• Circio's R&D strategy is centered on gene therapy, where circVec can deliver substantial improvement over current gold-standard approaches
• Circio has selected AAV-based gene therapy for Alpha-1-antitrypsin deficiency (AATD) as the lead program
• Circio aims to secure the first circVec partnering deal within twelve months
• The recent circVec data and a financial update are presented in a company update webcast available on Circio's website     

Oslo, Norway 17 April 2024 - Circio Holding ASA (OSE: CRNA), a biotechnology company developing next generation circular RNA vector technology for gene therapy, today 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.

"The circVec 2.1 design is performing very well in vitro, and this is now being translated in vivo with demonstration of enhanced expression level and durability for circVec 2.1 DNA vectors in mouse models. This provides an important technical proof-of-concept for Circio's technology platform in a real biological system, which we expect will translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "This new data indicate that circVec has the potential to outperform current gold-standard gene therapy approaches, and we are rapidly advancing to design and test circVec in several AAV and DNA-based therapeutic vector formats."  

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.

"Establishing a robust technical in vivo proof-of-concept is a major milestone for the development of the circVec platform. Based on this validation, Circio will now explore which targets and diseases represent the best therapeutic and commercial opportunities," said Dr. Erik Digman Wiklund, CEO at Circio. "Initially, we have selected AATD as the lead program where our unique `remove-&-replace' circVec design has an opportunity to solve two pathological issues in a single differentiated product. Our aim is to establish in vivo proof-of-concept in AATD within the next twelve months and select a lead therapeutic candidate by the middle of next year. We are confident that circVec can be highly effective in AATD and produce novel gene therapies that outperform current approaches."

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.

"Although AATD is Circio's lead internal focus, we are continuously exploring new applications and disease targets to build and broaden our technology platform and have several ongoing external dialogues to identify opportunities for future collaborations. Circio is currently working to address specific questions and requests from these prospective partners and aim to complete our first business development transaction within the next twelve months," said Dr. Lubor Gaal, CFO and Head of Business Development at Circio.

The recent circVec data, as well as a financial update and information about the intended fundraising during Q2 2024, are presented and discussed in a webcast available via Circio's webpage and the Redeye platform link:

Link to webcast - access via Redeye (https://redeye-3.wistia.com/medias/ghqlfw3t3i)

Circio company update 17 April PDF.pdf (https://mb.cision.com/Public/17093/3962653/90a924e82debe550.pdf)