Drug intelligence / Profile preview

AAV9-circVec

Development stage
Preclinical
Lead developer
Targovax
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

AAV9-circVec is a next-generation cardiac gene therapy platform developed by Circio AB that utilizes the proprietary circVec technology to deliver protein-coding circular RNA (circRNA) via adeno-associated virus serotype 9 (AAV9) vectors. Unlike conventional linear mRNA, circRNAs are resistant to exonucleolytic degradation, which significantly enhances their stability and leads to higher protein accumulation over time. The platform incorporates internal ribosome entry site (IRES) elements to drive translation and employs tissue-specific promoters, such as the cardiomyocyte-specific cTnT promoter, to maximize on-target cardiac expression while minimizing off-target effects in the liver. Preclinical data indicates that AAV9-circVec can achieve up to a 40-fold increase in transgene expression compared to standard AAV-mRNA vectors and reduces cellular stress by bypassing cap-dependent translation and minimizing the unfolded protein response (UPR).

Other names
circVec
02

Targets

IgG (Immunoglobulin G)Terminal beta-galactose residues on cell-surface glycoconjugates80S ribosome (Ribosome and ribosomal components)

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