GoKawiilHuidaGene's gene-editing DMD therapy showed limited benefit, Cell study confirms
A Cell publication details results from HuidaGene's first-in-human trial of a CRISPR base-editing gene therapy for Duchenne muscular dystrophy, confirming that one boy died after receiving a high dose. The study found the treatment edited some target genes in the four participants but produced only limited therapeutic effect, while the boy who died suffered a severe immune reaction beginning with abdominal pain and fever within 24 hours of dosing.