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CRISPR Therapeutics, Vertex get FDA fast-track designation for gene therapy

CRISPR Therapeutics AG and Vertex Pharmaceuticals Inc. said the U.S. Food and Drug Administration has granted fast-track designation to their sickle cell disease treatment.

The companies are developing CTX001 using the CRISPR gene-editing technology in which patient' stem cells are modified and infused back into the body.

Sickle cell disease is a group of inherited red blood cell disorders in which a dysfunctional form of hemoglobin causes the red blood cells to die early.

The designation comes after the FDA lifted a clinical hold on CTX001, which was placed to resolve certain questions as part of the investigational drug application.

The U.S FDA's fast-track program intends to expedite the review of drugs for serious conditions by offering benefits, including more frequent meetings with the agency and possibility of getting other pathways such as accelerated approval and priority review.

CRISPR and Vertex are collaborating to develop gene therapies using the CRISPR technology, with Vertex having exclusive rights to license up to six new treatments that emerge from the partnership.

CTX001, which is also being developed to treat blood disorders called β-thalassemia, is the first treatment to emerge from the joint research program.

Switzerland-based CRISPR and Boston-based Vertex are enrolling patients in two phase 1/2 studies to test CTX001 for treating β-thalassemia and sickle cell disease, respectively.