Kamau Therapeutics disclosed first clinical data from its revamped sickle cell gene-editing approach, reporting targeted correction of the β-globin mutation in the initial patient cohort. In early findings tied to the RESTORE Phase I/II trial, the company reported restored adult hemoglobin (HbA) expression and transfusion independence in all three patients, alongside no pain crises since gene-edited hematopoietic stem cell infusion. Investigators are using homology-directed repair to directly replace the sickle mutation, contrasting with HbF-induction strategies. Co-founder Matthew Porteus said the results match expectations for HDR performance in engraftable HSCs and that the company is moving toward discussions with the FDA on registration trial design.