circRNA expression systems for enhanced gene and cell therapies
Circio has established a unique and proprietary intracellular circular RNA (circRNA) expression system, circVec, that substantially enhances the potency and durability of gene expression which is critical for gene therapies. circVec is designed for efficient biogenesis of circRNA from DNA and viral vectors, with primary application in AAV gene therapy and in vivo CAR cell therapy. circVec has demonstrated up to 50-fold enhanced gene expression compared to conventional mRNA-based AAV gene therapy and achieves up to six months expression durability for LNP-delivered in vivo CAR.
Circio is the global leader in vector-based circRNA expression systems with no known competitors with comparable versatility and performance. The circVec R&D activities are being conducted in the wholly owned subsidiary Circio AB located in the Karolinska Institute Campus in Huddinge in Stockholm, Sweden.
Business and strategy
Circio’s mission is to build the world-leading circular RNA-based gene expression platform technology. The group is well underway to achieve this mission through its versatile and powerful circVec platform. To date, circVec has demonstrated ability to enhance conventional AAV gene therapy in vivo by up to 50-fold and prolong the expression window for in vivo cell therapy from days, to several months. These groundbreaking results highlight the potential of circVec as a disruptive platform technology for next generation gene and cell therapies, with several opportunities to expand into other therapeutic and non-therapeutic areas in the future.
Currently, Circio has no known competitors with comparative circRNA expression technologies that perform to the same level and have a similar breadth of applications. The Group has a uniquely experienced in house team, led by circRNA co-discoverers Dr. Thomas B Hansen (CTO) and Dr. Erik D Wiklund (CEO). The circVec platform is protected both by patents covering key genetic design features, as well as internal know-how and trade secrets. The circVec technology is continuously being optimized to maximize its potential and stay ahead of the competition. In sum, these aspects firmly place Circio as the clear global leader in the niche of circRNA-based expression systems, with the necessary team, tools and IP protection to remain at the forefront for years to come.
circVec circular RNA expression platform
The proprietary circVec technology is a versatile platform to build next generation viral and nucleic acid therapeutics for gene and cell therapy. circRNA offers several advantages over conventional mRNA-based expression, including substantially longer half-life and higher protein expression. circRNA pioneer Dr. Thomas B Hansen, CTO of Circio, is building the circVec platform technology together with an experienced scientific team located in the Karolinska Institute environment in Stockholm, Sweden.
At its core, circVec is a DNA-based genetic construct designed for efficient circRNA production from viral and non-viral vectors. The prolonged durability of circRNA drives enhanced and more durable protein expression in cells, which can be leveraged to create more efficient, safer and lower cost therapeutics for patients. By harnessing the natural biological advantages of circRNA, circVec brings the benefits of circRNA into the field of genetic medicine, an area dominated by AAV vectors that currently require high, and often toxic, dosing levels, and where synthetic circRNA and mRNA approaches do not offer sufficient expression durability to be therapeutically relevant.
To date, technical proof-of-concept (PoC) has been established for the circVec approach in multiple vector formats and delivery approaches. Key design features for optimal circRNA biogenesis and high protein expression levels have been identified and validated, and several patent applications covering the core elements of the circVec technology have been filed or are in the drafting stage to protect essential features of the platform.
Following generation of robust and statistically significant in vivo proof-of-concept data for its circVec-AAV gene therapy approach in heart and eye, Circio is currently moving forward to document the performance of circVec in relevant disease contexts. The aim is to generate in vivo efficacy validation in disease models within the next twelve months and subsequently proceed towards selecting one or more lead candidates to bring forward towards clinical entry to establish proof-of-concept in patients.
In parallel, Circio is actively pursuing external opportunities to capture the full potential of the platform. The Group’s strategic business development aims are three-fold i) capitalize on the platform to bring non-dilutive funding into the company to support in house development programs, ii) identify and test complementary technologies that can further enhance and expand the applications of the platform and iii) broaden the development scope in order to generate more validating data and spread R&D risk.