Biotechnology companie Circio Holding ASA (OSE:CRNA) and GenAssist Ltd announced on Monday that they have entered into a research collaboration to develop circVec-enhanced AAV vectors for in vivo cell therapy and targeted systemic gene therapy.
This partnership will combine Circio's circular RNA platform, which has demonstrated up to 50-fold enhanced gene expression from AAV vectors in vivo, with GenAssist's tissue-specific and liver de-targeting AAV capsids and promoters.
The companies aim to evaluate the potential synergy of their technologies to develop next-generation AAV gene therapies targeting genetic muscle diseases, where current high dosing requirements are associated with severe toxicity. The collaboration will focus on achieving high and broad muscle-specific expression at substantially lower therapeutic AAV doses than conventional gene therapy approaches.
Circio and GenAssist will also explore the development of in vivo CAR-T candidates for oncology and autoimmune applications by combining GenAssist's T-cell targeting technology with Circio's circVec expression cassette. The resulting AAVs will be tested in vitro and in vivo, with successful candidates advancing to nomination for pre-clinical development.
The collaboration forms part of Circio's partnering strategy to develop gene therapy vectors with improved performance and specificity to address unmet medical needs in cancer and genetic muscle disease.
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