Vertex Leads the CRISPR Therapeutics Partnership with $900M

Vertex has boosted the agreement with CRISPR Therapeutics to $900 million upfront with the companies racing towards beating bluebird bio to the market. They are working on the new gene-editing therapy for sickle cell disease and beta-thalassemia called CTX001. The company will pay CRISPR $900 million with a potential of $200 million in milestone payments on the approval of the therapy.

Almost 60% of the program cost will be taken up by Vertex and will receive the same in profits from the future worldwide sales of CTX001. An increase of 10% will be seen in the new agreement compared to the former deal. CTX001 is under development as a potential cure for blood diseases and beta-thalassemia which is transfusion dependent.

Sickle cell disease is an inherited disorder wherein the red blood cells turn into a sickle cell shape, which dies and causes a shortage. They could also clog the blood flow in the body causing stroke, infection, and other problems. African Americans are most affected by this disease. The only curative treatment for blood or bone marrow transplant. When caught early, the medicines or transfusions can manage complications.

Another inherited blood disorder, beta-thalassemia, causes the body to make less hemoglobin than normal. It can lead to severe or mild anemia in patients. Frequent blood transfusions are given to these patients. Vertex and CRISPR are currently in a race against bluebird bio to bring the market a new treatment option for patients with these two conditions.

Bluebird bio is ahead in its regulatory plans for Zynteglo with a European approval already in the bag. The therapy has been stumbled in the sickle cell disease after the safety concerns were raised.