CIRCIO HOLDING ASA Circio Holding ASA announces that it has established technical in vivo proof-of-concept for its proprietary circVec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression. The circVec technology has broad potential, particularly to enhance the potency and reduce cost of current gold-standard gene therapy, and the R&D strategy is centered on this rapidly expanding therapeutic area. In parallel to the in vivo characterization, Circio has tested and incorporated further features into the circVec platform.
A dual-function 'remove-&-replace' concept has been designed and validated in vitro for Alpha-1-antitrypsin deficiency (AATD), with the ability to both replace functional AAT protein and remove the disease variant. This genetic disease causes severe symptoms in the lung and liver, and there are currently no satisfactory therapeutic options available. AATD represents a major unmet medical need and there are over 200,000 patients affected in the USA and EU.
To Circio´s knowledge, circVec 2.1 far exceeds other known intra-cellular circRNA-based expression systems, both in terms of circRNA biogenesis efficiency and protein yield. The platform still has further potential, and Circio is continuously improving the technology towards circVec 3.0 and beyond. The platform is protected by deep internal expertise and know-how, with three patents protecting the core technological features filed to date, and additional applications in progress.
