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ABX-102
- Development stage
- Unknown
- Lead developer
- Ultragenyx Pharmaceutical
- Modality
- Gene Therapies
- Administration
- Intravenous Infusion
01
Overview
ABX-102, also known as ABO-102 and formerly UX111 (rebisufligene etisparvovec), is an investigational gene therapy developed by Abeona Therapeutics for Sanfilippo Syndrome type A (MPS IIIA). This therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the SGSH gene. The SGSH gene encodes the sulfamidase enzyme, which is deficient in MPS IIIA, leading to the accumulation of heparan sulfate and subsequent severe neurodevelopmental and physical decline. By introducing a functional SGSH gene, ABX-102 aims to restore the missing enzyme activity, thereby addressing the underlying genetic cause of the disease. It is administered as a one-time intravenous infusion and is currently in Phase 1/2 clinical development.
- Other names
- rebisufligene etisparvovec
02
Targets
SGSH (N-sulphoglucosamine sulphohydrolase)
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