Excitement over sickle cell now shifts to long slog of delivering treatments, in pivotal year for bluebird and gene therapy

SAN DIEGO — It is like­ly to take sev­er­al months be­fore the first new sick­le cell dis­ease gene ther­a­pies ac­tu­al­ly reach pa­tients fol­low­ing their land­mark ap­provals last week, ac­cord­ing to the com­pa­nies be­hind the ther­a­pies and the doc­tors who have been briefed on their roll­out.

As ex­cit­ing as the new treat­ments are — in­clud­ing the first-ever ther­a­py that us­es gene edit­ing — the ac­tu­al process of con­sent­ing pa­tients, mak­ing the treat­ments and de­liv­er­ing them is a com­pli­cat­ed slog. And while there are an es­ti­mat­ed 20,000 sick­le cell pa­tients liv­ing in the US who are el­i­gi­ble, on­ly a hand­ful of cen­ters that treat the dis­ease are ca­pa­ble of giv­ing the ther­a­pies.

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