Nov 4, 2026 —
Kamau Therapeutics has reported its first multi-patient clinical data for nula-cel, a gene-editing therapy that corrects the sickle cell mutation itself rather than working around it. The company was co-founded by Matthew Porteus of Stanford, where the approach was developed.
Most gene-editing approaches to sickle cell, including the approved therapy Casgevy, reactivate fetal hemoglobin to compensate for defective adult hemoglobin. Nula-cel instead uses ex vivo homology-directed repair to fix the disease-causing mutation in the HBB gene in a patient’s own blood stem cells, restoring normal adult hemoglobin.
Across three patients treated with the revised process, all became transfusion-independent before leaving the hospital, and no vaso-occlusive pain crises have occurred after treatment. In the two patients with longer follow-up, adult hemoglobin reached 76.8% at 12 months and 66.8% at 8 months, and more than 90% of circulating red cells carried protective levels of anti-sickling hemoglobin. The data were released ahead of the ASH annual meeting.
