Key Takeaways

  • Circio’s circVec-AAV 4.0 achieved up to 50-fold higher gene expression in the eye versus conventional mRNA-based AAVs, marking ophthalmology as a promising new development area

  • The platform maintained a 10-fold expression advantage even at a 90% reduced dose

  • Confirmatory heart data reproduced a 40-fold expression increase

Circio Holding announced new and confirmatory in vivo data for its circVec-AAV expression platform, demonstrating up to a 50-fold enhancement in gene expression in the eye compared to conventional mRNA-based AAV vectors. According to Circlo, the findings mark the first time such a significant improvement has been observed in ocular delivery and position ophthalmology as a promising new therapeutic area for circVec-based gene therapies.

Local administration of AAV-circVec 4.0 to the eye achieved the strongest performance improvement versus conventional mRNA-based AAV vectors reported to date, with in vivo data showing up to 50-fold higher gene expression. Notably, AAV-circVec demonstrated a 10-fold expression advantage even when delivered at a 90% reduced dose compared to a standard AAV construct.

The data suggest that circVec may enable lower dosing strategies while maintaining or improving therapeutic potency—an important factor in addressing safety, tolerability, and cost concerns associated with AAV gene therapy.

Growing industry interest in ophthalmology, underscored by recent strategic transactions involving major pharmaceutical companies and specialist investors, contributed to Circio’s decision to expand development into eye diseases. The company believes its circular RNA-based gene expression system has the potential to significantly enhance potency, reduce toxicity, and lower manufacturing costs for AAV-based treatments targeting genetic and degenerative eye disorders with high unmet medical need.

In parallel, Circio has continued to expand its data package for AAV-circVec 3.2 and 4.0 in cardiac applications. Newly completed experiments reproduced previously reported results showing a 40-fold increase in gene expression in heart tissue.

Earlier this quarter, Circio completed a financing raising NOK 68.6 million (approximately USD $7 million) through a 50% oversubscribed rights issue and parallel private placements. The capital raise provides funding to accelerate circVec research and development activities over the next 12 months.