rebisufligene etisparvovec

Rebisufligene etisparvovec is a one-time, intravenous, self-complementary adeno-associated virus serotype 9 gene-replacement therapy for neurologic manifestations of mucopolysaccharidosis type IIIA, also called Sanfilippo syndrome type A, in pediatric patients with preserved neurodevelopmental function. The nonreplicating AAV9 vector delivers a functional human **SGSH** transgene, enabling expression of lysosomal sulfamidase to restore degradation of accumulated heparan sulfate. It was initially developed as ABO-102 by Nationwide Children's Hospital and Abeona Therapeutics, licensed to Ultragenyx in 2022, and approved by the FDA in September 2026 as FAYUVI. ([fda.gov](https://www.fda.gov/media/194920/download?attachment=))

Brand names

FAYUVI

Other names

rebisufligene etisparvovec-hopfscAAV9.U1a.hSGSHscAAV-9.U1a.hSGSHscAAV 9.U1a.hSGSH