Competitive Positioning And Timeline RiskBeing behind competitors in development and unclear access to accelerated regulatory pathways increases the risk that rivals capture market share or regulatory advantage before clinical differentiation is established.
Platform Readthrough RiskReadthrough concerns from the discontinued DMD program raise questions about the proprietary EDO platform's long-term safety, which could increase due-diligence scrutiny and investor caution.
Residual Safety Signals - Electrolyte And Renal ConcernsReports of asymptomatic hypomagnesemia and prior renal signals indicate further safety de-risking may be required, potentially prompting additional monitoring or trial adjustments and adding regulatory complexity.