Clinical-stage biotechnology company
Appears in 1 story · Last updated Oct 21, 2026
Developer of RAG-18; presented first-in-human data at WMS 2026
Three boys with Duchenne muscular dystrophy received monthly infusions of an experimental drug. Four months later, muscle biopsies showed 3.5- to 5.3-fold increases in utrophin, a protein that can stand in for the missing dystrophin.
Updated Oct 21
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