Vertex, CRISPR Therapeutics ink deal on sickle cell gene therapy

By The Science Advisory Board staff writers

April 20, 2021 -- Vertex Pharmaceuticals and CRISPR Therapeutics have amended their collaboration agreement to develop, manufacture, and commercialize CTX001, an ex vivo CRISPR/Cas9-based gene editing therapy being developed for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia.

Vertex will leverage its manufacturing, development, regulatory, and commercialization expertise and resources to maximize the potential for CTX001 in the U.S., Europe, and beyond. CRISPR Therapeutics will continue to support the development of CTX001 and invest in further development.

Under the terms of the amended agreement, Vertex will lead global development, manufacturing, and commercialization of CTX001, with support from CRISPR Therapeutics. Vertex will be responsible for 60% of program costs and will receive 60% of profits from future sales of CTX001 worldwide, a 10% increase from the previous agreement. CRISPR will be responsible for 40% of costs and will receive 40% of profits.

Additionally, CRISPR will receive a $900 million upfront payment, with potential for a $200 million payment upon the first regulatory approval of CTX001.

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