Vertex Pharmaceuticals and CRISPR Therapeutics to Co-Develop Sickle Cell Treatment

Per their previously-announced collaboration, Vertex Pharmaceuticals and CRISPR Therapeutics have identified their first gene editing candidate for development and eventual commercialization. The two companies plan to initiate a European Phase I/II clinical trial of their gene-based treatment, CTX001, in β-thalassemia in 2018, as well as an early-phase study in sickle cell disease in the US.

According to the collaborators, preclinical studies have shown that CTX001 has a high editing rate and was associated with clinically relevant increases in fetal hemoglobin, supporting further development of the gene therapy. The companies presented their preclinical results at the recent American Society for Hematology meeting.

“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 Dr. David Altshuler, 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.”

Unlike other proposed applications of CRISPR which is used to edit a patient’s cells in vivo, CTX100 is an ex vivo CRISPR gene-edited therapy designed to engineer a patient’s hematopoietic stem cells to produce high levels of fetal hemoglobin. This could reduce the need for patients to get blood transfusions.

“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,” said Dr. Samarth Kulkarni, 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.”