Ed­i­tas gives first look at CRISPR da­ta in sick­le cell, be­ta tha­lassemia six months af­ter lay­offs, re­fo­cus

At the start of this year, Ed­i­tas Med­i­cine nar­rowed its pipeline and laid off 20% of its work­force, a move to save cash and fo­cus on its gene-edit­ed cell ther­a­py can­di­date ED­IT-301 for sick­le cell dis­ease and trans­fu­sion-de­pen­dent be­ta tha­lassemia.

Now the com­pa­ny has da­ta from a hand­ful of pa­tients to show for it, it an­nounced Fri­day.

In the com­pa­ny’s the Phase I/II RU­BY tri­al, Ed­i­tas re­port­ed da­ta for the first four pa­tients with sick­le cell dis­ease treat­ed with ED­IT-301, two of whom reached nor­mal he­mo­glo­bin lev­els five months af­ter treat­ment. Both pa­tients al­so main­tained nor­mal he­mo­glo­bin lev­els at check­ups 10 and six months af­ter treat­ment and had a lev­el of fe­tal he­mo­glo­bin greater than 40% dur­ing the same time frame.

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