HuidaGene reported evidence of editing at target genes and dystrophin expression in muscle. These molecular observations do not establish a lasting clinical benefit.
Nature highlighted limited efficacy and difficulties in selecting doses. The company acknowledged limits in preclinical models' ability to predict severe immune toxicity after systemic delivery of the viral vector.
The publication adds details about a death announced in August; it does not describe a new death on 27 October. Three participants remain in long-term follow-up, HuidaGene said.