In January 2021, REGENXBIO announced the development of a potential single-use gene therapy for the treatment of Duchenne disease. The RGX-202 is designed to deliver a new microdistrophin transgen that includes a broad coding region of the C-Terminal domain found in natural dystrophin.
REGENXBIO expects to submit a new drug research program (IND) to the FDA by mid-2021.