Verve shares first base edit­ing re­sults in hu­mans, sug­gest­ing ther­a­py can low­er cho­les­terol in pa­tients with ge­net­ic con­di­tion

PHILADEL­PHIA — Verve Ther­a­peu­tics’ next-gen­er­a­tion gene edit­ing treat­ment low­ered cho­les­terol lev­els in a hand­ful of pa­tients with het­erozy­gous fa­mil­ial hy­per­c­ho­les­terolemia, mark­ing the first time that base edit­ing has been used to di­rect­ly al­ter DNA in hu­mans.

The ear­ly Phase I da­ta, pre­sent­ed Sun­day at the Amer­i­can Heart As­so­ci­a­tion’s sci­en­tif­ic meet­ing, give a glimpse at how the in vi­vo treat­ment works in peo­ple who have been di­ag­nosed with the ge­net­ic dis­or­der that caus­es high cho­les­terol and of­ten heart dis­ease at an ear­ly age.

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