Vertex Pharmaceuticals and CRISPR Therapeutics announced that the companies will co-develop and co-commercialize CTX001, an investigational gene editing treatment, as part of the companies' previously announced collaboration aimed at the discovery and development of new gene editing treatments that use the CRISPR/Cas9 technology. CTX001 represents the first gene-based treatment that Vertex exclusively licensed from CRISPR Therapeutics as part of the collaboration. For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide. A Clinical Trial Application was submitted earlier this month for CTX001 to support the initiation of a Phase 1/2 trial in β-thalassemia in 2018 in Europe, and an Investigational New Drug (IND) Application is planned for submission in 2018 to support the initiation of a Phase 1/2 trial in sickle cell disease in the U.S. Preclinical data presented for CTX001 at the American Society for Hematology on December 10, 2017 showed clinically relevant increases in fetal hemoglobin and a high editing rate that support the advancement of CTX001 into the planned trials in β-thalassemia and sickle cell disease in 2018.

"Over the past two years, we've made significant progress with CRISPR Therapeutics on the discovery and preclinical development of multiple CRISPR/Cas9-based treatments, and we're pleased to select CTX001 as the first of these treatments to move into clinical development as part of our collaboration," said David Altshuler, M.D., Ph.D., Vertex's Executive Vice President, Global Research and Chief Scientific Officer. "The addition of CTX001 to our clinical development pipeline provides us with a near-term opportunity to generate the first proof-of-concept clinical data for a CRISPR/Cas9-based medicine in two genetic diseases that are highly aligned with our research strategy."

"The submission of a Clinical Trial Application for CTX001 in Europe, supported by the robust data presented at the recent ASH Annual Meeting, reflect the advances we have achieved in translating the potential of CRISPR/Cas9 science into transformative therapies. We now look forward to working closely with Vertex as we initiate clinical trials next year," commented Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "The study of CTX001 in β-thalassemia will be the first company-sponsored clinical trial of a CRISPR-based therapy and is a major step forward for both the treatment of certain inherited blood diseases and for our collaboration with Vertex."

Clinical Development Plans for CTX001
CRISPR Therapeutics and Vertex will co-develop and co-commercialize CTX001 for the treatment of hemoglobinopathies, including β-thalassemia and sickle cell disease. A Phase 1/2 trial of CTX001 is expected to begin in 2018 in Europe and will be designed to assess the safety and efficacy of CTX001 in adult transfusion dependent β-thalassemia patients. The companies also plan to file an IND Application for CTX001 with the United States Food and Drug Administration to support the initiation of a Phase 1/2 trial in sickle cell disease in 2018 in the U.S. Additional details on the trial designs will be provided upon study initiation.

About CTX001 and Recent Data Presented at the American Society for Hematology (ASH) Annual Meeting
CTX001 is an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia and sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen carrying hemoglobin that is naturally present at birth, and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion-requirements for β-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.

On December 10, 2017, CRISPR Therapeutics presented preclinical data at the 2017 ASH Annual Meeting that showed greater than 90% editing of hematopoietic stem cells at the target site, leading to clinically relevant increases in fetal hemoglobin. These data support the advancement of CTX001 into the planned trials in β-thalassemia and sickle cell disease in 2018.