Stuart Orkin has been researching sickle cell disease since the 1980s, when he showed that sickle cell patients with high levels of fetal hemoglobin express very few symptoms. Then, in 2011, he found a genetic switch that, when knocked out, turns fetal hemoglobin on. Collectively, that research offered the blueprint for the strategy that Vertex used to develop its sickle cell gene therapy Casgevy. Now Orkin has turned his attention to researching alternative treatments for sickle cell, such as a pill or infusion, that would be less costly and time-consuming for patients.
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Location
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Boston, Mass.
