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ABO-101
- Development stage
- Phase 2
- Lead developer
- Arbor Biotechnologies
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
ABO-101 is an adeno-associated virus (AAV)-based gene therapy developed by Abeona Therapeutics for the treatment of Sanfilippo syndrome type B (MPS IIIB). It utilizes the NAV AAV9 vector platform to deliver a functional gene, aiming to correct the underlying genetic defect responsible for the disease. The drug is currently in Phase 1/2 clinical development.
- Brand names
- ABO-101ABO101ABO 101
- Other names
- rAAV9-CMV-hNAGLUrAAV-9-CMV-hNAGLUrAAV 9-CMV-hNAGLUAAV9-NAGLU gene therapyAAV-9-NAGLU gene therapyAAV 9-NAGLU gene therapy
02
Targets
HAO1 (Glycolate Oxidase)
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