Af­ter good news in sick­le cell, blue­bird bio fol­lows up with pos­i­tive long-term da­ta in Loren­zo’s Oil dis­ease

Less than a week af­ter blue­bird bio said their sick­le cell gene ther­a­py was “very un­like­ly” to have caused a case of acute myeloid leukemia, the com­pa­ny is pre­sent­ing some more good news for a dif­fer­ent pro­gram.

Re­veal­ing long-term da­ta in the rare ge­net­ic dis­or­der cere­bral adrenoleukody­s­tro­phy (CALD), blue­bird said Mon­day that 27 of 30 evalu­able pa­tients in a piv­otal Phase II/III study were alive and free of ma­jor func­tion­al dis­abil­i­ties at the two-year mark. And they’re al­so high­light­ing da­ta from a fol­low-up study where 14 pa­tients re­main un­af­fect­ed by such dis­abil­i­ties through five years.

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