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Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06328764
Study type Interventional
Source CorrectSequence Therapeutics Co., Ltd
Contact
Status Enrolling by invitation
Phase Early Phase 1
Start date March 19, 2024
Completion date January 30, 2026

See also
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