OTQ923

OTQ923 is an investigational autologous hematopoietic stem and progenitor cell (HSPC) therapy developed using ex vivo genome editing with the CRISPR-Cas9 system. The therapy involves collecting a patient’s own CD34+ HSPCs, editing them with CRISPR-Cas9 to disrupt the promoters of the HBG1 and HBG2 (γ-globin) genes, thereby inducing sustained expression of fetal hemoglobin (HbF). The edited cells are then infused back into the patient following myeloablative conditioning. This approach aims to ameliorate sickle cell disease (SCD) by increasing HbF levels in red blood cells, which protects against sickling and related complications. Clinical studies have shown that OTQ923 leads to stable engraftment, increased HbF production, and clinical improvement in SCD patients[1][5][6][7][10].

Other names

CRISPR/Cas9-based engineered cell therapy Novartis/Intellia Therapeutics