Clinical TrialsThe registrational Phase 1/2 STAAR trial in Fabry disease has delivered positive top-line results across renal, cardiac, and quality-of-life endpoints.
PartnershipsThe proprietary STAC-BBB capsid has now attracted three blue-chip pharma partnerships, generating $88 million in upfront payments with more than $4.6 billion in potential milestones.
Regulatory PathwaySGMO announced alignment with FDA on a regulatory pathway to accelerated filing for their AAV-based GTx '920 for Fabry disease.