Vertex Pharmaceuticals and CRISPR Therapeutics, a Swiss-US-based company developing therapeutics based on CRISPR-Cas9 gene-editing technology, have formed a $1.1-billion pact to develop, manufacture, and commercialize CRISPR Therapeutics’ CTX001, an investigational therapy for treating sickle-cell disease and transfusion-dependent beta-thalassemia, a blood disorder that reduces the production of hemoglobin.

Under the agreement, CRISPR Therapeutics will receive a $900-million upfront payment with potential for a $200-million payment upon the first regulatory approval of CTX001. 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 the profits from future sales of CTX001 globally, representing a 10% increase in program economics compared to the companies’ previous agreement. CRISPR Therapeutics will be responsible for 40% of costs and will receive 40% of the profits.

CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments. CTX001 represents the first potential treatment to emerge from the joint research program.

The transaction is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Source: Vertex Pharmaceuticals and CRISPR Therapeutics