The Food and Drug Administration on Wednesday approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its maker, the biotech company Bluebird Bio.
Called Zynteglo, the one-time treatment works by replacing a defective gene with a normally functioning one, thereby allowing the body to produce healthy red blood cells. Gene therapy is an entirely new way of treating beta thalassemia, which in its most severe form tethers patients to chronic blood transfusions and other medical procedures, and shortens their lifespan.
In Bluebird’s clinical trials, 90% of beta thalassemia patients who received a one-time infusion of Zynteglo no longer required blood transfusions.
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