- circVec results demonstrate significantly enhanced protein expression and durability vs. conventional mRNA-based expression with DNA vectors in vivo
- 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
"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
"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.
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.
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
Circio company update 17 April PDF.pdf
For further information, please contact:
Phone: +47 413 33 536
Email: erik.wiklund@circio.com
Phone: +34 683343811
Email: lubor.gaal@circio.com
About Circio
Building next generation RNA therapeutics
Circio Holding ASA is a biotechnology company developing novel circular RNA gene therapies and immunotherapy medicines.
Circio has established a unique circular RNA (circRNA) platform for genetic medicine. The proprietary circVec technology is based on a modular genetic cassette design for efficient biogenesis of multifunctional circRNA from DNA and viral vectors, which can be deployed in multiple disease settings. The circVec platform has demonstrated enhanced and more durable protein expression than classic mRNA vector systems, and has the potential to become the new gold-standard for DNA and virus-based therapeutics in the future. The circRNA R&D activities are being conducted by the wholly owned subsidiary
In addition, Circio is developing a cancer vaccine, TG01, targeting KRAS driver mutations. TG01 is currently being tested in three clinical trials: RAS-mutated pancreatic cancer and lung and non-resectable pancreatic cancer in US, and multiple myeloma in
https://news.cision.com/circio/r/circio-announces-in-vivo-proof-of-concept-for-its-circvec-circular-rna-platform-technology-and-reinf,c3962653
https://mb.cision.com/Public/17093/3962653/90a924e82debe550.pdf
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