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Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-old
Clinical Trials Arena March 20, 2025
The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain muscle integrity compared with control.
Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study (NCT05693142), which is examining its investigational gene therapy RGX-202.
The therapy is designed to treat Duchenne muscular dystrophy (DMD) and was able to induce a 122.3% microdystrophin expression in one child aged three.
RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients.
DMD is a form of muscular dystrophy typically associated with the inability to produce...
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