Kamau Therapeutics released preliminary clinical data from its RESTORE trial cohort ahead of upcoming abstract presentations at ASH. The company reported gene-edited correction of the sickle mutation in three patients using homology-directed repair (HDR) and restored adult hemoglobin (HbA) expression. According to the update, patients have remained transfusion-independent and reported no pain crises since treatment, with HbS levels falling below 20% of total hemoglobin in patients who reached the six-month post-infusion mark. The Phase I/II RESTORE trial is enrolling about 15 participants. The therapy is framed as aiming for direct correction rather than the fetal-hemoglobin switching approach used in some approved sickle cell gene therapies. The company said it is moving quickly on additional enrollment and plans to discuss registration trial design with the FDA. For the field, the early readout adds one more HDR-based platform to a crowded competitive landscape where durability, safety, and a path to registration remain the primary gating factors.
Get the Daily Brief