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- circVec 2.1 DNA vectors have now demonstrated higher and more durable expression vs. conventional mRNA-based expression in vivo for up to five months
- Machine learning sequence optimization has resulted in a new and enhanced circVec 2.2 design, which increases protein expression by 2-4 -fold vs. circVec 2.1
- The enhanced circVec expression durability can deliver substantial improvement over current gold-standard gene therapy approaches
- Circio management presents and discusses these data in a webcast published today, 17 June 2024, watch the webcast here (https://redeye-3.wistia.com/medias/bst2y63ily)
Oslo, Norway 17 June 2024 - Circio Holding ASA (OSE: CRNA), a biotechnology company developing next generation circular RNA vector technology for gene therapy, today announces updated in vivo data. This new data demonstrates a substantial durability advantage of Circio's circVec technology over conventional mRNA expression. In addition, Circio has undertaken sequence optimization resulting in a new and enhanced circVec 2.2 design. The new data are presented by Circio's scientific leadership team in a webcast (https://redeye-3.wistia.com/medias/bst2y63ily) released today 17 June 2024.
"The circVec 2.1 design is performing very well in vivo, and Circio has now validated expression for up to five months. This is a clear demonstration of a substantial, and statistically significant, advantage over mRNA-based expression. As a result, the data provides an important proof-of-concept for Circio's circular RNA platform, which we expect will translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "In parallel, we have deployed machine learning to optimize circVec sequence composition. This has enabled us to generate a new and even more powerful circVec 2.2 design, which increases protein expression by 2-4 -fold vs. circVec 2.1 constructs."
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). This concept has the ability to both replace functional AAT protein and remove the disease variant. AATD is a genetic disease that causes severe symptoms in the lung and liver. There are currently no satisfactory therapeutic options available, and AATD represents a major unmet medical need with over 200,000 patients affected in the USA and EU.
"In recent months, Circio has made important progress with its circVec AATD gene therapy constructs. We have now technically validated a very efficient and selective `remove' functionality to complement the robust and durable circular RNA `replace' expression of the AAT protein," said Dr. Victor Levitsky, CSO at Circio. "Almost all gene therapies today are based on the AAV vector. We are confident that circVec-based expression can enhance this format. Circio's first circular RNA AAV constructs have now been validated in vivo, and their performance compared to conventional mRNA-AAVs will be tracked over the coming months. We expect that these data will be of considerable interest to prospective gene therapy partners."
The new circVec data is presented and discussed by Circio's CEO, CSO and CTO in a webcast released today 17 June 2024 available via Circio's webpage and the Redeye platform.
Watch the webcast here (https://redeye-3.wistia.com/medias/bst2y63ily).