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US FDA lifts clinical hold on CRISPR, Vertex's sickle cell disease therapy

The U.S. Food and Drug Administration lifted the clinical hold on CRISPR Therapeutics AG and Vertex Pharmaceuticals Inc.'s experimental sickle cell disease treatment CTX001.

Sickle cell disease is a group of blood disorders normally inherited from a person's parents.

The FDA also accepted the investigational new drug application for CTX001, a stem cell therapy being developed to treat severe hemoglobinopathies — a genetic defect that leads to abnormalities in the hemoglobin molecule in blood cells.

Previously, the FDA had placed a clinical hold on the drug to seek resolution of certain questions as part of its review of the application, which was submitted in April to support the planned initiation of a phase 1/2 trial in the U.S.

CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly developing the drug under a co-development and co-commercialization agreement, said in an Oct. 10 news release that clinical trial applications for CTX001 have been approved in multiple countries outside the U.S. for both beta-thalassemia and sickle cell disease.

The companies are enrolling patients in the phase 1/2 trial of the treatment in Europe for adult patients with transfusion-dependent beta-thalassemia, and expect to start a phase 1/2 clinical study of the drug in sickle cell disease by the end of 2018.