RGX-202 Gene Therapy in Duchenne Muscular Dystrophy

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Primary Completion
2/1/26
Study Completion
8/1/28
Sponsor
REGENXBIO Inc.
Ticker
$RGNX
Type
Phase 1/Phase 2/Phase 3
Trial Status
Recruiting
Trial Size
65
NCT
NCT05693142
Trial Description
Multicenter, open-label sequential Phase 1/2/3 study of one-time IV RGX-202 gene therapy in male participants with Duchenne muscular dystrophy. The trial is evaluating safety and tolerability in Part 1 and pharmacodynamic microdystrophin expression in Parts 2 and 3, with functional assessments followed over time.