PepGen reported early Phase 2 data for PGN-EDODM1 in myotonic dystrophy type 1, showing an average 46.5% correction of mis-splicing errors after 13 weeks in six treated patients, with four patients exceeding 50%. The company characterized results as among the highest splicing-correction levels seen in multiple-dose DM1 studies, with mild-to-moderate treatment-emergent side effects. The dataset also included functional signals tied to mobility and muscle strength, including a hand-muscle relaxation assessment. PepGen said there was no evidence of cumulative kidney damage during the 16-week observation period, while nausea was the most common adverse event and urine protein rose temporarily after the first dose. For biotech stakeholders, the readout matters because DM1 therapies have historically struggled to demonstrate clinically meaningful splicing and durability in multi-dose regimens. The update strengthens the RNA splicing-correction thesis and keeps attention on dose-response and safety monitoring for systemically delivered oligonucleotides.
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