STAT+: Once again, child dies in gene-editing trial in China, rekindling debate on transparency and safety

In a recent development, a child has succumbed to illness following a gene-editing trial in China, reigniting discussions surrounding transparency and safety in such experiments. Last year, a nascent Chinese biotech firm, HuidaGene, attracted significant attention at the American Society for Gene and Cell Therapy annual convention in New Orleans.

CEO Alvin Luk unveiled data from the first-ever clinical trial to evaluate the potential of CRISPR gene editing for treating Duchenne muscular dystrophy, a debilitating and lethal condition in children. Despite technical hurdles encountered in similar endeavors within the United States, HuidaGene had managed to surpass expectations, leveraging a regulatory pathway that permitted Chinese hospitals to commence studies without the scrutiny of government regulators.

The outcomes, derived from the initial two patients, were underwhelming, leaving uncertainty as to whether the therapy was efficacious. However, Luk asserted that the data suggested a positive effect, and the company was poised to administer a higher dosage to potentially achieve more favorable results. Subsequently, HuidaGene fell silent.

Over a span of 15 months subsequent to the conference, the Shanghai-based enterprise refrained from releasing any new press statements. Alvin Luk, along with Chief Technology Officer TJ Cradick, a seasoned gene editing professional who had only recently joined the company, departed quietly in the summer of the previous year.

In February, a reference on a clinical trial registry indicated that the study had been completed. However, the fate of the remaining participants in the study remained ambiguous. After an extensive investigation spanning several months and numerous inquiries to the company, HuidaGene finally released an update on Wednesday.