An investigational gene therapy for Duchenne muscular dystrophy has demonstrated a favourable safety profile and encouraging biomarker results as part of the Phase I/II AFFINITY DUCHENNE clinical trial.
Previously, five boys aged six to 12 years were treated with RGX-202, a single-dose gene therapy that aims to provide patients with a functional version of the microdystrophin gene (see BioNews 1293).
Now, as of the latest interim analysis, 13 participants have received treatment. RGX-202 was generally well tolerated, with no serious adverse events or adverse events of special interest observed. There was also no evidence of liver injury, a potential safety concern associated with high-dose adeno-associated virus gene therapies.
Among participants treated at the pivotal dose level, motor function results at 12 months were reported to exceed predicted disease trajectories.
The trial is ongoing and will continue to evaluate the therapy's long-term safety and clinical efficacy. Additional cohorts are currently enrolling as part of the programme.
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