OTQ923 + HIX763

OTQ923 + HIX763 is an investigational ex vivo CRISPR/Cas9-edited autologous hematopoietic stem and progenitor cell (HSPC) combination therapy being developed for sickle cell disease and related hemoglobinopathies.[2][6][13][15] In this program, patient-derived CD34+ HSPCs are collected, genome-edited to disrupt the erythroid-specific enhancer of the B-cell lymphoma/leukemia 11A (BCL11A) gene and/or the BCL11A binding motif in the γ-globin (HBG) promoter, and then reinfused after myeloablative conditioning to increase fetal hemoglobin (HbF) levels and ameliorate sickling complications.[2][13][15] OTQ923 and HIX763 represent closely related CRISPR-edited HSPC products generated using RNA guides identified in a Novartis–Intellia collaboration, and are being evaluated in first‑in‑human Phase I/II trials to assess safety, engraftment, HbF induction, and reduction of vaso‑occlusive and other clinical events in sickle cell disease.[2][4][6][9][13][15]