Blue­bird bio steps up with a promis­ing snap­shot of its gene ther­a­py up­grade

As a pi­o­neer in the new wave of gene ther­a­py treat­ments that have been steadi­ly wind­ing their way to­ward reg­u­la­tors with the promise of a once-and-done ge­net­ic fix for a wide range of ail­ments, blue­bird bio $BLUE al­so got the first taste of the kind of back­lash that can oc­cur when a new tech­nol­o­gy fails to live up to the hope and hype that spurs bil­lions of dol­lars of in­vest­ments.

For blue­bird, that mo­ment of truth came a lit­tle more than two years ago, when the com­pa­ny re­port­ed that a hand­ful of pa­tients had in­ad­e­quate re­spons­es to its gene ther­a­py for sick­le cell dis­ease and be­ta-tha­lassemia. Com­pa­ny in­ves­ti­ga­tors went back to the draw­ing board, changed the man­u­fac­tur­ing process on Lenti­Glo­bin with a new ap­proach they had been work­ing on that they felt would de­liv­er a bet­ter gene ther­a­py. And to­day they un­wrapped a snap­shot of the im­pact they’ve had.

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