China has established a robust clinical research framework for thalassemia gene therapy through collaboration among leading medical centers and biotech companies. With the accumulation of positive clinical data and optimization of protocols, gene therapy is poised to offer a potential functional cure for many thalassemia patients, demonstrating China's growing capability and potential in the global advanced therapy landscape.

Thalassemia is an inherited hemolytic anemia caused by globin gene defects. First discovered along the Mediterranean coast, it derives its name from this region. Its core characteristic is impaired hemoglobin (Hb) synthesis due to globin gene mutations, leading to ineffective erythropoiesis and ultimately chronic anemia of varying severity.