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AAVB-039
- Development stage
- Phase 2
- Lead developer
- AAVantgarde Bio
- Modality
- Gene Therapies
- Administration
- Intra-retinal
01
Overview
**AAVB-039** is an investigational, subretinally administered dual adeno-associated virus serotype 8 gene-augmentation therapy developed by AAVantgarde for Stargardt disease caused by biallelic mutations in **ABCA4**. It uses an intein-mediated dual-vector platform to enable delivery and reconstitution of the full-length ABCA4 protein, addressing the underlying genetic deficiency in this inherited retinal disease. AAVB-039 is being evaluated in the Phase 1/2 CELESTE study. ([aavantgarde.com](https://www.aavantgarde.com/en/news/aavantgarde-receives-fda-ind-clearance-to-progress-stargardt-disease-program-aavb-039/))
- Other names
- dual AAV8.ABCA4
02
Targets
ABCA4
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