Gene therapy for sickle cell disease remains a tough business model — and may never work

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The deep financial restructuring announced by Bluebird Bio this week offered another reminder that groundbreaking genetic medicines capable of transforming the lives of people with inherited disease are not now — and may never become — a sustainable business. 

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I have respect for gene-editing evangelists like Berkeley’s Fyodor Urnov who paint utopian pictures of a future where broad access to gene therapies and CRISPR cures will eliminate devastating diseases. But today, in the real world, Bluebird is in trouble because its personalized gene therapies, including Lyfgenia for sickle cell disease, cost too much to produce, and are too cumbersome and time-consuming to administer at a commercially viable scale. 

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