Michael Makris/LinkedIn
Apr 3, 2026, 15:51
Michael Makris: Approved but Unavailable Hemophilia B Gene Therapy Remains Out of Reach
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, shared a post on LinkedIn:
”If you are hoping to have gene therapy for hemophilia B you will have to wait, as currently there is no product available for use outside China.
Hemgenix is the only FDA/EMA approved gene therapy that is commercially available, but the company making it has announced that they do not have any product due to a manufacturing issue. They stress that this is not a safety issue.
The Chinese product is only approved for use in China, and it is at least theoretically possible to travel to have the infusion in Shanghai.”
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