Potential one-time gene therapy for the treatment of Duchenne includes a novel, optimized microdystrophin transgene and REGENXBIO's proprietary NAV® AAV8 vector
Innovative trial design, including comprehensive immunosuppressive regimen, to evaluate safety and optimal dose
cGMP process material made at commercial-scale to be used throughout clinical development of RGX-202
REGENXBIO expects to initiate trial in the first half of 2022
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