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Gene-editing therapy OTQ923 cuts stroke risk in 3 SCD patients

The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 clinical trial. Findings suggest the treatment could help lower the risk of stroke in patients. The study (NCT04443907), sponsored by Novartis Pharmaceuticals, was stopped early due to commercial reasons, as the company discontinued its SCD gene-editing …
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Sickle Cell Disease News broke the news in on Thursday, July 10, 2025.
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