FDA fast-tracks stem cell therapy for SCDMarch 7, 2019
Fast track designation has been granted for CTX001 for the treatment of sickle cell disease (SCD) by USFDA.
CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.
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 ß-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.
In October 2018, CRISPR and Vertex announced the FDA acceptance of the Investigational New Drug application (IND) for CTX001 for the treatment of SCD, and enrolment in a phase 1/2 trial in SCD is currently underway in the US. The companies are also evaluating CTX001 for the treatment of ß-thalassemia, and enrolment in a Phase 1/2 trial in ß-thalassemia is currently open at multiple clinical trial sites in Europe.