zhang
Read the Full Article on Nature: Signal Transduction and Targeted Therapy
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