appConfigEntityId

bodyType

customContents

entityId

entityUuid

isVideo

articlePageQuery

article

articleSchemaContent

content

hooksArticlePageAdvertisementConfigArticle

hooksArticlePageFloatingButtonConfigArticle

appConfig

filter

json

liveArticle

Dannie Peng

The death of a six-year-old girl after receiving an experimental gene-editing therapy has been described by scientists as a tragedy that highlights the need to improve trial safeguards.
But some in the biotechnology industry fear Chinese policymakers may curtail the regulatory flexibility that scientists have long enjoyed in initiating clinical trials – a key mechanism behind China’s competitive edge in frontier treatments.
“I hope this tragedy does not have a lasting negative impact on China’s investigator-initiated clinical-research system, but there is a real risk of a regulatory backlash,” said Wang Haoran, chief executive of Neoland Biosciences, based in Shandong province.
“China has advanced rapidly in several frontier areas, partly because the mechanism has provided greater flexibility for carefully supervised early clinical exploration. That speed has increased the sense of urgency across the biotechnology industries in the United States and Europe.
“This case appears to reveal serious failures in risk assessment, transparency and institutional oversight. Stronger safeguards are clearly necessary, especially for first-in-human gene-editing treatments involving children.”


The girl, identified only as Mei, died days after receiving a bespoke therapy designed to correct a neurodevelopmental genetic mutation in March last year, according to a joint investigation by Retraction Watch and the journal Science published on July 23.
The revelations sent shock waves through global research and biomedical communities, with the report framing the tragedy as “a new stumble in China’s push to rival the United States as a biotech power”.
The trial was led by Qiu Zilong, a neuroscience researcher at Shanghai Jiao Tong University’s brain centre, through an investigator-initiated trial (IIT), according to the report.
Under China’s IIT framework, researchers need clearance only from research hospitals rather than national regulators to test novel therapies on humans – an institutional set-up that has helped drive China’s biotech boom.
Unlike traditional clinical trials requiring rigorous approval from national authorities, IITs offer a nimble route for companies and institutions to determine whether experimental treatments are promising, according to industry outlet Endpoints News in an article published in December.
This mechanism had helped China’s biotech sector catch up with Western rivals, particularly by cementing China’s position as a competitive force in cell and gene therapy, the article said.
However, Wang criticised the culture among some Chinese researchers, particularly younger academics whom he said rushed to generate quick clinical data to secure grants, projects and promotions.
“In this case, many scientific and technical problems had not been solved, yet the principal investigator still pushed forward brashly,” Wang said.
“Cell and gene therapies are often easy to design, but the closer they get to clinical application, the harder they become. Academics often underestimate that difficulty.”

Mei suffered from Snijders Blok-Campeau syndrome, a rare neurodevelopmental disorder caused by mutations in the CHD3 gene, which can lead to developmental delays, speech impairment, intellectual disability and autistic traits. Her symptoms, however, were relatively mild.
In July 2023, Mei’s father contacted Qiu, saying the family was willing and able to cover the research and treatment costs. After months of preparation, the trial was conducted in March last year at the Shanghai-based Xinhua Hospital.
Qiu’s team used an adeno-associated virus (AAV) vector – the most common delivery vehicle in gene therapy – to deliver a base editor into Mei’s cerebrospinal fluid, aiming to alter the disease-causing CHD3 gene in her brain.
Had it succeeded, Mei would have been the first person in the world to receive a brain-directed gene-editing therapy. Instead, she died days later of a severe immune reaction reportedly linked to the treatment.
The therapy carried high risks, mainly because of the massive viral dose required, according to several life scientists.
Because only a small fraction of these viruses reach their target cells and activate the base-editing machinery, correcting enough brain cells would demand hundreds of trillions of viral particles.
“Gene-editing therapies using AAV vectors are highly dose-dependent in terms of toxicity; and because Qiu’s therapy was injected directly into the cerebrospinal fluid, the toxicity profile may have been different,” said Cui Xiang, a senior scientist at the multinational biotech firm, Novartis.
The investigation revealed that all four monkeys receiving the therapy during preclinical testing developed moderate to severe liver damage regardless of dosage, yet the human trial was not suspended.
Cui noted that the hospital ethics committee had failed to adequately verify the safety of the technology or properly assess the treatment’s risk-benefit ratio.
The Novartis scientist pointed out that while an infant with a life-threatening metabolic disorder successfully received a personalised CRISPR gene therapy in the US in May last year, Mei’s condition was “far from life-threatening”.
This incident might “cause a long-lasting impact” on the reputation of China’s rapidly rising biotech industry, Cui said.
China’s efficiency and lower development costs have attracted international pharmaceutical giants seeking cheaper cutting-edge assets and clinical research capabilities.


But whether this incident would change how foreign investors and multinational drug makers viewed China’s biopharmaceutical sector remained to be seen and deserved close attention, Cui said.
If, in the aftermath, the Chinese government handles the case seriously and strengthens oversight through legislation – including imposing tougher penalties for violations – it may yet be able to rebuild investors’ confidence, according to Cui.
However, a bioethics expert cautioned that the full impact could only be evaluated once a comprehensive investigation was published.
“Currently, the relevant information comes mainly from the media, and an authoritative independent third-party investigation has not yet been published. If what is said in the Science report is true, this exposes many loopholes in ethics reviews, scientific regulation and institutional oversight,” said Zhai Xiaomei, executive director of the Centre for Bioethics at the Chinese Academy of Medical Sciences.
“We should adhere to laws, regulations and objective facts when judging this case, and not be influenced by public opinion.
“We look forward to the national independent investigation publishing the truth as soon as possible so that further judgments can be made based on the facts.”


T. rex was warm-blooded and had body temperature close to ours, study finds

China uses 100,000 home-grown AI chips to build leading weather forecast system

Geologists find unexpectedly large water sources in Xinjiang beneath China’s largest desert

Chinese scientists build world’s smallest CT scanner, weighing just 6kg

Science

Is China’s biotech boom at risk? Fear of policy backlash after death in gene-editing trial

Scientists in the biotech sector fear China will impose restrictive regulations after a child died in a gene-editing trial in 2025. Photo: Shutterstock

The trial on “Mei” was conducted at Xinhua Hospital affiliated with Shanghai Jiao Tong University medicine school. Photo: Handout

Chinese scientist faces probe after girl, 6, dies ‘following experimental gene therapy’

Shanghai’s XInhua Hospital approved the treatment. Photo: Handout

Lab monkey prices at record highs as China’s biotech boom fuels windfall for breeders

China accounts for about 30 per cent of all new medicines currently under development worldwide, ranking second globally. Photo: Shutterstock Images

Some primate breeding farms were now quoting crab-eating macaques at around 200,000 yuan each. Photo: Shutterstock Images

Record Chinese biotech out-licensing meets rising US scrutiny

Washington is increasing scrutiny of Chinese biotech at a time when the innovation pipeline in the world’s second-largest economy is experiencing rapid growth. Photo: Shutterstock

US biotech firm developing therapies for infant brain injuries plans Hong Kong listing

Axiom Biosciences joins a swelling roster of healthcare firms seeking IPO approval in Hong Kong. Photo: Shutterstock

Axiom would be the first biotech firm operating in the US to list in Hong Kong, according to Remo Moomiaie-Qajar. Photo: Handout

China’s biotech firms challenge US with rapidly developing RAS-targeted cancer therapies

Chinese biotech and innovative drug firms are rapidly closing the gap with its US rivals as it continues to develop RAS-targeted cancer therapies. Photo: Shutterstock

A worker checks a bottle filling fixture at a preparation workshop at Hengrui Biomedical Industrial Park in Lianyungang, eastern China’s Jiangsu province on December 13, 2021. Photo: Getty Images

News

China

Scientist says child’s death reveals ‘serious failures in risk assessment, transparency and institutional oversight’


Scientist says child’s death reveals ‘serious failures in risk assessment, transparency and institutional oversight’.


Is China’s biotech boom at risk? Fear of policy backlash after death in gene-editing trial

.css-1c6uqr6{color:inherit;font-weight:inherit;font-size:inherit;font-family:inherit;line-height:inherit;overflow-wrap:break-word;}Scientist says child’s death reveals ‘serious failures in risk assessment, transparency and institutional oversight’

Scientist says child’s death reveals ‘serious failures in risk assessment, transparency and institutional oversight’