Minhua Chu: CorrectSequence Therapeutics’ CS-101 Clinically Cures First Sickle Cell Disease Patient

Minhua Chu, Managing Partner at TransitionValue Partner, shared a post on X:

“CorrectSequence Therapeutics announced that an IIT, conducted in collaboration with the First Affiliated Hospital of Guangxi Medical University, for its base-editing drug CS-101 for sickle cell disease (SCD) has successfully “cured” the first patient. CS-101 injection, developed by CorrectSequence, is a base-editing drug for β-hemoglobinopathies. It works by collecting a patient’s autologous hematopoietic stem cells and using targeted base editing (tBE) to precisely edit the HBG1/2 promoter region, mimicking beneficial base mutations naturally occurring in healthy individuals to reactivate γ-globin expression.

The edited stem cells are then reinfused into the patient. The first SCD patient treated with CS-101 was a 21-year-old female with a baseline total hemoglobin concentration of 67.3 g/L and recurrent vaso-occlusive crises (VOCs). After receiving tBE-edited autologous stem cell transplantation in Feb. 2025, the patient achieved rapid and effective hematopoietic reconstitution, with neutrophil engraftment on day 13 and platelet concentration reaching above 50×10^9/L by day 21. One month post-treatment, fetal hemoglobin (HbF) levels rose from a baseline of 4.4% to 34.6%, and by three months, HbF levels consistently exceeded 60%, with sickle hemoglobin (HbS) levels below 40%.

Six months post-treatment, no VOCs occurred, and no product-related adverse events were observed. Currently, the Phase 1 clinical trial of CS-101 for β-thalassemia has been completed, with all patients achieving transfusion independence post-treatmenPivotal clinical trials (Phase 2/3) are set to begin soon. CorrectSequence Therapeutics announced that an Investigator-Initiated Trial (IIT), conducted in collaboration with the First Affiliated Hospital of Guangxi Medical University, for its base-editing drug CS-101 injection targeting sickle cell disease (SCD) has successfully “cured” the first patient.

CS-101 injection, developed by CorrectSequence Therapeutics, is a base-editing drug for β-hemoglobinopathies.

It works by collecting a patient’s autologous hematopoietic stem cells and using targeted base editing (tBE) to precisely edit the HBG1/2 promoter region, mimicking beneficial base mutations naturally occurring in healthy individuals to reactivate γ-globin expression. The edited stem cells are then reinfused into the patient. The first SCD patient treated with CS-101 was a 21-year-old female with a baseline total hemoglobin concentration of 67.3 g/L and recurrent vaso-occlusive crises (VOCs).

After receiving tBE-edited autologous stem cell transplantation in February 2025, the patient achieved rapid and effective hematopoietic reconstitution, with neutrophil engraftment on day 13 and platelet concentration reaching above 50×10^9/L by day 21. One month post-treatment, fetal hemoglobin (HbF) levels rose from a baseline of 4.4% to 34.6%, and by three months, HbF levels consistently exceeded 60%, with sickle hemoglobin (HbS) levels below 40%. Six months post-treatment, no VOCs occurred, and no product-related adverse events were observed. Currently, the Phase 1 clinical trial of CS-101 injection for β-thalassemia has been completed, with all patients achieving transfusion independence post-treatment. Pivotal clinical trials (Phase 2/3) are set to begin soon.”

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