Circio's circular RNA platform demonstrates enhanced protein expression with unique tissue distribution patterns that could expand gene therapy applications, according to data presented at the American Society of Gene and Cell Therapy (ASGCT) annual meeting in New Orleans.
The Norwegian biotechnology company Circio Holding ASA (OSE: CRNA) showcased strengthened in vivo data for its circVec platform, a circular RNA expression system designed to enhance both viral and non-viral gene and cell therapies. The results were presented on May 13, 2025, at the ASGCT meeting taking place from May 13-17 in New Orleans.
Distinct Tissue Expression Profile Reveals New Therapeutic Potential
A key finding from the presentation highlights that circVec vectors display tissue expression patterns fundamentally different from equivalent mRNA-based vectors. The circVec platform demonstrated increased protein expression in muscle, heart, and spleen tissues, while maintaining consistently low expression in the liver.
"Circio is rapidly expanding the in vivo circVec data package, with several recent intriguing advances. It has become evident that circVec not only offers increased protein expression level and durability in general, but also that this effect is associated with a distinct tissue profile," said Dr. Thomas B. Hansen, CTO of Circio.
This unique expression pattern suggests the technology could address therapeutic areas where conventional gene therapy approaches have limitations, particularly for conditions requiring targeted expression in specific tissues while avoiding liver accumulation.
Enhanced Expression and Durability Across Multiple Formats
The circVec platform has demonstrated up to 15-fold enhanced protein expression compared to classic mRNA vector systems, with significantly improved durability. These advantages were observed in both viral and non-viral delivery formats, suggesting broad applicability across different gene therapy modalities.
Longitudinal in vivo data extending to six months showed a general advantage of circVec expression at low dose levels. This could potentially allow for lower therapeutic doses, potentially reducing manufacturing challenges and side effects associated with high-dose gene therapy administration.
Advancing Development for Muscular Dystrophies and Cardiomyopathies
Circio is progressing its circVec-AAV gene therapy development specifically for muscular dystrophies and cardiomyopathies. The enhanced target tissue expression and reduced liver accumulation observed with circVec-AAV vectors are particularly relevant for these indications, where efficient muscle and cardiac expression with minimal off-target effects are critical.
The company is currently conducting follow-up analyses and testing novel circVec-AAV variants to further explore these observations and identify the most favorable opportunities for circVec-AAV gene therapy development.
Technology Platform Background
Circio's proprietary circVec technology is based on a modular genetic cassette design for efficient biogenesis of multifunctional circular RNA inside cells. The platform can be deployed in multiple therapeutic settings, including genetic medicine, cell therapy, and treatments for chronic diseases.
Circular RNAs differ from linear mRNAs in their structure and stability. By forming a continuous loop without free ends, circular RNAs are inherently more resistant to exonuclease degradation, potentially contributing to their enhanced expression durability.
Strategic Implications
Dr. Hansen emphasized the strategic value of these findings: "These results provide a valuable roadmap to direct our R&D activities and develop a therapeutic strategy focused on areas that are not well served by existing gene therapy approaches."
The company selected the prestigious ASGCT meeting to present these results as it provided an opportunity to showcase their technology to potential partners and the broader life science community.
In addition to its circRNA platform, Circio is also developing TG01, a pan-RAS cancer vaccine targeting KRAS driver mutations, currently being tested in clinical trials for RAS-mutated pancreatic and lung cancer in the USA and multiple myeloma in Norway.
The poster presentation titled "CircVec: a powerful circular RNA expression platform to enhance viral and non-viral gene and cell therapies" was presented on May 13, 2025, at the Ernest N. Morial Convention Center in New Orleans.
