Kamau Therapeutics Releases Encouraging Sickle Cell Clinical Data Ahead of ASH Meeting
Kamau Therapeutics, a California-based biotech company, has released preliminary clinical data for its gene-edited sickle cell disease (SCD) therapy, nula-cel. The data, to be presented at the American Society of Hematology (ASH) conference, show precise correction of the SCD mutation in the β-globin gene in three patients. These patients have also demonstrated restored expression of adult hemoglobin (HbA) and have become transfusion-independent, experiencing no pain crises since receiving their gene-edited hematopoietic stem cells (HSCs). Unlike FDA-approved Casgevy, which increases fetal hemoglobin (HbF), Kamau's nula-cel therapy directly replaces the SCD mutation using homology-directed repair (HDR). The company reports that sickle hemoglobin (HbS) levels fell below 20% of total hemoglobin in two patients who reached the 6-month post-infusion mark. The Phase I/II RESTORE trial is currently enrolling 15 participants.
