EU stamps his­toric OK on blue­bird’s gene ther­a­py for β-tha­lassemia — now sit back and wait for the price

It’s of­fi­cial: blue­bird bio has scored its first drug ap­proval with an EU mar­ket­ing au­tho­riza­tion for its gene ther­a­py for trans­fu­sion-de­pen­dent β-tha­lassemia.

Zyn­te­glo is the new name for a treat­ment once known as Lenti­Glo­bin, a process that takes CD34+ cells from a pa­tient’s hematopoi­et­ic stem cells, in­serts func­tion­al copies of the βA-T87Q-glo­bin gene, then re­in­fus­es the mod­i­fied HSCs. Pa­tients are giv­en chemother­a­py to prime their bone mar­rows be­fore the re­in­fu­sion.

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