NCT07000318

CS-206 in Patients With Sickle Cell Disease

An Open-Label Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CD34+ Human Hematopoietic Stem Cells Modified Using Transformer Base Editor in Participants With Severe Sickle Cell Disease

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Children's Hospital of Fudan University · Academic / Other
• Sex: All
• Age: 12 Years – 18 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-206 injection in treating sickle cell disease.

Detailed description

CS-206 is an autologous CD34+ cell suspension, edited by ex vivo 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

• Sickle Cell Disease

Interventions

Type	Name	Description
GENETIC	CS-206	Autologous CD34+ hematopoietic stem cell suspension modified by ex vivo base editing technique

Timeline

• Start date: 2025-06-30
• Primary completion: 2027-09-30
• Completion: 2027-12-31
• First posted: 2025-06-02
• Last updated: 2025-06-02

Locations

1 site across 1 country: China