When the FDA approved two gene therapies for sickle cell disease on the same day, adjectives like “momentous” were wheeled out. While the approvals were certainly landmarks, the commercialization that follows may disappoint.
Two weeks before the Christmas holidays, the FDA approved two gene therapies for sickle cell disease (SCD) – an inherited genetic disease for which the only disease-modifying treatment had been a bone marrow transplant. In such a large patient population the approvals were rightly hailed by the US president as a “significant medical advancement
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