An Early Phase 1 interventional study of CS-101 in Beta-Thalassemia, sponsored by CorrectSequence Therapeutics Co., Ltd. Enrolling by invitation at 1 site in China. Open to participants aged 6 Years to 35 Years. Per ClinicalTrials.gov, last updated 2026-06-09.
Sponsored by CorrectSequence Therapeutics Co., Ltd · Early Phase 1, Interventional, and Treatment
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
CS-101 is an autologous CD34+ cell suspension, edited by in vitro base editing technology, which modifies the BCL11A binding site in HBG promoter, so that it loses the ability to bind to BCL11A, which can re-induce the production of γ-globin chain and increase the concentration of fetal hemoglobin(HbF) in the blood, compensating for the function of missing adult hemoglobin HbA to achieve clinical cure. The therapy addresses two major challenges in the current treatment of the disease: lack of matching donors and graft-versus-host diseases in allogeneic hematopoietic stem cell transplantation.
305 studies on the registry are indexed under beta-Thalassemia; 70 are open to participants now.
This study's planned enrollment of 10 is below the median of 38 across 211 interventional studies indexed under beta-Thalassemia.
Browse beta-Thalassemia studies →CorrectSequence Therapeutics Co., Ltd is the lead sponsor of 9 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Subjects who have received or are receiving thalidomide and/or Luspatercept, when their drug-drug interaction on the efficacy and safety of CS-101 cannot be ruled out, unless at least there are 3 test results showing the total hemoglobin level before transfusion is below 9g/dL in the past 6 months before screening.
Previously received allogeneic hematopoietic stem cell transplantation or gene(edited) therapy.
Subjects have available related fully matching donors and are eligible and prepared for allogeneic hematopoietic stem cell transplantation.
Those with active infections, including but not limited to: HIV, hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum test positive, or known tuberculosis, parasitic infection, etc. who are judged by the investigator to be unsuitable to participate in this study.
Echocardiography results with ejection fraction below 45%. Advanced liver disease, defined as：
Aspartate aminotransferase (AST), alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) or:
Baseline International Normalized Ratio (INR) >1.5 × ULN.
MRI during the screening period showed heavy iron overload and is judged by the investigator to be unable to participate in the study.
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Genetic: CS-101
Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique
Frequency and severity of adverse events(AEs）as assessed by CTCAE v5.0
Time frame: From signing informed consent to 12 months post-CS-101 infusion
Time to neutrophil and platelet engraftment
Time to neutrophil engraftment is defined as first day of 3 consecutive measurements of absolute neutrophil count≥0.5×10\^9/L on three different days； Time to platelet engraftment is defined as first day of 3 consecutive measurements of absolute platelet count≥20×10\^9/L on three different days and without platelet transfusion；
Time frame: Days post-CS-101 infusion
Proportion of subjects with engraftment
Subjects with engraftment is defined as neutrophil engrafted
Time frame: within 42 days post-CS-101infusion
Incidence of transplant-related mortality
Time frame: From baseline to 100 days post-CS-101 infusion
All-cause mortality
Time frame: From signing informed consent to 12 months post-CS-101 infusion
Proportion of subjects achieving transfusion independence for at least 6 consecutive months
Time frame: From 3 months up to 12 months post-CS-101 infusion
Time to last red blood cell(RBC) transfusion
Time frame: Days post-CS-101 infusion
Change in total hemoglobin(Hb) concentration over time
Total hemoglobin concentration change from baseline to 12 months post-CS-101 infusion
Time frame: up to 12 months post-CS-101 infusion
Change in fetal hemoglobin(HbF) concentration over time
γ-globin concentration change from baseline to 12 months post-CS-101 infusion
Time frame: up to 12 months post-CS-101 infusion
Chimerism level in Peripheral blood and bone marrow
Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time
Time frame: up to 12 months post-CS-101 infusion
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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CorrectSequence Therapeutics Co., Ltd