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ABO-101

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

N/A Most advanced trial (none active)

ABEO Developer

1 Registered trials

What is ABO-101?

ABO-101 is a recombinant adeno-associated virus 9 (rAAV9) gene therapy that delivers the human NAGLU gene to replace the deficient alpha-N-acetylglucosaminidase enzyme. It is being studied for mucopolysaccharidosis III-B, a lysosomal storage disorder.

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: N/A

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04655911	N/A	Mucopolysaccharidosis III-B	Terminated	ABEO

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

Conditions studied

Mucopolysaccharidosis III-B

More from Abeona Therapeutics

EB-101 Phase 3 LZRSE-Col7A1 Phase 1/2 MSI-78 Phase 3 Pz-cel Phase 4 rAAV9.CMV.hNAGLU Phase 1/2

Sources

ClinicalTrials.gov NCT04655911

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

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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