Circio, AaviGen Launch Heart Gene Therapy Collaboration

Circio Holding ASA, a biotechnology company developing novel circular RNA expression technology for gene and cell therapy, and AaviGen Gmbh, a biotechnology company focused on precision gene therapies for human cardiac diseases, have jointly entered a research collaboration to develop circVec-enhanced AAV vectors specifically engineered for targeted, low dose heart gene therapies.

By integrating these complementary technologies, the collaboration seeks to pioneer the next generation of AAV gene therapy candidates. The focus is on addressing genetic and chronic heart conditions through therapies that can be administered at low therapeutic doses, ensuring high and confined expression in heart while minimizing off-target effects.

“The future clinical success of cardiac gene therapies will be determined by precision elements that ensure both efficacy and safety,” said Dr. Martin Busch, COO of AaviGen. “AaviGen’s human heart-derived next-generation cardiac-targeted vector technology enables precision delivery of therapeutic genes and lays the groundwork for the targeted integration of components tailored to specific indications, with Circio’s innovative expression-enhancing platform being a key element. The combination of AaviGen’s targeted capsids with the circVec platform could therefore open the door to a new level of efficiency and safety — an opportunity we are very excited to explore together with the Circio team.”

Dr. Victor Levitsky, CSO of Circio, added, “The combination of our technologies has the potential to maximize on-target gene expression and minimize off-target delivery, resulting in substantial dose reduction and an improved toxicity profile. This can make AAV-based gene therapy accessible for patients with severe inherited heart diseases as well as address large and growing medical need in chronic heart failure.”

The collaboration will involve three stages:

1. Production of novel AAVs combining the proprietary heart-targeted capsids from AaviGen with the circVec expression cassette from Circio
2. Testing and characterization of the novel AAVs in vitro
3. Confirmation and validation of AAV performance in vivo with focus on AAV biodistribution and gene expression profiles between different design combinations