REGENXBIO Inc. announced on July 10, 2025, the publication of preclinical results in Molecular Therapy Methods and Clinical Development. The study compared a microdystrophin gene therapy construct with the C-terminal (CT) domain to one without it.
The results demonstrated that the microdystrophin with the CT domain, which is unique to RGX-202, improved functional benefit in mdx mice, a preclinical model of Duchenne Muscular Dystrophy. This construct led to higher microdystrophin levels, more effective recruitment of the dystrophin-associated protein complex, and increased muscle force and resistance to damage.
These findings validate the differentiated design of RGX-202, suggesting that the inclusion of the CT domain enhances microdystrophin accumulation and may improve functional benefits for patients. This preclinical data reinforces the positive interim clinical results observed in the Phase I/II AFFINITY DUCHENNE trial.
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