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PGN-EDODM1 was generally well-tolerated, with all adverse events being mild or moderate, and no serious adverse events reported. The mean splicing correction observed with PGN-EDODM1 (n=6) was 7.3%, compared to 6.8% for the placebo (n=2). Excluding one outlier patient, the treatment group showed a mean splicing correction of 22.9% (n=5). Promising trends were seen in vHOT in the PGN-EDODM1 treated group. The company is on track to report clinical data from the 10 mg/kg multiple dose cohort in the second half of 2026, with sufficient cash expected to fund operations into the second half of 2027.
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