In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Pre­lim­i­nary da­ta on two pa­tients with blood dis­or­ders that have been ad­min­is­tered with Ver­tex and part­ner CRISPR Ther­a­peu­tics’ gene-edit­ing ther­a­py sug­gest the tech­nol­o­gy is safe and ef­fec­tive, mark­ing the first in­stance of the ben­e­fit of the use of CRISPR/Cas9 tech­nol­o­gy in hu­mans suf­fer­ing from dis­ease.

Pa­tients in these phase I/II stud­ies give up pe­riph­er­al blood from which hematopoi­et­ic stem and prog­en­i­tor cells are iso­lat­ed. The cells are tin­kered with us­ing CRISPR/Cas9 tech­nol­o­gy, and the edit­ed cells — CTX001 — are in­fused back in­to the pa­tient via a stem cell trans­plant. The ob­jec­tive of CTX001 is to fix the er­rant he­mo­glo­bin gene in patents with two blood dis­or­ders: be­ta-tha­lassemia and sick­le cell dis­ease, by un­leash­ing the pro­duc­tion of fe­tal he­mo­glo­bin.

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