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Collaboration targets lower-dose therapies for genetic muscle disease and in vivo cell therapy.
By: Charlie Sternberg
Associate Editor
Circio Holding ASA, a biotechnology company developing novel circular RNA expression technology for gene and cell therapy, and GenAssist Ltd, a company pioneering next-generation genetic drugs through its tissue‑specific AAV delivery platform, have entered a research collaboration to develop circVec-enhanced AAV vectors specifically engineered for in vivo cell therapy and targeted, low dose systemic gene therapy.
Genetic muscle disease is an area of major unmet medical need, where current gene therapy’s high dosing requirements are associated with severe toxicity. By integrating Circio’s and GenAssist’s complementary technologies, the parties aim to develop joint next generation of AAV gene therapy candidates. The focus is on addressing genetic muscle conditions where high and broad muscle-specific expression is required at substantially lower therapeutic AAV doses than can be achieved by conventional AAV gene therapy.
“We are incredibly impressed by Circio’s unique circular RNA technology and see tremendous potential in how it can dramatically boost gene expression. Our second-generation AAV platform establishes a new benchmark for safety, utilizing highly tissue-specific, de-targeted capsids to dramatically lower systemic dosing while eliminating off-target toxicity,” said Dr. Chunyan He, CEO of GenAssist. “Through our collaboration with Circio, we integrate their unique circular RNA technology—whose exceptional stability and superior transcriptional output ensure robust, sustained expression even at significantly reduced vector loads. This partnership directly addresses the core demands of next-generation genetic medicine, overcoming the traditional dose-expression trade-off to deliver safer and more effective therapies.”
In addition, Circio and GenAssist will explore the potential of generating joint in vivo CAR-T candidates for oncology and autoimmune applications. The collaboration will involve production of novel AAVs combining GenAssist’s proprietary T-cell targeting with the circVec expression cassette from Circio. The combined AAVs will subsequently be tested in vitro and in vivo, and if successful, candidates for further development will be nominated for pre-clinical development.
Dr. Thomas B Hansen, CTO of Circio, said, “The partnership between Circio and GenAssist will aim to evaluate whether the enhanced circVec expression acts synergistically with these targeted capsids and promoters. This fits perfectly into Circio’s strategy of testing circVec in multiple tissues using different AAV variants, both internally and externally. This will allow us to identify new therapeutic avenues where circVec delivers a benefit, and forge partnerships potentially enabling multiple future development opportunities.”
In May, Circio and AaviGen Gmbh jointly entered a research collaboration to develop circVec-enhanced AAV vectors specifically engineered for targeted, low dose heart gene therapies.
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