Child dies in Chinese CRISPR trial for Duchenne muscular dystrophy, raising safety alarms

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Last year, a little-known Chinese startup took center stage at the American Society for Gene and Cell Therapy annual convention in New Orleans.

Speaking before a sprawling conference hall for the presidential symposium, HuidaGene CEO Alvin Luk presented data from one of the world’s first trials to test if CRISPR gene editing could cure children with Duchenne muscular dystrophy, an intractable, fatal disease. Similar efforts in the U.S. ran aground amid technical challenges, but HuidaGene had surged ahead, using a regulatory pathway that lets Chinese hospitals start studies without oversight from government regulators.

The results, from the first two patients, were not impressive. It wasn’t clear the therapy worked at all. But Luk said the data indicated benefit and the company was about to test a higher dose. Hopefully, that would have far better effects.

Then HuidaGene went dark. For 15 months following that conference, the Shanghai-based company did not issue a single new press release. Luk quietly departed last summer, alongside Chief Technology Officer TJ Cradick, a longtime U.S.-based gene editing executive who had been there less than a year. In February, a listing on a clinical trial registry was updated to announce the study was “complete.”

What happened to the remainder of patients in the study was unclear. But after a monthslong STAT investigation and repeated questions to the company, HuidaGene on Wednesday issued an update.

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