PR Newswire BiotechOriginal article
Ractigen Therapeutics Presents Positive First-in-Human Data for RAG-18 at WMS 2026, Establishing Clinical Proof-of-Mechanism for RNA Activation in Duchenne Muscular Dystrophy
Data ReadoutpositiveRAG-18Positive
AI Analysis
Summary
Ractigen Therapeutics presented positive first-in-human clinical data for RAG-18, demonstrating that a systemically delivered small activating RNA can safely upregulate endogenous utrophin in human skeletal muscle for Duchenne Muscular Dystrophy, with 3.5- to 5.3-fold increases in sarcolemmal utrophin expression observed in paired muscle biopsies.
Clinical Trial Data
Phase
Phase 1
Primary Endpoint
Met
Outcome Details
First-in-human data demonstrating systemically delivered small activating RNA safely upregulates endogenous target protein in human skeletal muscle; paired muscle biopsies reveal 3.5- to 5.3-fold increases in sarcolemmal utrophin expression
Importance:7/10
Sentiment:
0.85
RNA ActivationDuchenne Muscular DystrophyFirst-in-HumansaRNAUtrophinClinical Proof-of-Mechanism
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Published by PR Newswire Biotech on October 3, 2026 11:30 AM