A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

Inclusion Criteria:

• 12 to 35 years old(inclusive) male or female subjects at the time of informedconsenting. For minors, their legal representative is required to sign the informedconsent form, besides, if the subjects aged 8 years or older, they should provide asigned and dated.

• Diagnosis of β-thalassemia major.

• Generally in good condition, Karnofsky performance score≥60 points for subjects≥16years old, or Lansky Play-Performance score≥60 points for subjects under 16 yearsold.

• For female subjects of childbearing potential: use effective contraceptive measuresfrom the start of screening and agree to continue using such measures forcontraception throughout the study

• For male subjects who have a potential ability to father a child: use condoms orother methods continuously from the start of mobilization to ensure effectivecontraception for sexual partners during the study period

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 in thepast 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

• Patients with coexisting α-thalassemia and more than 2 deletions or non-deletionalmutations in the α-globin chain coding genes

• Known to be allergic to drugs used during autologous hematopoietic stem celltransplantation (including but not limited to granulocyte colony-stimulating factor,busulfan, dextran), excipients(such as dimethyl sulfoxide), or instruments(such asintravenous catheters) as determined by the investigator are deemed unsuitable toparticipate in this study

• Those with positive results in HIV, cytomegalovirus, Epstein-Barr virus andtreponema pallidum test, active infection of hepatitis B, hepatitis C, or knowntuberculosis, parasitic infection, etc. Hepatitis B stabilized on medication(HBV-DNAtest negative) and cured hepatitis C(HCV-RNA test negative) can be consideredacceptable.

• Echocardiography shows ejection fraction below 45%

• Laboratory indicators, defined as：Aspartate aminotransferase(AST), alanineaminotransferase(ALT) >3× upper limit of normal(ULN) or Baseline InternationalNormalized Ratio(INR)>1.5×ULN.

• MRI during the screening period shows severe cardiac iron overload and otherconditions, and are judged by the investigator to be intolerable or inappropriatefor autologous hematopoietic stem cell transplantation

• Patients with past/present history of cancer

• Known neurological disorders, psychological problems or mental illness, and isjudged by the investigator to be unable to cooperate with the study procedures

• Known history of uncontrolled epileptic seizures and is judged by the investigatorto be unfit to participate in this study

• The investigators determined that a non-hypersplenism-induced white blood cell countof<3×10^9/L, and/or a platelet count of<100×10^9/L.

• Known history of other serious cardiovascular, pulmonary, renal diseases, digestivetract conditions, liver diseases and / or other conditions, etc., and are judged bythe investigator to be intolerable or inappropriate for autologous hematopoieticstem cell mobilization, collection, and myeloablative conditioning and infusion

• Pregnant or lactating women