FDA ques­tions off-tar­get ed­it test­ing on Ver­tex and CRISPR's po­ten­tial sick­le cell gene ther­a­py ahead of ad­comm

The FDA is con­ven­ing an ad­vi­so­ry com­mit­tee next Tues­day to dis­cuss whether Ver­tex Phar­ma­ceu­ti­cals and CRISPR Ther­a­peu­tics did enough to ad­e­quate­ly mea­sure the off-tar­get al­ter­ations for their po­ten­tial gene ther­a­py for sick­le cell dis­ease.

While the ef­fi­ca­cy of the po­ten­tial gene ther­a­py, which em­ploys CRISPR/Cas9 edit­ing and is known as ex­agam­glo­gene au­totem­cel or exa-cel, does not seem to be a prob­lem for the agency, FDA ques­tioned whether Ver­tex used an ap­pro­pri­ate sam­ple size for its off-tar­get edit­ing analy­sis, ac­cord­ing to FDA brief­ing doc­u­ments re­leased Fri­day.

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