Class Safety ConcernsSafety issues with a competing gene therapy and reported deaths in non-ambulatory patients increase scrutiny of gene therapy immunosuppression strategies and could heighten regulatory and payer caution.
Information And Disclosure RiskRegulatory disclaimers, limits on redistribution, and reliance on sources presented without guarantees create information uncertainty that may constrain investor visibility and market interest.
Patient Response VariabilityVariable expression levels across patients and age-related differences in response raise the risk of inconsistent treatment outcomes in larger trials, complicating efficacy readouts and regulatory assessment.