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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