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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.

Vertex told Scrip that if the two ongoing studies continue at their current pace with further positive results, CTX001 could be filed for approval in the US and other geographies in the next 18 to 24 months. The two studies, designed for 45 patients each, have dosed 30 patients and reported data on 10 so far

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