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A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.
CRISPR Therapeutics and Vertex Pharmaceuticals are jointly developing CTX001 for the treatment of hemoglobin-associated diseases, including SCD and transfusion-dependent beta thalassemia (TDT).
These preliminary findings, along with positive interim results from the CLIMB-Thal-111 study (NCT03655678) testing the therapy in TDT patients, were presented at the European Hematology Association 2021 Virtual Congress, held June 9–17.
“We are excited about these results and look forward to additional longer-term data and to moving this investigational medicine forward for a larger population of patients with these two devastating diseases,” Samarth Kulkarni, PhD, CRISPR Therapeutics’ CEO, said in a press release.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.