China reins in a fast route for gene therapy trials after patient deaths

A shortcut that made China one of the fastest places in the world to test new gene and cell therapies in people has slowed sharply. STAT reported on November 5 that what had been a flood of controversial trials has “slowed to a trickle,” leaving families of people with rare diseases in limbo as approvals go through what it called a “shakedown” phase¹. The change followed deaths, news reports and new government rules this summer, according to the outlet¹.

What changed

The shortcut is known as an investigator-initiated trial, or IIT. In plain terms, it is a study started by the doctors running it rather than by a drug company going through the national regulator. In China, this route allowed investigators to begin clinical trials without sign-off from central regulators¹. A business outlet describing the system in July said institutions could manage certain early-stage studies with hospital-level ethical approval instead of national approval².

That looser setup let Chinese companies test drugs in humans faster and more cheaply¹. It also pulled in foreign players: according to STAT, it prompted US companies either to come to China or to push the US Food and Drug Administration to adopt a similar approach¹.

Now, STAT reports, the government has become “very strict,” in the words of one patient advocate¹. The reporting available to Biohack does not spell out the specific new requirements, such as which trials now need national approval or what extra safety data regulators want. That detail matters and remains to be confirmed.

The case that drew scrutiny

One case put the system under a spotlight. In late July, Business Fortune reported on the death of a 6-year-old girl, identified as Mei, after an experimental gene-editing treatment². She had Snijders Blok-Campeau syndrome, a rare genetic disorder linked to changes in a gene called CHD3².

The treatment was personalized to her. Researchers tried to correct the faulty gene using base editing, a form of gene editing that changes single letters of DNA, delivered by an AAV vector, a harmless virus used to carry genetic material into cells². It was given through an investigator-initiated trial at Xinhua Hospital in Shanghai².

According to that report, earlier testing in animals had reportedly shown serious problems, and experts noted that risks can rise when large doses of these viral carriers are used². Bioethics specialists quoted there said the full picture would depend on an independent investigation and verified findings². STAT refers to “deaths” in the plural as part of what changed this summer¹, but the sources available here describe only this one case in detail.

Families caught in the middle

STAT’s report centers on Juan Huang, a former engineer in Shanghai, and her 13-year-old daughter, Huangxi¹. In 2014, at 16 months old, Huangxi suddenly could no longer stand or walk, and doctors in Shanghai had no answers¹. It took two years and a flight to Massachusetts to diagnose a genetic disease that had never been identified in China, and even doctors at Boston Children’s Hospital could not offer any treatment¹.

In the decade since, STAT reports, Chinese hospitals became sophisticated centers for diagnosing and treating rare disease¹. Huang now works for the Hope for Rare Foundation, which helped push genetic treatments into trials at a fraction of what they would cost in the US¹. The foundation was on track to start two more trials until this summer’s events upended those plans¹.

Her situation shows the trade-off at the heart of the story. A fast, cheap route can get experimental treatments to children with diseases that drug companies may never take on. The same speed can mean less outside checking before a first-in-human test.

Old worries about light oversight

Concerns about China’s hospital-led route are not new. In 2018, Gizmodo, summarizing reporting by The Wall Street Journal, wrote that doctors in China could proceed with a clinical trial after approval from their hospital’s ethics board³. At that time, at least 86 patients in China had had their DNA edited with CRISPR, a gene-editing tool, as of January 2018³.

That report described one trial run by a private startup, Anhui Kedgene Biotechnology, that lost touch with patients whose DNA had been altered; the company’s founder said the trial did not finish as planned³. A doctor who ran another of its trials, in 18 cancer patients, said he had been asked by the science ministry for a report, the first time Beijing had sought information since the trial began more than a year earlier³. Long-term follow-up matters for gene therapies because unintended effects could appear years later, the article noted³.

Those 2018 reports came weeks after scientist He Jiankui claimed to have created the world’s first gene-edited babies³. The current tightening follows a long run of debate over how much freedom hospitals should have.

What it means for companies and investors

China’s biotech industry has become a major global competitor in genetic medicine, attracting international drugmakers looking for research capacity and lower development costs². Analysts quoted in July said the outcome of the girl’s case could affect investor confidence and future partnerships². Some industry figures also said researchers may face pressure to produce quick results for academic recognition and funding².

For US companies that moved early human testing to China because of the IIT route, or that pointed to it while lobbying the FDA for a similar path¹, a slowdown could change timelines. The sources here do not name specific programs that have been paused or delayed, beyond the two trials Huang’s foundation had planned¹.

What is still unknown and what to watch

• The rules themselves. The available reporting describes stricter oversight but not the text of the new rules or when each part takes effect.
• The investigation. Whether an independent review of the Shanghai girl’s death is published, and what it finds about the animal data and the approval process².
• Whether approvals pick up again. STAT calls the current period a “shakedown” phase¹, which leaves open whether this is a pause or a lasting shift.
• The US debate. Whether the slowdown changes the case some companies have made for a faster, China-style path at the FDA¹.
• Rare-disease families. Whether foundations like Huang’s can move their planned trials forward under the new system¹.

Biohack will follow this thread as the rules, the investigation and individual trials develop.