Sek Kathiresan, Verve Therapeutics CEO

With first base edit­ing clin­i­cal tri­al un­der­way, Verve dis­clos­es sec­ond car­dio­vas­cu­lar dis­ease pro­gram

In Ju­ly, Verve Ther­a­peu­tics dosed its first pa­tient with a base edit­ing ther­a­py for het­erozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (FH), mark­ing the first time base edit­ing, which un­like CRISPR does not make a dou­ble-strand­ed cut in the DNA, has been used in a hu­man.

With that tri­al un­der­way in New Zealand, Verve an­nounced its sec­ond base edit­ing pro­gram on Mon­day morn­ing — one that it has hint­ed at be­fore. While Verve’s first pro­gram was for het­erozy­gous FH, in which pa­tients have one gene mu­ta­tion that im­pairs their liv­er’s abil­i­ty to re­move bad cho­les­terol, its sec­ond pro­gram is for ho­mozy­gous FH, or HoFH for short, a rar­er type of heart dis­ease in which pa­tients have in­her­it­ed two gene mu­ta­tions.

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