Circio and GenAssist Collaborate on Gene Therapy for Muscle Disease and In Vivo Cell Therapy

Oslo, Norway-based Circio and Suzhou, China-based GenAssist entered into 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, according to a Circio spokesperson. 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.

“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 Chunyan He, PhD, CEO of GenAssist. “Through our collaboration with Circio, we integrate their unique circular RNA technology. 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 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 preclinical development.

“The targeted AAVs developed by GenAssist have the ability to specifically and efficiently transduce muscle or T-cells upon systemic delivery with near-complete liver de-targeting,” added Thomas Hansen, PhD, CTO of Circio. “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. China is a particularly interesting geography, with cutting edge science and accelerated pathways to establish early clinical data.”