Β-Thalassemia Treatment with KL003 Cell Injection

Exclusion Criteria15

• Diagnosis of composite α thalassemia;
• Prior receipt of gene therapy or allo-HSCT;
• Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match;
• Participants with severe iron overload at the time of screening;
• Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody;
• Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.);
• Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator;
• Subjects positive with the following etiological tests: human immunodeficiency virus(HIV-1-2),human cytomegalovirus (HCMV-DNA),EB virus（EBV-DNA）,HBV (HBsAg/HBV-DNA positive),HCV antibody (HCV-Ab), Human T-lymphotropic virus antibody (HTLV-Ab), Treponema pallidum antibody (TP-Ab);
• Uncorrectable coagulation dysfunction or history of severe bleeding disorder;
• History of major organ damage including:
• Liver function test suggest AST or ALT levels >3× upper limit of normal（ULN）;
• Total serum bilirubin value>2.5×ULN;if combined with Gilbert syndrome, total bilirubin>3×ULN and direct bilirubin value>2.5×ULN;
• Left ventricular ejection fraction <45%;
• Baseline calculated eGFR<60mL/min/1.73m2;
• Pulmonary function:FEV1/FVC<60% and/or diffusion capacity of carbon monoxide (DLco) <60% of prediction;