Abstract
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that
includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT)
domain.
This is a multicenter, open-label dose evaluation clinical study to assess the safety,
tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in
participants with Duchenne.
