GOP lawmakers call on FDA to increase scrutiny of Chinese clinical trial data after gene therapy deaths

Two Republican House members, John Moolenaar and Ben Cline, are calling on the FDA to reject clinical trial data from China unless trial sites undergo recent FDA audits, following three patient deaths in gene therapy trials conducted in China. The deaths include a boy with Duchenne muscular dystrophy who died in August 2025 after receiving HuidaGene's CRISPR therapy, a six-year-old girl who died seven days after receiving brain-directed gene editing for Snijders Blok-Campeau syndrome in May 2025, and a systemic sclerosis patient who died after RiboX Therapeutics' CAR T therapy in August 2026. The lawmakers' request comes as China has overtaken the U.S. in annual registered trials since 2020, with China's regulator approving 94 oncology drugs between 2020-2025 compared to the FDA's 87.

Gene therapy deaths in China

• ▪A young boy with Duchenne muscular dystrophy died in August 2025 after receiving HuidaGene's investigational CRISPR-based therapy HG302.
• ▪In May 2025, a six-year-old girl diagnosed with Snijders Blok-Campeau syndrome received the world's first gene-editing therapy directed at the brain.
• ▪The investigational gene-editing therapy for Snijders Blok-Campeau syndrome was administered at Xinhua Hospital, which is affiliated with the Shanghai Jiao Tong University School of Medicine.
• ▪Shanghai Jiao Tong University School of Medicine launched an internal investigation into the gene-editing trial after the Science report, an effort led by neuroscientist Zilong Qiu.
• ▪On August 9, 2026, RiboX Therapeutics reported a death in a patient with systemic sclerosis who had received the biotech's experimental in vivo CAR T cell therapy.
• ▪The boy who received HuidaGene's HG302 therapy 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.
• ▪HuidaGene submitted full findings on the death for peer review in January 2026.

Congressional FDA scrutiny request

• ▪Representatives John Moolenaar (R-MI) and Ben Cline (R-VA) penned a letter to Acting FDA Commissioner Kyle Diamantas requesting specific measures to lower patient risk in the U.S. following three patient deaths in gene therapy clinical trials in China.
• ▪John Moolenaar chairs the House Select Committee on China.
• ▪Ben Cline is a member of the BIOtech Caucus.
• ▪Representatives John Moolenaar and Ben Cline are asking the FDA to conduct a wide-ranging review of products that have received U.S. approval based on clinical results from China.
• ▪Representatives John Moolenaar and Ben Cline are asking the FDA to reject clinical data from China unless the trial site has recently undergone an FDA audit.

China clinical trial growth

• ▪By 2020, China had overtaken the U.S. in annual registered trials.
• ▪The FDA approved 87 oncology drugs between 2020 and 2025.
• ▪In 2010, fewer than 8% of global clinical trials were run in China.
• ▪China's National Medical Products Administration greenlighted 94 oncology drugs between 2020 and 2025.

U.S. trial modernization challenges

• ▪The Department of Health and Human Services rolled out a new clinical trial modernization initiative designed to speed up early trials in hopes of bringing them back to the U.S.
• ▪The FDA launched a new investigational new drug pilot program as part of HHS's broader clinical trial modernization initiatives.
• ▪Major delays in early phase clinical trials occur mainly at the site level rather than from the FDA.
• ▪Early-phase trials conducted in Spain, Australia or China come in more quickly, more cost effective and with less administrative burden when compared to U.S. trials.

Perspective of Republican lawmakers John Moolenaar and Ben Cline

• ▪Representatives John Moolenaar and Ben Cline believe the three patient deaths in Chinese gene therapy trials demonstrate a need for specific measures to lower patient risk in the United States.
• ▪Representatives John Moolenaar and Ben Cline view clinical data from China as requiring FDA on-site audits before being accepted for regulatory purposes.

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