4-Year-Old Pakistani Girl Cured of Thalassemia Using Chinese Gene-Editing Breakthrough

Aiza becomes the first foreign child successfully treated with Chinese-developed CS-101 gene therapy

A groundbreaking milestone in gene therapy has been achieved as a 4-year-old Pakistani girl named Aiza has been cured of severe beta-thalassemia through a revolutionary Chinese gene-editing drug. This marks the first instance of a foreign minor being successfully treated with China’s domestically developed therapy, according to China Daily, citing the Children’s Hospital of Fudan University in Shanghai.

Previously dependent on frequent blood transfusions, Aiza’s life has been transformed after receiving CS-101, an experimental base-editing gene therapy developed to treat critical cases of beta-thalassemia. The treatment, initiated in January 2025, was carried out under the supervision of Professor Zhai Xiaowen and Dr Qian Xiaowen, in partnership with CorrectSequence Therapeutics, a Shanghai-based biotech firm.

“The child’s hemoglobin levels exceeded 100 grams per liter, eliminating her need for transfusions,” stated Dr Qian. “The treatment progressed smoothly with zero complications.”

Aiza is one of four patients globally to have received CS-101 so far, and doctors report promising safety and efficacy outcomes.

💡 The Breakthrough Therapy:

• Doctors extracted Aiza’s autologous hematopoietic stem cells
• Applied the CS-101 gene-editing drug to modify specific genes
• Re-infused the edited cells, restoring normal hemoglobin production

This cutting-edge therapy reactivated key protein expressions and restored the oxygen-carrying function of her blood, allowing Aiza to live a transfusion-free, normal life.

Her father, Muhammad Adeel, a 40-year-old physicist and former postdoctoral researcher in Hong Kong, praised the exceptional medical and emotional care his family received throughout the journey. He had found out about the experimental treatment in 2024 and reached out to CorrectSequence Therapeutics.

At a special ceremony hosted by the hospital, Aiza received gifts and well-wishes for her bright future. The biotech company presented a touching card:

“Wish your life be filled with prosperities and happiness. Your smile and love shall enlighten more lives in the world.”

🌍 A Hope for Millions

Thalassemia is a life-threatening inherited blood disorder prevalent across South Asia, the Mediterranean, the Middle East, and parts of China. Traditionally, patients face a lifetime of monthly transfusions and complications. This breakthrough offers real hope for a one-time cure, potentially transforming the lives of millions of children around the world.

Professor Zhai emphasized that the hospital and CorrectSequence Therapeutics are now exploring applications of this gene-editing platform for other genetic metabolic disorders in children.