A four-year-old Pakistani girl suffering from severe thalassemia has made a full recovery after receiving an innovative gene-editing treatment in China, marking the first time a foreign minor has been successfully treated with the cutting-edge therapy. According to China Daily, the milestone was achieved at the Children’s Hospital of Fudan University in Shanghai, where the girl, nicknamed Aiza, underwent the experimental procedure earlier this year. Previously dependent on regular blood transfusions to survive, she has now resumed a normal, healthy life.
Aiza’s condition dramatically improved after receiving CS-101, a base-editing drug developed to treat severe beta-thalassemia. Her parents brought her to Shanghai in January as part of a clinical research initiative led by Professor Zhai Xiaowen in partnership with CorrectSequence Therapeutics, a Shanghai-based biotech firm. The hospital reported that the therapy eliminated her need for transfusions, a breakthrough that could offer hope to thousands of children with similar blood disorders.
On Tuesday, a small ceremony was held at the hospital to celebrate Aiza’s recovery, attended by her family, medical staff, and researchers involved in the project. The successful treatment highlights China’s advancements in gene-editing technology, positioning it as a leader in next-generation medical solutions for genetic diseases. Professor Zhai emphasized that this case could pave the way for broader clinical trials and eventual global accessibility of the therapy.
Thalassemia, a genetic blood disorder affecting millions worldwide, particularly in regions like South Asia, often requires lifelong treatment. Aiza’s recovery not only represents a personal triumph but also a significant step forward in precision medicine. Experts believe this success could accelerate the development of similar therapies, offering new possibilities for patients in countries with limited medical resources. Pakistan, where thalassemia is prevalent, may explore further collaborations with Chinese institutions to expand access to such treatments.