Kamau Therapeutics released preliminary clinical data from its revamped sickle cell gene editing program ahead of ASH. The report describes three gene-edited patients with precise correction of the sickle mutation in the β-globin gene using homology-directed repair (HDR), with restored expression of adult hemoglobin (HbA). All three patients were transfusion-independent and reported no pain crises since receiving gene-edited hematopoietic stem cells; in two patients with about six months follow-up, sickle hemoglobin fell below 20% of total hemoglobin. The company is enrolling roughly 15 participants in its Phase I/II RESTORE trial. Co-founder Matthew Porteus said the early readout matched target expectations for HDR working in engraftable HSCs, and the team plans to discuss registration trial design with the FDA. The update adds a new HDR-driven benchmark as the gene-editing field broadens beyond HbF induction approaches.