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rAAV9.CMV.hNAGLU

Gene therapy (AAV vector) · Gene replacement · developed by Abeona Therapeutics

Phase 1/2 Most advanced trial (none active)

ABEO Developer

1 Registered trials

What is rAAV9.CMV.hNAGLU?

rAAV9.CMV.hNAGLU is an adeno-associated virus serotype 9 vector carrying the human NAGLU gene, delivered intravenously as a one-time infusion. It is studied in mucopolysaccharidosis type 3B, a lysosomal storage disorder caused by NAGLU deficiency.

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

At a glance

Drug type: Gene therapy (AAV vector)
Mechanism: Gene replacement
Therapy area: Metabolic
Developer: Abeona Therapeutics, Inc (ABEO)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1/2

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT03315182	Phase 1/2	Mucopolysaccharidosis Type 3 B	Terminated	ABEO

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

Conditions studied

Mucopolysaccharidosis Type 3 B

More from Abeona Therapeutics

ABO-101 N/A EB-101 Phase 3 LZRSE-Col7A1 Phase 1/2 MSI-78 Phase 3 Pz-cel Phase 4

Sources

ClinicalTrials.gov NCT03315182

Updated Oct 3, 2026. Something wrong or missing? Report it on the ABEO page.

Follow ABEO, the company behind rAAV9.CMV.hNAGLU

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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