Spark, Pfiz­er pass an­oth­er mile­stone with im­pres­sive re­sults treat­ing he­mo­phil­ia B

Spark and its part­ners at Pfiz­er have now shown that a group of 10 men treat­ed with their gene ther­a­py for he­mo­phil­ia B can lead nor­mal lives for about a year with no ev­i­dence of the life-threat­en­ing symp­toms that plagued them. And they’re bet­ting that it can last a life­time.

Their new study pub­lished in the New Eng­land Jour­nal of Med­i­cine con­tin­ues to un­der­score the con­sid­er­able promise of gene ther­a­py in he­mo­phil­ia. By in­tro­duc­ing a cor­rec­tive gene, pa­tients are able to make clot­ting fac­tor in a nor­mal range. All but one no longer need clot­ting fac­tor. And the sole pa­tient doesn’t need near­ly as much of it.

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