Clinical EfficacySustained functional improvements in Duchenne patients treated with the lead gene therapy support the potential for an accelerated regulatory filing and bolster the case for future approval.
Commercial OpportunityPotential first-in-class approval for the Hunter syndrome candidate, combined with company-led manufacturing and a possible Priority Review Voucher, could provide meaningful commercial upside and strengthen financial prospects.
Manufacturing CapabilityIn-house, state-of-the-art manufacturing with high-purity production and full fill-finish capability enables faster development timelines, lower dosing strategies, and capacity to supply commercial demand with attractive cost of goods.