NCT06328764

CS-101 in Patients With β-thalassemia

A Clinical Study Evaluating the Safety and Efficacy of In-vitro tBE Edited Autologous Hematopoietic Stem Progenitor Cells(CS-101) in Treating Subjects With β-thalassemia

• Status: Enrolling By Invitation
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: CorrectSequence Therapeutics Co., Ltd · Industry
• Sex: All
• Age: 6 Years – 35 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
GENETIC	CS-101	Autologous CD34+ hematopoietic stem cell suspension modified by in vitro base editing technique

Timeline

• Start date: 2024-03-19
• Primary completion: 2026-07-31
• Completion: 2026-07-31
• First posted: 2024-03-25
• Last updated: 2026-02-10

Locations

1 site across 1 country: China