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# CRISPR Therapeutics, Vertex Gain Fast Track for CTX001
- URL: https://www.fdaweb.com/crispr-therapeutics-vertex-gain-fast-track-for-ctx001/
- Published: 2019-01-04T12:00:00.000Z
- Updated: 2026-09-15T00:52:28.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5143084

FDA has granted CRISPR Therapeutics and Vertex Pharmaceuticals a fast track designation for CTX001 for treating sickle cell disease. The companies are developing the investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. Late last year, they filed an IND for a Phase 1/2 trial that is currently underway in the U.S. CTX001 is also being evaluated for treating B-thalassemia, and enrollment in a Phase 1/2 trial in B-thalassemia is currently open at multiple clinical trial sites in Europe.