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Gene therapy uptake in sickle cell stays slow, despite patient interest
BioPharma Dive December 9, 2024
A lengthy treatment process, coupled with weighty risks for recipients to consider, has resulted in plodding adoption of Casgevy and Lyfgenia during their first year on market.
One year on from the landmark U.S. approval of two powerfully effective gene therapies for sickle cell disease, the treatments have been barely used, a sluggish start that reflects the myriad challenges of launching them.
While some five dozen people with the blood disorder have begun the treatment process for one or the other therapy, only two had actually received an infusion through mid-November, according to the therapies’ developers, Vertex Pharmaceuticals and Bluebird bio. That’s because the process typically lasts at least several months, involving a precise choreography of medical consultations, preparatory treatments...
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