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Rebisufligene Etisparvovec · Fayuvi, UX-111

Gene therapy (AAV vector) · Gene replacement · developed by Ultragenyx Pharmaceutical

Approved FDA, Sep 2026

2 Registered trials

What is Rebisufligene Etisparvovec?

Rebisufligene etisparvovec is a self-complementary adeno-associated virus serotype 9 (scAAV9) carrying the human SGSH gene under control of a U1a promoter. It is administered as a single intravenous dose to deliver functional SGSH gene to patients with mucopolysaccharidosis IIIA (Sanfilippo syndrome type A), a lysosomal storage disorder.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Fayuvi, UX-111, ABO-102, scAAV9.U1a.hSGSH
Drug type: Gene therapy (AAV vector)
Mechanism: Gene replacement
Therapy area: Metabolic
Developer: Ultragenyx Pharmaceutical Inc.
In trials of: ABEO
FDA status: Approved as Fayuvi (September 17, 2026), BLA125845
UNII: V28L3Q0LVZ

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT02716246	Phase 2/3	MPS IIIA	Recruiting	ABEO
NCT04088734	Phase 1/2	MPS IIIA	Terminated	ABEO

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

MPS IIIAMucopolysaccharidosis IIISanfilippo ASanfilippo Syndrome

Sources

Updated Oct 3, 2026.

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z