First Beta Thalassemia Patient Treated with CRISPR/Vertex CTX001 Now Transfusion-Independent

November 20, 2019 – CRISPR Therapeutics and Vertex have announced early results from the first beta thalassemia patient treated with their CTX100 gene editing therapy in the CLIMB-Thal-111 Study. CTX100 is a therapy that involves gene editing of a patient’s own hematopoietic stem cells (HSCs) using CRISPR/Cas9 gene editing technology in order to reactivate fetal hemoglobin production.

This initial patient had transfusion-dependent beta thalassemia with a β0/IVS-I-110 genotype. She required 16.5 transfusions per year prior to enrolling in the study. Nine months after treatment with CTX001, this patient is transfusion-independent with a total hemoglobin level of 11.9 g/dL (with 10.1 g/dL of the total hemoglobin attributable to fetal hemoglobin). Two serious adverse events were observed in this patient: (1) pneumonia in the presence of neutropenia and (2) veno-occlusive liver disease attributed to busulfan conditioning. Neither of these serious adverse events were considered to be related to CTX001, and both events subsequently resolved.

Further information about the study can be found in the press release at:

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