Michael Makris on Japan’s Breakthrough FVIII Variants for Hemophilia A
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, shared on LinkedIn:
”Important paper from Japan published in Blood.
The authors have developed FVIII variants with increased specific activity, which can potentially be used for gene therapy in hemophilia A.
These would be the equivalent of the Padua variant for FIX and can be used at much lower doses.
Even when used at 1/30th of the dose of Roctavian in macaques, supraphysiological FVIII levels were achieved.
No human studies yet.”
Read the full article here.
Article: Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy
Authors: Yuji Kashiwakura, Yuto Nakajima, Kio Horinaka, Tiago J. S. Lopes, Yuma Furuta, Yuki Yamaguchi, Nemekhbayar Baatartsogt, Morisada Hayakawa, Yuko Katakai, Susumu Uchiyama, Osamu Nureki, Keiji Nogami, Tsukasa Ohmori
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