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Zafinofusp alfa

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Brain-penetrant SGSH enzyme replacement therapy in Phase 1/2 follow-up for Sanfilippo syndrome type A.

Modality
intravenous blood-brain-barrier-crossing SGSH enzyme replacement protein
Availability (US)
Phase 1/2 study Active, not recruiting; Phase 3 and marketing application remain planned
Biomarkers
cerebrospinal-fluid heparan sulfate; urine heparan sulfate; serum and CSF pharmacokinetics; anti-drug antibodies; treatment-emergent adverse events
Hallmark targeted
lysosomal dysfunction, substrate accumulation, neurodegeneration
Kind
investigational enzyme replacement therapy
Clinical trials
NCT06181136 — Phase 1/Phase 2, Active, not recruiting on July 13, 2026 registry update