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Vertex’s Price Tag For Taking More Control Of CRISPR-Partnered Candidate: At Least $900m

Change To Partnership Comes After First Positive Pivotal Data For Blood Disorders

 
• By Joseph Haas

Vertex and partner CRISPR Therapeutics hope CDX001 can offer a single-use curative regimen for sickle cell disease and beta thalassemia patients. A regulatory filing is possible within 18-24 months.

Sickle cell anemia, 3D illustration showing blood vessel with normal and deformated crescent-like red blood cells
Vertex thinks CTX001 may offer a cure for sickle cell disease, beta thalassemia

Vertex Pharmaceuticals Incorporated increased its wager on a gene-editing therapy for sickle cell disease and transfusion-dependent beta thalassemia on 20 April, paying CRISPR Therapeutics AG $900m up front to increase both its responsibility and its economic share of CTX001, now in potentially registrational studies for both of the rare hemoglobinopathies.

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