A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia

Exclusion Criteria

• •1. History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
• •2. Diagnosed with compound alpha-thalassemia.
• •3. Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
• •4. White blood cell count (WBC) \<3×10\^9/L and/or platelet count \<100×10\^9/L not due to hypersplenism, as judged by the investigator.
• •5. Uncorrected bleeding disorders.
• •6. Received treatment with erythropoietin (EPO) within the three months prior to enrollment.
• •7. Severe iron overload, with serum ferritin levels ≥5000 ng/ml.
• •8. Positive for hepatitis B surface antigen (HBsAg), antibodies to hepatitis C virus, antibodies to human immunodeficiency virus (HIV), or specific antibodies to Treponema pallidum (syphilis).
• •9. History of hematopoietic stem cell transplantation, gene therapy, or gene editing therapy.
• •10. Participation in another clinical study and use of investigational drugs within 3 months prior to starting the study drug.
• •11. History or current presence of malignant tumors or myeloproliferative diseases or immunodeficiency diseases.
• •12. Presence of severe mental illness that prevents cooperation with treatment; significant pulmonary arterial hypertension requiring medical intervention; recent malaria; a history of hematological tumors in immediate family members.
• •13. Any past or current disease, treatment, or laboratory abnormality that may interfere with the study results, affect the patient's full participation in the study, or that the investigator deems unsuitable for participation in this clinical study.