NCT07436767

Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 3 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

It is a single-arm, single-center, open-label, single-dose study. A total of three subjects with severe sickle cell disease (SCD), aged 12-50 years (inclusive), are planned to receive cell infusion. After successful hematopoietic stem cell engraftment is achieved in the first subject, cell infusion will be initiated for subsequent subjects.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
GENETIC	KL003 Cell Injection	KL003 is an autologous CD34⁺ hematopoietic stem cell gene therapy product in which the βA-T87Q-globin gene is transduced via a lentiviral vector. Through genetic modification, the patient's autologous CD34⁺ hematopoietic stem cells are engineered to differentiate into red blood cells expressing functional β-globin, thereby increasing overall hemoglobin levels, improving anemia, and ultimately eliminating transfusion dependence.

Timeline

• Start date: 2026-03-01
• Primary completion: 2028-09-30
• Completion: 2028-12-31
• First posted: 2026-02-27
• Last updated: 2026-02-27