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# FDA Places Clinical Hold on Sickle Cell Disease Drug
- URL: https://www.fdaweb.com/fda-places-clinical-hold-on-sickle-cell-disease-drug/
- Published: 2018-05-30T12:00:00.000Z
- Updated: 2026-09-15T00:08:56.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141566

FDA has placed a clinical hold on a CRISPR Therapeutics and Vertex Pharmaceuticals trial of CTX001 for treating sickle cell disease pending the resolution of certain questions that the agency presented as part of its IND review. The IND was submitted in April to support the planned initiation of a Phase 1/2 trial.

CTX001 is an investigational, gene-edited autologous hematopoietic stem cell therapy for patients suffering from B-thalassemia and sickle cell disease. The planned initiation of a Phase 1/2 trial of CTX001 in Europe in adult patients with transfusion dependent B-thalassemia is unchanged, and the companies say they expect to initiate that trial in the second half of this year.