CRISPR Therapeutics and Vertex Report Results of CTX001 from First Two Patients with Severe Hemoglobinopathies

 CRISPR Therapeutics and Vertex Report Results of CTX001 from First Two Patients with Severe Hemoglobinopathies

CRISPR Therapeutics and Vertex Reports Results of CTX001 from First Two Patients with Severe Hemoglobinopathies

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: CRISPR Therapeutics

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