Ver­tex and CRISPR spot­light an­oth­er im­por­tant gene edit­ing ad­vance in a march to a hoped-for cure

Ver­tex and CRISPR Ther­a­peu­tics have suc­cess­ful­ly nav­i­gat­ed an­oth­er stretch of the maid­en clin­i­cal jour­ney of the gene edit­ing ther­a­py CTX001 in a tiny group of pa­tients suf­fer­ing from be­ta tha­lassemia and sick­le cell dis­ease. And the re­searchers in­volved say they have good rea­son to be­lieve that they are still on the right track to prov­ing they are ad­vanc­ing a po­ten­tial­ly cu­ra­tive ap­proach with a shot at best-in-class sta­tus.

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