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Results from the first human trial of an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD), in which a young Chinese boy died, have been published 1 . Although the trial provides evidence that the treatment developed by HuidaGene, based in Shanghai, China, edited some target genes, the therapeutic benefit was limited and led to a catastrophic immune reaction in one participant. Researchers say there is an urgent need to establish safe dosages for these kinds of experimental treatment.

The trial gained attention when HuidaGene announced on 5 August that one participant, a boy, had died roughly a year earlier. Researchers criticized the company for waiting so long to publicly report the death — and the event put a spotlight on China ’ s fast-track clinical trials . On 27 August, HuidaGene told Nature that the firm wanted to “establish the clinical facts and undertake a comprehensive investigation” before communicating with the broader public.…