Competitive Pressure On Established FranchisePotential competition from rival exon-skipping candidates and prior confirmatory trial shortcomings could accelerate erosion of the established franchise and pressure future revenue.
Leadership Succession RiskPlanned CEO retirement creates execution risk unless a seamless internal successor is chosen to maintain strategic, financial, and patient-focused continuity.
Serious Safety ConcernsReported serious adverse events, including a fatal arrhythmia in a trial patient, raise material safety concerns that could slow development or complicate regulatory review.