Third patient death revealed in China's CAR-T and gene therapy trials, spotlighting IIT safety and transparency

A third patient death has been uncovered in China’s largely opaque investigator-initiated clinical trials (IITs), this time in an in vivo CAR-T study for an autoimmune disease.

The patient, a man in his 50s with systemic sclerosis, died in March after receiving RiboX Therapeutics’ experimental in vivo CAR-T therapy RXIM002, which uses circular RNA delivered via lipid nanoparticles.

RiboX, a Shanghai-based biotech, recently obtained FDA clearance to start a U.S. trial of the same candidate but did not publicly disclose the earlier death in China until questioned by Endpoints News.

The death follows two other recent, initially undisclosed fatal events in Chinese gene-editing IITs:
a 6-year-old girl with a neurodevelopmental disorder and a young boy with Duchenne muscular dystrophy.

Collectively, these fatalities have reignited debate over the transparency and regulatory oversight of China’s IIT pathway, which allows academic and commercial researchers to run trials with less formal national drug-regulator supervision.