Circio and Certest to Collaborate on LNP Formulations

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Editor's note: this story was originally published on BioPharmInternational.com.

Circio Holding, a Norway-based biotechnology company specializing in next-generation circular RNA (circRNA) vector technology for gene therapy, announced on Nov. 19, 2024, that it has entered into a collaboration with Spain-based Certest Biotec, an independent biotechnology company focused on the development and manufacturing of in-vitro diagnostic products. The companies aim to develop Circio’s circVec technology into lipid nanoparticles (LNPs) to be used in formulations of therapeutics that will enable tissue-specific delivery of DNA vectors for addressing genetic disease (1).

Certest has successfully performed packaging and in-vitro validation of circVec–LNP formulations that are targeted for delivery to lung, liver, and spleen tissue with positive technical results. Under the collaboration, Circio will next move on to test these circVec–LNP formulations in vivo over the next three to six months. The goal is to assess delivery efficiency, expression level, and durability. If the experiments are successful, they may lead to the development of multiple in-house and partnered circVec therapeutic candidates for treating genetic diseases, according to a company press release.

“Circio’s powerful circVec-DNA technology achieves more than six months durability in mouse models on a single intra-muscular injection, with up to 15-fold increase in payload expression levels vs. standard mRNA [messenger RNA]-based vector systems,” said Victor Levitsky, MD, PhD, chief scientific officer (CSO), Circio, in the press release. “Based on these promising findings in vivo, we are now looking for targeted delivery technology that can enable therapeutic application of circVec in specific tissues and diseases of high unmet medical need. We selected Certest as a preferred partner for circVec–LNP formulation due to the impressive tissue specificity demonstrated for their proprietary nanoparticles.”

According to the release, Certest’s proprietary ionizable lipids and optimized LNP formulations have shown notable delivery specificity to several tissues of interest for Circio (lung, liver, and spleen). In addition, technical formulation and in-vitro analysis of circVec–LNP formulations by Certest have been successful, allowing the companies to advance to in-vivo delivery testing.

“The library of ionizable lipids developed by our team is engineered for a wide range of nucleic acid payloads, exhibiting a great delivery capacity in preclinical studies and allows the production of thermostable LNPs,” said Juan Martínez Oliván, PhD, CSO of Certest Pharma, specialized unit in nucleic acids delivery systems at Certest Biotec, in the press release. “Our capability to passively target different tissues unlocks the possibility to treat organ-specific diseases. We are very enthusiastic about the joint formulation of Circio’s new approach to circular RNA vector technology with our versatile ionizable lipids, creating a unique LNP-strategy for gene therapy treatment.”

Circio’s proprietary circVec technology is based on a modular genetic cassette design, which allows for efficient biogenesis of multifunctional circRNA from DNA and viral vectors. This circRNA can be deployed in multiple disease settings, including cell and gene therapy and chronic diseases, according to the press release. CircRNAs are a class of single-stranded RNAs that are being engineered as novel treatments for disease treatment and prevention (2).

References

1. Circio Holding. Circio Announces circVec LNP-delivery Collaboration with Certest Progressing to In Vivo Testing. Press Release. Nov. 19, 2024.
2. Niu, D.; Wu, Y.; Lian, J. Circular RNA Vaccine in Disease Prevention and Treatment. Signal Transduction Targeted Ther. 2023, 8, 341. DOI: 10.1038/s41392-023-01561-x

Source: Circio Holding