Vertex Pharmaceauticals inc issued the following announcement on May 30.
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND. The IND was submitted to the FDA in April to support the planned initiation of a Phase 1/2 trial in the U.S. in adult patients with sickle cell disease. CRISPR and Vertex expect to obtain additional information on the FDA's questions in the near future and plan to work rapidly with the FDA toward a resolution.
CTX001 is an investigational, gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease (SCD). The planned initiation of a Phase 1/2 trial of CTX001 in Europe in adult patients with transfusion dependent β-thalassemia is unchanged, and the companies expect to initiate the trial in the second half of 2018.
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