Blue­bird’s gene ther­a­py shows promise in keep­ing lethal Loren­zo’s Oil dis­ease in check

Af­ter an av­er­age of 29 months fol­low­ing a one-time gene ther­a­py in­fu­sion, a clear ma­jor­i­ty of boys suf­fer­ing from lethal cas­es of cere­bral adrenoleukody­s­tro­phy were alive and free of ma­jor com­pli­ca­tions from the dis­ease — the pri­ma­ry end­point in the Phase II/III study spon­sored by blue­bird bio $BLUE.

Pub­lish­ing in the New Eng­land Jour­nal of Med­i­cine, in­ves­ti­ga­tors at Dana-Far­ber, Boston Chil­dren’s Hos­pi­tal and Mass Gen­er­al re­cruit­ed 17 pa­tients with this dis­ease, mak­ing it one of the largest gene ther­a­py stud­ies now un­der­way. And they con­clud­ed that at this in­ter­im stage they have good ev­i­dence of dis­ease sta­bil­i­ty for this very vul­ner­a­ble group, with 88% still alive and com­pli­ca­tion free.

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