The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. CRISPR Therapeutics's main competitors include Sarepta Therapeutics, Intellia Therapeutics, ARCA biopharma and Hemera Biosciences. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. Each patient will be asked to participate in a long-term follow-up trial. Backlinks from other websites are the lifeblood of our site and a primary source of new traffic. -Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-, -Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-, -Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-. October 26, 2015, Vertex and CRISPR Therapeutics announced collaboration to use CRISPR-Cas9 Gene Editing Technology to discover and develop new treatments for Genetic Diseases. October 5, 2016, CRISPR loses Nobel Prize to tiny machines. CRISPR Therapeutics’ mission is to develop transformative gene-based medicines that offer the possibility of curative treatments. Each patient will be asked to participate in a long-term follow-up trial. Your browser will redirect to your requested content shortly. CRISPR Therapeutics AG published this content on 03 September 2020 and is solely responsible for the information contained therein. If you use our chart images on your site or blog, we ask that you provide attribution via a "dofollow" link back to this page. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. We have provided a few examples below that you can copy and paste to your site: Your image export is now complete. March 17, 2016, CRISPR Therapeutics Adds Pablo J. Cagnoni, M.D., to Board of Directors and names Tony Coles, M.D., Chairman of the Board to enhance drug development and commercialization expertise. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany. June 8, 2016, CRISPR Therapeutics and Anagenesis biotechnologies announced strategic in-licensing and collaboration agreement to develop CRISPR/Cas9-based cell Therapies for Muscle Diseases. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites.