Vertex Pharmaceuticals and CRISPR Therapeutics Amend Collaboration for Development, Manufacturing and Commercialization of CTX001 in Sickle Cell Disease and Beta Thalassemia
- 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 -
BOSTON and CAMBRIDGE, Mass. and ZUG, Switzerland, April 20, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) today 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.
“Cell and genetic therapies are key to our strategy of developing transformative therapies for serious diseases, and this agreement is an important next step in cementing our leadership in these modalities as we bring forward our broad gene and cell-based therapeutics portfolio. As we take the lead on CTX001, we want to acknowledge the foundational contributions by the team at CRISPR Therapeutics,” said Jeffrey Leiden, M.D., Ph.D., Executive Chairman of Vertex. “Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialization. We see a significant commercial opportunity for CTX001, and we believe we will be able to further enhance that opportunity by fully leveraging the breadth of Vertex’s capabilities – including our established and proven R&D and commercialization expertise in serious diseases – to bring CTX001 to more patients around the world, more quickly.”