Shots:

• The two P-I/ II studies- CLIMB-Thal-111 & CLIMB-SCD-121 studies will assess CTX001 in patients with transfusion-dependent beta-thalassemia & sickle cell disease aged 18-35yrs. and enrolls up to 45 patients with a follow up of ~2yrs. after infusion at six clinical sites       respectively
• The studies demonstrated a total hemoglobin level of 11.9 g/dL & 10.1 g/dL fetal hemoglobin @9mos. after CTX001 infusion in TDT patient and showed free of vaso-occlusive crises with a hemoglobin level of 11.3 g/dL & 46.6% fetal hemoglobin @4mos. after CTX001 infusion in a patient with SCD
• CTX001 is an investigational ex vivo CRISPR gene-edited therapy- being evaluated to treat TDT and severe SCD in which patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin

Click here to­ read full press release/ article 

 Ref: Vertex | Image: Vertex