Up­dat­ed: #ASH20 Best of the Rest: Pfiz­er, Sang­amo launch a dura­bil­i­ty charge at Bio­Marin in hem A; The da­ta be­hind Mer­ck­'s $2.75B buy­out

UniQure may be get­ting all the ASH at­ten­tion for he­mo­phil­ia B, but Pfiz­er is try­ing to carve out a niche in he­mo­phil­ia A.

The phar­ma gi­ant and its gene edit­ing part­ner Sang­amo an­nounced long-term da­ta from their Phase I/II gene ther­a­py, show­ing that all five pa­tients that have been on the ther­a­py have seen “sus­tained fac­tor VI­II lev­els.” The me­di­an fac­tor VI­II lev­els were 56.9%, with the av­er­age com­ing to 70.4%.

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