CRISPR Therapeutics and Vertex Announce FDA Has Lifted a Clinical Hold

CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD).

As quoted in the press release:

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

In addition to the acceptance of the IND announced today, CRISPR and Vertex previously announced that they had obtained approvals of Clinical Trial Applications for CTX001 in multiple countries outside the U.S. for both β-thalassemia and SCD. The companies remain on track to initiate a Phase 1/2 clinical study in SCD by the end of 2018 and are currently enrolling patients with transfusion dependent β-thalassemia in a Phase 1/2 trial in β-thalassemia in Europe.

Click here to read the full press release.