Child died in Chinese biotech firm’s gene editing trial

HONG KONG – A child with Duchenne muscular dystrophy died after receiving a gene-editing therapy in China, sparking new concerns about regulatory oversight and transparency as China seeks to cement its position as a biotechnology leader.

In a statement on Aug 5, the treatment’s developer, Shanghai-based HuidaGene Therapeutics, attributed the August 2025 death to acute respiratory distress syndrome as part of a severe immune reaction to the therapy.

Though the company did not identify or provide details about the patient, clinical trial records show the study only enrolled 4- to 8-year-old boys.

HuidaGene’s disclosure follows a months long investigation by STAT News. The firm did not immediately respond to questions on next steps for the therapy, and could not be reached by phone.

The announcement also comes weeks after Science revealed the previously unreported death of a young girl in a separate investigator-initiated trial (IIT), a type of study led by researchers rather than pharmaceutical companies.

Chinese authorities and the Shanghai university affiliated with the study have since launched probes.

The cases highlight the opaque nature of China’s IIT system, which permits researchers at major hospitals to test novel therapies without approval from the national drug regulator.

Such studies have surged in recent years, helping accelerate the development of cutting-edge cell and gene therapies that are fuelling China’s biotech boom.

But the deaths are likely to intensify scrutiny of whether oversight, safety reporting and public disclosure have kept pace with the sector’s rapid growth.

The patient was the fourth and last participant to receive HuidaGene’s HG302, which uses modified viruses known as adeno-associated virus (AAV) vectors to deliver DNA-editing components to cells.

The patient also received a high dose. High-dose AAV administration has caused fatal immune reactions in other clinical trials around the world, including in young boys with Duchenne. 

HuidaGene said the fatal event was reported through the hospital’s ethics and study-oversight processes “within the applicable reporting time frame after the site became aware of it, in accordance with Good Clinical Practice”.

The analysis was submitted for peer review in January and findings will be shared upon publication, the company said. BLOOMBERG