On May 13, 2024 Circio Holding ASA (OSE: CRNA), a biotechnology company developing circular RNA-based gene therapy, reported that it has presented two posters that demonstrate in vivo proof-of-concept for its powerful and differentiated circVec platform approach to gene therapy (Press release, Circio, MAY 13, 2024, View Source [SID1234643139]). The two posters were presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 annual meeting 7-11 May in Baltimore, USA

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"Circio has generated results demonstrating that the circVec 2.1 design performs very well in vitro. We have now confirmed this in vivo with statistically significant higher expression level and durability for circVec 2.1 DNA vectors compared to standard linear mRNA-based expression. These results provide an important technical proof-of-concept for Circio´s technology platform in an animal model. We now have confirmation for our expectation that this could translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "In recent experiments, Circio has observed up to four months circVec durability in vivo. This substantially outperforms mRNA vector expression. Following these results, we can rapidly advance to design and test circVec in several AAV and DNA-based vectors. This will validate these very promising data in therapeutically relevant formats."

At ASGCT (Free ASGCT Whitepaper), Circio also presented the dual-function ‘remove-&-replace’ concept for Alpha-1-antitrypsin deficiency (AATD). This genetic disease causes severe symptoms in the lung and liver. There are currently no satisfactory therapeutic options available for this indication and AATD still represents a major unmet medical need. There are over 200,000 AATD patients affected in the USA and EU alone. With the technologically differentiated circVec remove-&-replace format, Circio has developed a unique gene therapy concept that can deal with both the lung and liver-associated symptoms in one single therapeutic.

"AATD is a challenging genetic disease to treat. This is in part due to the two distinct pathologies in the liver and lung," said Dr. Victor Levitsky, CSO at Circio. "We have now established and technically validated circVec constructs that can both replenish functional wild-type AAT and specifically remove more than 90% of the mutated protein. This is challenging to achieve because the functional and mutant forms are very similar. By using circular RNA-based AAT expression, Circio is uniquely able to separate the two species for mutant-specific knockdown, thereby solving two problems with one single product."

Optimization and In Vivo Performance of circVec, a Vector-Based Circular RNA Expression Platform; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024

Expressing AAT from circular RNA-encoding vectors as a promising gene therapy approach for Alpha 1-antitrypsin deficiency; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024