factor VIII Tochigi-24

factor VIII Tochigi-24 (T24) is a next-generation engineered coagulation factor VIII (FVIII) variant developed by Jichi Medical University and Nezu Biotech for the gene therapy of hemophilia A. T24 was designed by refining the 36 amino acid substitutions of a previous engineered FVIII variant (Ver.4) to reduce the number of substitutions while maintaining or enhancing its functional benefits. It combines 12 substitutions for activity enhancement, 13 for unfolded protein response (UPR) reduction, and 4 for improved secretion. In preclinical models, T24 demonstrated significantly higher FVIII activity and antigen levels compared to wild-type FVIII, with suppressed UPR activation. Delivered via an adeno-associated virus (AAV) vector (such as AAV5), T24 represents a promising transgene candidate for hemophilia A gene therapy, potentially allowing for lower vector doses and improved long-term durability.

Other names

FVIII T24FVIII Tochigi-24Tochigi-24Tochigi24Tochigi 24