CX0066p

CX0066p is an experimental lentiviral gene therapy vector designed for the treatment of sickle cell disease (SCD). Developed through a collaboration involving Emory University, Caring Cross, and the National Heart, Lung, and Blood Institute (NHLBI), the vector utilizes a forward-oriented β-globin expression cassette. This orientation is intended to optimize viral titers and improve the transduction efficiency of hematopoietic stem and progenitor cells (HSPCs) compared to traditional reverse-oriented vectors. In addition to delivering a functional human β-globin (HBB) gene to promote the production of healthy adult hemoglobin (HbA), CX0066p incorporates a microRNA-adapted short hairpin RNA (shmiR) system. This shmiR specifically targets and suppresses the expression of endogenous sickle-globin (HbS) mRNA. By combining gene addition with gene silencing, CX0066p aims to shift the balance of hemoglobin production toward HbA and away from the polymerization-prone HbS, thereby correcting the sickling phenotype of red blood cells.

Other names

LCR-Enhancer-P-HBB-shmiR-3g1.223 + 3g2.30a-R1