On May 14, 2025 Circio Holding ASA (OSE: CRNA), a biotechnology company developing powerful circular RNA technology for next generation nucleic acid medicine, today announces the presentation of new and strengthened in vivo data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting 2025 in New Orleans, USA (Press release, Circio, MAY 14, 2025, View Source [SID1234652961]).
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In the poster presentation, Circio is showcasing its latest in vitro and in vivo results for the circVec platform. circVec continues to demonstrate broad capability to significantly boost protein expression level and durability for both viral and non-viral gene and cell therapy.
Importantly, new results show that both viral and non-viral circVec vectors display tissue patterns that are distinct from equivalent mRNA-vectors. The pattern is hallmarked by increased protein expression in muscle, heart and spleen, and consistently low in liver. These surprising observations demonstrate a fundamental biological difference between circRNA and mRNA-based expression and show that the circVec platform has advantages that can open new therapeutic opportunities where current gene therapy approaches fall short.
"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. 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." said Dr. Thomas B. Hansen, CTO of Circio. "We selected the prestigious ASGCT (Free ASGCT Whitepaper) meeting to present our latest results as it provided a great opportunity to showcase our technology to a broad life science audience and potential partners."
In addition, Circio is continuing to progress its circVec-AAV gene therapy development for muscular dystrophies and cardiomyopathies. Longitudinal in vivo data up to six months show a general overall advantage of circVec expression at low dose levels. Enhanced target tissue expression and reduced liver accumulation is also observable with circVec-AAV vectors, indicating that the circVec advantage is more pronounced in specific tissues and therapeutic settings. Follow-up analyses and testing of novel circVec-AAV variants are currently ongoing to further explore these observations and identify the most favorable opportunities for circVec-AAV gene therapy development.
Title of presentation:
CircVec: a powerful circular RNA expression platform to enhance viral and non-viral gene and cell therapies
Time and poster number:
13 May 2025, 18:00-19:30hrs CDT, poster #655
Location:
Ernest N. Morial Convention Center, New Orleans, Louisiana, USA
The poster is attached to this press release and is available on Circio´s webpage