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Home Global Pharma News

Vertex & CRISPR to collaborate for Development of gene therapy in Sickle Cell Disease and Beta Thalassemia

Vertex Pharmaceuticals and CRISPR Therapeutics Amend Collaboration for Development, Manufacturing and Commercialization of CTX001™ in Sickle Cell Disease and Beta Thalassemia

PRN by PRN
April 21, 2021
in Global Pharma News
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Vertex & CRISPR to collaborate for Development of gene therapy in Sickle Cell Disease and Beta Thalassemia
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– Under terms of amended agreement, Vertex to lead worldwide development, manufacturing and commercialization of CTX001-

– Revised agreement provides Vertex with 60% and CRISPR with 40% of program economics-

– CRISPR to receive $900 million upfront payment with potential for additional $200 million milestone payment upon CTX001 regulatory approval 

Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the companies have amended their collaboration agreement to develop, manufacture and commercialize CTX001, an investigational CRISPR/Cas9-based gene editing therapy that is being developed as a potentially curative therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). With this revised agreement, Vertex will deploy the breadth of its established global capabilities and proven experience in manufacturing, development, regulatory, and commercialization to maximize the potential for CTX001 to transform the lives of tens of thousands of patients in the U.S., Europe and other countries. CRISPR Therapeutics will continue to support the development of CTX001 and invest in further innovation to maximize its potential.

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, representing a 10% increase in program economics compared to 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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