Clinical Hold Lifted on CRISPR Gene Therapy

FDA has lifted a clinical hold against a CRISPR Therapeutics and Vertex Pharmaceuticals IND for CTX001 for treating sickle cell disease. The investigational, autologous, gene-edited hematopoietic stem cell therapy is intended for patients suffering from severe hemoglobinopathies. The companies says they remain on track to initiate a Phase 1/2 clinical study in sickle cell disease by the end of the year, and are currently enrolling patients with transfusion dependent B-thalassemia in a Phase 1/2 trial in B-thalassemia in Europe.

The companies describe CTX001 as an ex vivo CRISPR gene-edited therapy in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. “The elevation of HbF by CTX001 has the potential to alleviate transfusion-requirements for B-thalassemia patients and painful and debilitating sickle crises for sickle cell patients,” they say.