Discover and read the best of Twitter Threads about #FVIII
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Another FVIII variant to tune into Dr. Denise Sabatino from CHOP because we are all looking to do the following in 2nd gen AAV gene therapy. Follow this thread to learn more (read my prior one on #FVIII-QQ from yesterday also) #ASH22 1/n 
Canine FVIII has increased expression due to a variant of the furin and a3 cleavage site which they put into this product (using AAV8 vector). (known as delta3-SP/BDD) #ASH22 2/n
Blue lines here show 2-4x higher factor VIII activity in canine hemophilia models and they moved onto non-human primate models (with endogenous f8 production (hence the %of F8) that developed immune response). #ASH22 3/n
