Kamau Therapeutics shared first clinical data from its revamped gene editing program for sickle cell disease ahead of ASH. In three patients enrolled in its Phase I/II RESTORE trial, the company reported precise correction of the sickle mutation in the beta-globin gene using homology-directed repair and restored adult hemoglobin expression. Kamau said all three patients have remained transfusion-independent without pain crises since treatment, with reported sickle hemoglobin levels falling below 20% in the two patients that had reached the six-month post-infusion mark. The company described the results as an early proof point for safety and durability as it continues enrolling additional participants. The program’s strategy—direct mutation replacement rather than fetal hemoglobin induction—sets up a comparative narrative against existing CRISPR-based therapies and could influence how future gene editing trials are designed for regulatory registration.