First patients with sickle cell disease treated with experimental gene-editing cell therapy remains VOC-free after 9 months
The first patients with severe sickle cell disease (SCD) treated with an experimental gene-editing cell therapy is free of vaso-occlusive crises (VOCs) 9 months after treatment, according to data from Phase 1/2 clinical trial presented at the European Hematology Association (EHA) virtual congress.
Nine months after infusion with the investigational CRISPR/Cas9 gene-editing therapy, CTX001, the patient was free of VOCs and was transfusion independent. The patient had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. At 6 months, bone marrow allelic editing was 81.4%.
Three serious adverse events (sepsis in the presence of neutropenia, cholelithiasis and abdominal pain) occurred after infusion, although none were considered related to CTX001.
“With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients,” said Samarth Kulkarni, PhD, Chief Executive Officer of CRISPR Therapeutics, in a press release. “These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases.”
Read the full press release here.