WEB DESK: A 4-year-old Pakistani girl with severe thalassemia has been successfully treated with a Chinese-developed gene-editing drug, marking the first time the technology has been used on a foreign minor, according to the Children’s Hospital of Fudan University in Shanghai.
The young patient, affectionately called Aiza, was reliant on frequent blood transfusions to sustain her life. However, after receiving the novel treatment earlier this year, her condition improved dramatically, eliminating her dependency on transfusions and allowing her to resume a normal childhood.
Aiza traveled to Shanghai in January with her family to undergo experimental treatment with CS-101, a cutting-edge base-editing drug designed to address severe beta-thalassemia. This experimental therapy is part of a clinical research program initiated in 2023, led by Professor Zhai Xiaowen and in collaboration with CorrectSequence Therapeutics, a biotechnology company based in Shanghai.
According to the hospital, four patients, including Aiza, have now received the therapy, with initial results demonstrating promising safety and effectiveness.
Dr. Qian Xiaowen, who oversaw Aiza’s treatment, stated, “Thanks to the careful and coordinated efforts of our multidisciplinary team, her hemoglobin levels now exceed 100 grams per liter, enabling her to live without transfusions. The entire process was smooth and encouraging.”
Medical professionals emphasized that children with severe thalassemia typically require lifelong monthly transfusions and face significant health risks if untreated. This successful case offers hope for future treatments and potentially curative options for similar patients worldwide.
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