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Drug intelligence / Profile preview
AAV9-ABE9
- Development stage
- Preclinical
- Lead developer
- Mayo Clinic
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-ABE9 is a preclinical gene therapy and base editing system developed by the Mayo Clinic for the treatment of autosomal dominant polycystic kidney disease (ADPKD). It consists of an adenine base editor (ABE9) delivered via dual adeno-associated virus serotype 9 (AAV9) vectors. The system is designed to correct a pathogenic single-nucleotide variant (such as the R3277C mutation) in the PKD1 gene, which encodes polycystin-1. Two versions have been developed: a broadly expressed system utilizing the chicken beta-actin hybrid (Cbh) promoter and a kidney-specific system utilizing the kidney-specific cadherin (Ksp) promoter. In preclinical mouse models, a single dose of AAV9-ABE9 effectively corrected the Pkd1 mutation, delayed renal cyst growth, reduced cardiac hypertrophy, and improved survival.
- Other names
- ABE9-AAV9ABE-9-AAV9ABE 9-AAV9Cbh-ABE9-dual-AAV9Cbh-ABE-9-dual-AAV9Cbh-ABE 9-dual-AAV9Ksp-ABE9-dual-AAV9Ksp-ABE-9-dual-AAV9Ksp-ABE 9-dual-AAV9PKD1 targeted-dual-ABE9-AAV9PKD-1 targeted-dual-ABE9-AAV9PKD 1 targeted-dual-ABE9-AAV9dual-AAV9-ABE9dual-AAV-9-ABE9dual-AAV 9-ABE9
02
Targets
ENPP1
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