Hemostasis Today
NewsFeed
Headlines Hematology Health Medicine VTE ASH Plasma Donation
April, 2026
April 2026
M T W T F S S
 12345
6789101112
13141516171819
20212223242526
27282930  
Michael Makris: Early Clinical Results of AAV8-BDD FVIII Gene Therapy in Hemophilia A
Michael Makris/LinkedIn
Mar 12, 2026, 20:13

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 

Michael Makris: Early Clinical Results of AAV8-BDD FVIII Gene Therapy in Hemophilia A

Stay updated on all scientific advances with Hemostasis Today.

AAV Bingqi Xu bleeding disorders Feng Xue haemophilia Health Hematology Hemostasis Hemostasis News Hemostasis Today Hui yuan Li Lei Zhang Lingling Chen Medicine Michael Makris Nature Renchi Yang Rongfeng Fu Signal Transduction and Targeted Therapy steroids tacrolimus Tenghui Yu Wei Liu Wentian Wang Xiaofan Liu Xiaolei Pei Xiaolin Wang Xinyue Dai Ying Chi Yuhua Wang Yunfei Chen
NewsFeed