First Patient Infused with CTX001 for TDT Treatment

| By | Cell Therapy, Clinical Trials, CRISPR, Vertex
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CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated reviewed recent progress in the clinical development programs for CTX001, an investigational, autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for patients suffering from severe hemoglobinopathies. The companies announced that the first patient has been treated with CTX001 in a Phase 1/2 clinical study of patients with transfusion-dependent beta thalassemia (TDT), marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial. In parallel, the companies are investigating CTX001 for the treatment of severe sickle cell disease (SCD) and also announced that the first patient has been enrolled in a Phase 1/2 clinical study of CTX001 in severe SCD in the U.S. and is expected to be infused with CTX001 in mid-2019.

We have made tremendous progress with CTX001 and are pleased to announce that we’ve treated the first patient with beta thalassemia in this clinical study. Treating the first patient in this study marks an important scientific and medical milestone and the beginning of our efforts to fully realize the promise of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases

said Samarth Kulkarni, Ph.D.,
Chief Executive Officer of CRISPR Therapeutics.

Beta thalassemia and sickle cell disease are serious, life-threatening diseases, and we are evaluating ex vivo treatment with CTX001 with the goal of creating a one-time potential curative therapy. Our collaboration with CRISPR Therapeutics offers an exciting new potential therapeutic approach that complements our strategy of using scientific innovation to create transformative medicines for serious diseases

said David Altshuler M.D.,
Ph.D., Executive Vice President
and Chief Scientific Officer at Vertex.