Image by Betty Pace
A sickled red blood cell beside a normal one
The US Food and Drug Administration (FDA) has placed a clinical hold on the investigational new drug application (IND) for CTX001. The agent is being developed for the treatment of sickle cell disease (SCD) and β-thalassemia.
The IND was submitted to the FDA in April to support the initiation of a phase 1/2 trial in the US in adult patients with SCD. The hold will be in place pending the resolution of questions as part of the FDA review.
The phase 1/2 trial in Europe in adult patients with transfusion-dependent β-thalassemia is expected to proceed according to schedule. Trial initiation is planned for the second half of 2018.
CTX001 is being co-developed and co-commercialized by CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated.
The agent is an investigational ex vivo CRISPR gene edited therapy for patients with β-thalassemia or sickle cell disease.
The patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.
The increase in HbF levels by CTX001 may potentially alleviate transfusion requirements for β-thalassemia patients and sickle crises for SCD patients.