Fulcrum Therapeutics said Sunday that a higher dose of its experimental pill for sickle cell disease was more effective at inducing an alternative form of the oxygen-carrying molecule hemoglobin — boosting hopes it could one day provide a simple and effective treatment for the disease.
Sickle cell is caused by a mutation in the hemoglobin gene, leading to misshapen blood cells that clog blood vessels and don’t adequately deliver oxygen throughout the body. The pill, pociredir, is designed to induce fetal hemoglobin, an alternate form of the protein usually only active in the womb and infancy.
In the early stage study presented at the American Society of Hematology annual meeting, 12 participants who received 20mg daily pociredir saw their levels of fetal hemoglobin rise from 7.1% of th

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