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Vertex and CRISPR have amended their collaboration agreement to include the development, manufacture, and commercialization of CTX001, an investigational CRISPR/Cas9-based gene editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia.
Vertex Pharmaceuticals and CRISPR Therapeutics announced on April 20, 2021 that they have amended their collaboration agreement to include the development, manufacture, and commercialization of CTX001, an investigational CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
Under the terms of the agreement, CRISPR will receive an upfront payment of $900 million, with the potential for an additional $200-million payment upon the first regulatory approval of CTX001, Vertex said in a company press release. Vertex will handle the global development, manufacturing, and commercialization of the gene editing therapy and will be responsible for 60% of program costs, while receiving 60% of profits from future sales. CRISPR will be accountable for 40% of costs and will obtain 40% of profits.
“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, MD, PhD, executive chairman of Vertex, in the press release. “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.”
“Given the transformative results and momentum that we have generated with this program, we are adopting a new operating model to enable a globally coordinated launch of CTX001, leveraging Vertex’s best-in-class global capabilities and leadership in development, manufacturing, and commercialization to enable this medicine to reach all patients that can benefit from it as quickly as possible,” added Samarth Kulkarni, PhD, CEO of CRISPR Therapeutics, in the press release. “We remain deeply committed to the sickle cell and thalassemia patient communities and look forward to continued success in our partnership with Vertex.”