CTX-001

Unassigned

New Medicines

Beta thalassaemia, transfusion-dependent

Information

Advanced therapy medicinal product (ATMP)
Vertex
Vertex

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Oct 19 · Committee for Orphan Medicinal Products (COMP) of the EMA adopted a positive opinion, recommending the granting of orphan drug status for CTX 001, for treatment of transfusion-dependent beta thalassemia [3].
Apr 19 · Granted fast track status in US [3].

Category

Autologous gene-edited haematopoietic stem cell therapy, containing an autologous CD34+ human haematopoietic stem and progenitor cells (hHSPCs), modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene.
1.5% (80-90 million people) of the population of the world are carriers of beta thalassaemia [1].
Beta thalassaemia, transfusion-dependent
Intravenous infusion

Trial or other data

Apr 20 · PI/II study (NCT03655678) recruiting [2].
Nov 19 · Interim results from PI/II study (NCT03655678) released from two patients, one with TDT. This patient achieved neutrophil engraftment within 33 days after the administration of CTX 001 and platelet engraftment within 37 days after infusion. At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9g/dL, 10.1g/dL fetal hemoglobin, and 99.8%F-cells (erythrocytes expressing fetal hemoglobin) [3].
Sep 18 · CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, initiates a PI/II trial to evaluate the safety and efficacy of single dose CTX 001 intravenous infusion, in patients aged between 18 to 35 years with transfusion-dependent β-thalassaemia (TDT) (CTX001-111; NCT03655678). The primary endpoint is defined as the proportion of subjects achieving transfusion reduction for at least six months, from nine to 24 months post drug infusion. 45 patients will be recruited in the USA, Germany, Canada and in the UK (Imperial College Hospital in London). Collection of primary outcome data is due to complete Feb 21 [2].