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AVGN7
- Development stage
- Preclinical
- Lead developer
- Aavogen
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intramuscular
01
Overview
AVGN7 is an adeno-associated virus (AAV)-based gene therapy developed by Aavogen for the treatment of muscle-wasting and fibrotic conditions, including inclusion body myositis (IBM), heart failure (HFrEF and HFpEF), and sarcopenic obesity. The therapy delivers the Smad7 gene directly to muscle tissue. Smad7 acts as an intracellular inhibitor of the Smad2/3 signaling pathway, which is activated by myostatin (MSTN) and other ligands of the activin receptor type IIB (ActRIIb). By blocking this pathway, AVGN7 prevents muscle protein degradation and fibrosis. Simultaneously, Smad7 expression activates the PI3K-Akt-mTOR and PGC1α pathways, promoting muscle protein synthesis and mitochondrial biogenesis to restore muscle mass and strength.
- Other names
- AVGN7AVGN-7AVGN 7
02
Targets
Mothers against decapentaplegic homolog 2/3 signaling complex (SMAD2/3 complex)GDF8 (Myostatin)ACVR2B (Activin receptor type-2B)SMAD7 (Mothers against decapentaplegic homolog 7)
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