Look­ing for leg up in he­mo­phil­ia B gene ther­a­py race, uniQure flash­es da­ta that helped lead to $2B CSL deal

In the two-head­ed race to de­vel­op the first gene ther­a­py for he­mo­phil­ia B, uniQure just got a ma­jor leg up.

In a late-break­ing ab­stract at ASH, the com­pa­ny re­leased da­ta from their piv­otal 54-per­son Phase III tri­al, show­ing that it in­creased lev­els of Fac­tor IX from be­low 2% of nor­mal to 37% of nor­mal af­ter 26 weeks. Al­though the com­pa­ny will still need to col­lect 52-week da­ta, the lev­els come in above what Pfiz­er has shown in ear­ly stud­ies for their ri­val gene ther­a­py and at or above what an­a­lysts sug­gest­ed would be need­ed to show ef­fi­ca­cy.

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