CRISPR crew’s lab test spot­lights lead pro­gram in sick­le cell dis­ease, be­ta-tha­lassemia

CRISPR/Cas9 tech is still at a very ear­ly stage of de­vel­op­ment. But one of the top biotechs look­ing to make a break­through in the clin­ic got a chance to­day to ex­plain why one of its pre­clin­i­cal stud­ies helps demon­strate gene edit­ing’s promise in de­vel­op­ing a rad­i­cal­ly new kind of ther­a­py.

The com­pa­ny is CRISPR Ther­a­peu­tics $CR­SP and its pre­clin­i­cal pro­gram fo­cus­es on a dif­fer­ent kind of ap­proach in treat­ing sick­le cell dis­ease as well as be­ta-tha­lassemia — two dis­eases trig­gered by a ge­net­ic mu­ta­tion that slash­es the nat­ur­al pro­duc­tion of he­mo­glo­bin.

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