Lawmakers urge FDA to enact new China policies after 3 gene therapy deaths

 

Three fatalities occurring recently in China clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients.

After three patient deaths were reported in separate gene therapy clinical trials in China, two members of the House of Representatives are asking the FDA to implement specific measures to lower patient risk in the U.S.

Reps. John Moolenaar (R-MI) and Ben Cline (R-VA) have reportedly penned a letter to Acting FDA Commissioner Kyle Diamantas, writing that “accepting Chinese clinical data poses real risks for patients,” according to Endpoints News.

The two lawmakers are asking the agency to reject clinical data from China unless the trial site has recently undergone an FDA audit, and to also conduct a wide-ranging review of products that have received U.S. approval based on clinical results from China, according to the letter obtained by Endpoints.

“In short, the offshoring of early-stage clinical trials to China risks rewarding a system that has shown it is willing to treat children’s deaths as an acceptable cost of faster, cheaper research,” the lawmakers wrote, as cited by Endpoints.

As of publication, the House Select Committee on China—which Moolenaar chairs—and the BIOtech Caucus—of which Cline is a member—have not responded to BioSpace’s request for comment. The FDA also did not respond.

At least three patient deaths occurring in China trials have been reported over the last few months. One was the death of a young boy enrolled in a gene therapy study, biotech HuidaGene said in an Aug. 5 statement that followed a STAT investigation.

The boy had Duchenne muscular dystrophy (DMD) and died a year earlier, in August 2025, after receiving HuidaGene’s investigational CRISPR-based therapy, HG302. The child “developed acute respiratory distress syndrome in the setting of severe complement and cytokine activation following high-dose systemic administration of an adeno-associated virus vector,” according to HuidaGene.

Full findings on the death were submitted for peer review in January and will be shared upon publication, per the release. The biotech has not responded to BioSpace’s request for comment.

On Aug. 9, China-based RiboX Therapeutics reported a death in a patient with systemic sclerosis who had received the biotech’s experimental in vivo CAR T cell therapy—a gene therapy designed to generate CAR T cells within the body. The patient had “significant pre-existing medical conditions,” RiboX said. The company has not responded to BioSpace’s request for more information.

Finally, in May 2025, a six-year-old girl diagnosed with an extremely rare genetic neurodevelopmental disorder received the world’s first gene-editing therapy directed at the brain. The child had Snijders Blok-Campeau syndrome, which is caused by mutations in the CHD3 gene and is characterized by developmental delays, speech impairment and low muscle tone.

Seven days later, she died from a severe immune reaction tied to the therapy, Science reported last month.

The investigational therapy was administered at Xinhua Hospital, which is affiliated with the Shanghai Jiao Tong University School of Medicine. After the Science report, the university launched an internal investigation into the trial—an effort led by neuroscientist Zilong Qiu.

The death reports have ratcheted up U.S. scrutiny on China’s biotech scene across several facets, including national security and innovation concerns.

The Department of Health and Human Services recently rolled out a new clinical trial modernization initiative designed to speed up early trials in hopes of bringing them back to the U.S. However, experts say the effort by itself won’t do much to tip the scales.

“FDA is not the primary source of most delays in early phase clinical trials,” Harpreet Singh, a former division director of oncology at FDA, told BioSpace earlier this month. Major delays instead occur mainly at the site level.

For example, early-phase trials conducted in Spain, Australia or China are “going to come in more quickly, more cost effective and with less administrative burden,” when compared to U.S. trials, Singh said.

In 2010, fewer than 8% of global clinical trials were run in China, according to an April report from the National Bureau of Economic Research. By 2020, the Asian country had overtaken the U.S. in annual registered trials. China’s National Medical Products Administration also outpaced the FDA in terms of cancer approvals, greenlighting 94 oncology drugs between 2020 and 2025 compared to the 87 that entered the U.S. market in the same timeframe, per Health Affairs Scholar.

“I still am not fully clear on how HHS will address the major issues,” Singh said.

https://www.biospace.com/policy/lawmakers-urge-fda-to-enact-new-china-policies-after-3-gene-therapy-deaths