Ver­tex and CRISPR send exa-cel for FDA ap­proval; Aridis Ther­a­peu­tics ax­es 20% of its work­force

Ver­tex and CRISPR Ther­a­peu­tics have fin­ished the BLA for its treat­ment ex­agam­glo­gene au­totem­cel, or exa-cel, and will be go­ing to­ward the FDA for ap­proval.

Exa-cel is in­tend­ed to treat sick­le cell dis­ease and trans­fu­sion-de­pen­dent be­ta-tha­lassemia. The BLA al­so has a pri­or­i­ty re­view at­tached and, if giv­en, could bring down the FDAs re­view of the ap­pli­ca­tion to eight months in­stead of 12 months. Ver­tex and CRISPR are back­ing the sub­mis­sion with da­ta from two Phase III tri­als and a fol­low-up study.

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