This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with transfusion-dependent β-thalassemia; to preliminarily evaluate the impact of a single -dose of YOLT-204 on the levels of fetal hemoglobin in the plasma
Age between 18 to 35 years, gender not limited.
The patient has fully understood this study and voluntarily signed an informed consent form.
Clinically diagnosed as a patient with transfusion-dependent β-thalassemia, excluding the genotype: β0β0.
Karnofsky Performance Status (KPS) score of at least 70.
History of multiple drug allergies or a history of allergic reactions to oligonucleotides or lipid nanoparticles (LNPs).
Diagnosed with compound alpha-thalassemia.
Clinically significant active bacterial, viral, fungal, or parasitic infections at the time of screening, as judged by the investigator.
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.