At the American Society of Hematology conference in San Diego (Max Gelman for Endpoints News)

Af­ter two his­toric ap­provals, Ed­i­tas un­veils promis­ing ear­ly re­sults for its sick­le cell ther­a­py: #ASH23

SAN DIEGO — As its com­peti­tors cel­e­brate the land­mark ap­proval of two sick­le cell gene ther­a­pies, Ed­i­tas Med­i­cine de­tailed promis­ing ear­ly re­sults in a small group of pa­tients for its own gene-edit­ed sick­le cell treat­ment.

Across two stud­ies in sick­le cell dis­ease and trans­fu­sion-de­pen­dent β-tha­lassemia, Ed­i­tas re­port­ed that 11 pa­tients have not had pain crises known as va­so-oc­clu­sive events since re­ceiv­ing its gene-edit­ed ther­a­py, known as ED­IT-301. But, on av­er­age, the pa­tients have not been fol­lowed for very long in the study — be­tween a few weeks and one and a half years. The da­ta were pre­sent­ed at the Amer­i­can So­ci­ety of Hema­tol­ogy an­nu­al meet­ing on Mon­day.

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