Kamau Therapeutics emerged from stealth with preliminary clinical data from its Phase I/II RESTORE trial, showing gene-edited β-globin correction in the first cohort of sickle cell disease patients. In three patients, the company reported precise mutation correction using homology-directed repair (HDR) after editing hematopoietic stem cells. Kamau said all three patients are currently transfusion-independent with no pain crises since treatment, and that sickle hemoglobin (HbS) fell below 20% of total hemoglobin in two patients by the six-month post-infusion mark, alongside predominant adult hemoglobin expression. The company plans to enroll more patients and discuss registration trial design with the FDA. For gene-therapy developers, the report adds another HDR-focused sickle cell effort—distinct from therapies that primarily increase fetal hemoglobin—to a key set of ASH-season readouts.