This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
Age 3-35 years old (inclusive), male or female;
The subject and/or his/her legally recognized representative/parent/guardian fully understands the study and all information related to the study and has signed the informed consent form;
Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)/kg/year (or ≥ 100 mL/kg/year) or ≥ 8 times/year of suspended RBC transfusions in at least 1 consecutive 12-month period;
Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged < 16 years) of ≥ 80;
Diagnosis of associated α-thalassemia: > 1 alpha chain deletion or alpha gene functional defect;
Have available HLA-fully matched donors and acceptable for allogeneic hematopoietic stem cell transplantation;
Irregular antibody or platelet antibody positive;
Prior allogeneic bone marrow transplantation or gene therapy;
Single group
VGB-Ex01
Biological/VaccineCRISPR-Cas12b editing hematopoietic stem cells
Incidence of adverse events and serious adverse events
An adverse event is any untoward medical occurrence in a clinical investigation/participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event is any untoward medical occurrence at any dose that resulted in death; life threatening;require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.
Number of subjects with neutrophil implantation ≤ 42 days
Neutrophil implantation was defined as 3 consecutive days with 3 tests for ANC≥500/μL.
Number of subjects transfusion independence (TI) for at least 6 months after transfusion
TI defined as Hb≥9g/dL without red blood cell infusion
Number of subjects transfusion independence (TI) for at least 12 months after transfusion
TI defined as Hb≥9g/dL without red blood cell infusion
Fetal hemoglobin (HbF) concentration
Changes in HbF concentration from baseline after transfusion
Total hemoglobin (Hb) concentration
Changes in Hb concentration compared with baseline after transfusion
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Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborator
