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genisi

05/23/15 5:15 PM

#191644 RE: DewDiligence #191636

Why is BXLT’s gene-therapy candidate for hemophilia A about two years behind its gene-therapy candidate for hemophilia B?

I think the large size of human FVIII cDNA (much larger than that of factor IX) was a challenge for delivery by the viral vector (few more problems I can think of but not as challenging), and it took time to overcome this problem.