Patient death in China gene-editing trial draws fire: Journal

SHANGHAI – A young girl died after receiving an experimental gene-editing treatment in a clinical trial in Shanghai, Science Journal reported, raising questions about regulatory oversight and transparency as China rapidly expands its biotechnology ambitions.The six-year-old, who had a genetic mutation that affected her neurodevelopment, received a treatment designed to correct the underlying defect at Shanghai Xinhua Hospital in March 2025, according to an investigation by Science and Retraction Watch. She died days later from a severe immune reaction linked to the therapy, the magazine reported, citing documents and accounts provided by the girl’s parents. The death had not been previously disclosed, and was not mentioned in a Nature paper published earlier in 2026 detailing animal studies of the therapy, which uses a technology known as base editing to precisely alter a single DNA letter. The paper also omitted troubling kidney and liver issues that emerged in earlier monkey experiments, according to Science.Qiu Zilong, a neuroscientist at Shanghai Jiao Tong University School of Medicine who led the effort to create the therapy, and Yongguo Yu, the doctor overseeing the study, did not immediately respond to a request for comment, and calls to Xinhua Hospital were unanswered. A call to the phone number listed on ClinicalTrials.gov page referenced by Science was answered by someone who said they were not involved in the study and knew nothing about it.The case highlights the opaque nature of some investigator-initiated trials, or IITs, in China, in which researchers at major hospitals test novel therapies after local review, without approval from the national drug regulator. Such studies have surged in recent years, growing 11-fold from 2015 to 2023 to top 1,000. Deals spreeThe IIT pathway has helped accelerate development of cutting-edge cell and gene therapies, and promising data have promoted Western drugmakers to license drugs from Chinese biotechs.The hospital was fined about US$3,600 (S$4,650) for its failures in the trial, and Yu, the physician responsible, received verbal counselling. The child’s parents, who contributed US$860,000 to the research and clinical work for her treatment, were offered no compensation, according to the Science report.China has worked to tighten oversight of emerging medical technologies since scientist He Jiankui shocked the world by creating the world’s first genetically altered babies, testing the balance between supervision and its ambition to lead in biotech innovation.In June, China revamped regulations around cell and gene therapy research, restricting cutting-edge trials to certain hospitals and banning institutions from charging patients fees related to clinical research. At the same time, it outlined ways for qualified hospitals to commercialise some advanced procedures such as cell therapies, without requiring traditional drug registration. BLOOMBERG