Regenxbio Inc. has announced the publication of preclinical results demonstrating the functional benefits of their investigational gene therapy, RGX-202, for Duchenne Muscular Dystrophy. The study compared a microdystrophin construct that includes the C-terminal (CT) domain to one without it. Findings indicate that the inclusion of the CT domain leads to higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage. These results support the positive functional data observed in the Phase I/II AFFINITY DUCHENNE trial of RGX-202, which is the only gene therapy candidate for Duchenne that includes the CT domain. Interim results from this trial show promising changes in disease trajectory and a favorable safety profile. Regenxbio is currently enrolling participants in the pivotal portion of the AFFINITY DUCHENNE trial and plans to submit a Biologics License Application in mid-2026.
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