Ver­tex, fresh off an R&D miss, flash­es da­ta sug­gest­ing their sick­le cell CRISPR ther­a­py is work­ing

In one of the strongest bits of ev­i­dence yet for the ef­fect CRISPR gene edit­ing can have in peo­ple with ge­net­ic dis­eases, Ver­tex and CRISPR Ther­a­peu­tics showed their ex­per­i­men­tal ther­a­py kept two sick­le pa­tients from ex­pe­ri­enc­ing painful in­ci­dences and kept pa­tients with an­oth­er blood dis­or­der from re­quir­ing trans­fu­sions.

The da­ta, re­leased to­day in an ab­stract for the Amer­i­can So­ci­ety of Hema­tol­ogy, show that five pa­tients with trans­fu­sion-de­pen­dent be­ta tha­lassemia — a dis­or­der where pa­tients pro­duce in­suf­fi­cient he­mo­glo­bin — who re­ceived the ther­a­py haven’t need­ed trans­fu­sions since the sec­ond month af­ter ad­min­is­tra­tion. Pre­vi­ous­ly, the pa­tients had re­ceived be­tween 23.5 and 61 units of blood per year.

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