Data demonstrates superiority of circular RNA vs linear mRNA

RNA strand

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting.

The in vivo data showed statistically significant higher expression level and durability for circVec 2.1 DNA vectors compared to standard linear mRNA-based expression.

“We now have confirmation for our expectation that this could translate into improved gene therapies for patients in the future,” said Dr Thomas 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, Circio also presented the dual-function ‘remove-&-replace’ concept for Alpha-1-antitrypsin deficiency (AATD).

There are currently no satisfactory therapeutic options available for AATD, which still represents a major unmet medical need. There are over 200,000 AATD patients affected in the USA and EU alone.

“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.”

Diana Spencer, Senior Digital Content Editor, DDW

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