Intellectual Property And Commercial DefensibilityU.S. patent protection for PGN-EDODM1 secures long-term exclusivity, strengthening commercial defensibility and improving prospects for partnerships or strategic transactions.
Pharmacodynamics / EfficacyApproximately 53% splicing correction in single-dose data indicates a strong molecular effect that could translate into meaningful improvements in mobility, pulmonary function, and gastrointestinal outcomes.
Safety And Dose MarginTolerability observed at the 15 mg/kg single-dose level, coupled with absence of hypomagnesemia and only transient renal signals, provides a safety margin for the planned 5 mg/kg multi-dose regimen and lowers clinical development risk.