Up­dat­ed: Sick­le cell gene edit­ing start­up halts tri­al af­ter flag­ging se­ri­ous side ef­fect in first pa­tient

The first-ever pa­tient to be treat­ed with a new, gene-edit­ed stem cell ther­a­py be­ing de­vel­oped for sick­le cell dis­ease ex­pe­ri­enced a se­ri­ous side ef­fect, forc­ing the drug­mak­er to halt the tri­al.

Graphite Bio said it vol­un­tar­i­ly paused fur­ther dos­ing in the Phase I/II CEDAR tri­al af­ter con­clud­ing that the se­ri­ous ad­verse event is like­ly re­lat­ed to its ther­a­py, nu­la­be­glo­gene au­to­gedtem­cel (nu­la-cel). It al­so re­port­ed the event to the FDA.

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