China gene therapy trials linked to deaths of two children

Two children have died following separate experimental gene-editing clinical trials in China, sparking international concerns regarding regulatory oversight and biomedical ethics.

One fatality involved a boy during a trial conducted by the Shanghai-based company HuidaGene Therapeutics. The second death involved a six-year-old girl receiving a treatment for Snijders Blok-Campeau syndrome. This trial was led by neuroscientist Zilong Qiu at Shanghai Jiao Tong University and clinician Yongguo Yu at Xinhua Hospital. Reports indicate the girl died from a severe immune reaction and microangiopathy following the administration of an adenine base editor delivered via an AAV vector.

These incidents have highlighted risks associated with investigator-initiated trials (IITs), which allow researchers to test therapies with less direct supervision from drug regulators. Experts suggest these deaths may damage the credibility of Chinese scientific institutions and could lead to stricter government regulations for the country’s fast-track clinical trial processes.