Pakistani Girl cured through Chinese gene therapy

Pakistani Girl cured through Chinese gene therapy and a small event was organized to celebrate the remarkable recovery.

A four-year-old Pakistani girl suffering from severe thalassemia has become the first foreign minor to be treated successfully with a Chinese-developed gene-editing drug, according to Shanghai’s Children’s Hospital of Fudan University.

The hospital organized a small event on Tuesday to celebrate the recovery of the girl, affectionately known as Aiza. Before receiving treatment earlier this year, she had depended on regular blood transfusions. Following gene-editing therapy, she no longer needs transfusions and has resumed a normal lifestyle.

Aiza arrived in Shanghai with her parents in January. Her treatment involved a base-editing drug called CS-101, developed for severe beta-thalassemia. This therapy is part of a clinical trial launched in 2023 by Professor Zhai Xiaowen in partnership with CorrectSequence Therapeutics, a biotech company based in Shanghai.

So far, four patients including Aiza have undergone the therapy, which has shown promising results in both safety and effectiveness, according to the hospital.

Dr. Qian Xiaowen, who led Aiza’s care, stated that with the comprehensive efforts of the medical team, her hemoglobin levels surpassed 100 grams per liter—sufficient to free her from blood transfusions. The treatment went smoothly throughout.

Doctors highlighted that individuals with severe thalassemia typically need lifelong monthly transfusions and may face life-threatening complications without them.

Muhammad Adeel, Aiza’s father and a 40-year-old physicist who once held a postdoctoral position in Hong Kong, expressed heartfelt thanks to the medical team by offering flowers and a card.

Adeel shared that the support they received extended beyond physical treatment—medical staff also provided emotional encouragement during the difficult months when Aiza’s mood fluctuated due to the illness and its demands.

Aiza is the second of three siblings; the other two children are healthy.

While still in Hong Kong, Adeel began searching for potential treatments. In April last year, he found out online that this gene-editing therapy had been used successfully in young patients and subsequently contacted the biotech firm.

At Tuesday’s event, the hospital presented Aiza with gifts and conveyed hopes for her long-term well-being. CorrectSequence Therapeutics also gave a card that wished her a joyful and fulfilling life, noting that her smile would bring light to others.

Thalassemia, a hereditary blood disorder, is particularly prevalent in regions like the Mediterranean, Middle East, Africa, Southeast Asia, and southern China.

For Aiza’s treatment, doctors harvested her own hematopoietic stem cells and used CS-101 to edit specific genetic sequences. This activated necessary protein expression and restored hemoglobin’s oxygen-carrying function. The modified cells were then reintroduced into her body.

As a result, Aiza’s hemoglobin reached healthy levels, eliminating her dependence on transfusions.

Professor Zhai, also the hospital’s vice president, remarked that this achievement could accelerate the global application of China’s homegrown gene-editing therapies and potentially offer a one-time cure for patients worldwide.

He further noted that ongoing research aims to expand the use of this gene-editing technology to treat other inherited metabolic disorders in children.