Durable Enzyme ExpressionUpdated clinical results show dose-dependent, durable increases in the therapeutic enzyme for Fabry disease, supporting a sustained biological effect that strengthens confidence in the program.
Pipeline Diversification And Financial FlexibilityProgress across central nervous system and metabolic programs, together with a strong balance sheet, preserves strategic optionality to pursue multiple regulatory or development strategies.
Therapy-sparing Clinical BenefitSeveral treated patients were able to discontinue enzyme replacement therapy while maintaining stable disease biomarkers, indicating meaningful clinical benefit and a potential reduction in long-term treatment burden.