A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

Key Inclusion Criteria：

• 6 to 35 years old(inclusive) male or female subjects at the time of informedconsenting

• Diagnosis of β-thalassemia, genotypes include but are not limited to β+β0，βEβ0，β0β0,etc

• History of at least≥8 units/year of packed RBC transfusions in the prior 12 monthsprior to the screening period

• Generally in good condition, Karnofsky performance score≥60 points for subjects≥16years old at the time of autologous hematopoietic stem cell collection, or LanskyPlay-Performance score≥60 points for subjects under 16 years old, or equivalentclinical evaluation as the investigator site's common practice

Key Exclusion Criteria:

• Treatment with other investigational medications or other experimental interventions 30 days prior to signing informed consent or within 6 half-lives of the drug,whichever is longer.

• Subjects who have received or are receiving thalidomide and/or Luspatercept, whentheir drug-drug interaction on the efficacy and safety of CS-101 cannot be ruledout, unless at least there are 3 test results showing the total hemoglobin levelbefore transfusion is below 9g/dL in the past 6 months before screening.

• Previously received allogeneic hematopoietic stem cell transplantation orgene(edited) therapy.

• Subjects have available related fully matching donors and are eligible and preparedfor allogeneic hematopoietic stem cell transplantation.

• Those with active infections, including but not limited to: HIV, hepatitis B,hepatitis C, cytomegalovirus, Epstein-Barr virus and treponema pallidum testpositive, or known tuberculosis, parasitic infection, etc. who are judged by theinvestigator to be unsuitable to participate in this study.

• Echocardiography results with ejection fraction below 45%.

• Advanced liver disease, defined as：

Aspartate aminotransferase (AST), alanine aminotransferase (ALT) >3 × upper limit of normal (ULN) or:

Baseline International Normalized Ratio (INR) >1.5 × ULN.

• MRI during the screening period showed heavy iron overload and is judged by theinvestigator to be unable to participate in the study.