Gene-edited stem cell therapy for thalassemia

June 11, 2019 0 By FM

The US FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia (TDT).

CTX001 is an investigational, autologous, gene-edited haematopoietic stem cell therapy for patients suffering from severe haemoglobinopathies.

In February 2019, CRISPR Therapeutics and Vertex announced that the first patient had been treated with CTX001 in a phase 1/2 clinical study of patients with TDT, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial.

The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT, non-beta zero/beta zero subtypes. The companies are also evaluating CTX001 for the treatment of sickle cell disease (SCD) and received Fast Track Designation for CTX001 from the FDA in January 2019 for SCD.

In CTX001 therapy, a patient’s haematopoietic stem cells are engineered to produce high levels of foetal haemoglobin (HbF) in red blood cells. HbF is a form of the oxygen-carrying haemoglobin that is naturally present at birth and is then replaced by the adult form of haemoglobin.

The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.