Blue­bird’s gene ther­a­py for be­ta-tha­lassemia falls short of a cure, but still wows in 22 pa­tients

Af­ter go­ing back to the draw­ing board to come up with a bet­ter gene ther­a­py for be­ta-tha­lassemia, a dis­ease that in­ter­feres with the body’s abil­i­ty to pro­duce he­mo­glo­bin, in­ves­ti­ga­tors work­ing for blue­bird bio $BLUE have pro­duced some com­pelling da­ta to demon­strate that their ther­a­py can elim­i­nate or re­duce the need for blood trans­fu­sions.

But they don’t have a cure — yet.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, the re­searchers re­port that they test­ed the treat­ment in 22 pa­tients. In the 9 pa­tients with the most se­vere form of the dis­ease, they re­port they were able to dra­mat­i­cal­ly re­duce the need for blood trans­fu­sions by 73% and elim­i­nate it for now in 3. Six of those pa­tients, though, have need­ed to con­tin­ue trans­fu­sions. And 12 of 13 pa­tients with a less se­vere form of the dis­ease cur­rent­ly no longer re­quire blood trans­fu­sions af­ter a sin­gle treat­ment — with a me­di­an of 26 months trans­fu­sion-free.

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