Biotechnology company Circio Holding has released preclinical findings as ASGCT in Boston, showing its circVec circular RNA expression platform delivered substantially enhanced gene expression in AAV-based therapies.
Circio has announced new preclinical data highlighting the potential of its circular RNA technology to improve the effectiveness and safety of gene therapies, following presentations at the annual meeting of the American Society of Gene and Cell Therapy in Boston.
The biotechnology company presented both a keynote session and a scientific poster detailing recent progress with its circVec circular RNA expression platform.
Focus on safer and more effective gene therapies
During the keynote presentation, Chief Executive Officer, Dr Erik Wiklund, explained how the circVec platform is designed to enhance and prolong gene expression by harnessing the stability of circular RNA.
Circio reported that, in preclinical studies involving adeno-associated virus (AAV) gene therapy, circVec generated up to 40-fold greater gene expression in heart tissue and up to 50-fold greater expression in the eye compared with conventional approaches.
The company believes the findings could help address one of the key limitations facing AAV gene therapy: the high doses currently required to achieve therapeutic effects. These elevated doses have been linked to safety concerns and toxicities, which continue to restrict broader clinical use.
Circio said the data suggests circVec may enable substantial reductions in AAV dosing requirements, potentially improving patient safety while widening treatment opportunities for people living with rare genetic disorders.
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Heart tissue data strengthens platform potential
The poster presentation focused specifically on in vivo heart tissue data generated using the circVec-AAV platform.
According to the company, circVec delivered a 40-fold increase in gene expression from AAV9 vectors in heart tissue compared with standard AAV technologies. Molecular analyses of the tissue also indicated that the improved performance was driven by the durability of circular RNA molecules.
Researchers also observed consistent results across a wide dose range, with enhanced expression and distribution throughout heart tissue seen at every tested dose level.
The findings highlight the platform’s potential application in treating inherited cardiac diseases, an area where durable and targeted gene expression remains a major challenge.
Industry visibility grows
Commenting on the presentations, Dr Wiklund said the conference provided an important opportunity for Circio to showcase its technology to the wider global gene therapy sector.
“ASGCT is the most important event of the year in the gene therapy field and Circio is very honoured to be selected for an oral presentation in this forum of global expertise,” he said. “The ASGCT meeting is also an excellent opportunity to connect with existing and prospective collaboration partners. The robust circVec data package, combined with high demand for novel circular RNA approaches, continues to trigger substantial visibility for and interest in Circio and our unique technology platform.”
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