▎WuXiEdited by Kant Content Team

Today, ViGeneron announced in the prestigious academic journal *Nature Communications* (Nature Communications) published a peer-reviewed paper showcasing the potential of its proprietary dual adeno-associated virus (AAV) vector technology, REVeRT, based on mRNA trans-splicing.Data shows,This technology can deliver genes larger than the conventional AAV packaging size with high recombination efficiency, enabling gene replacement, or simultaneous knockout and transcriptional activation of different genes.

Although therapeutic AAV vectors are one of the most effective and widely used gene delivery methods in current gene therapy, their limitation of less than 5 kb genomic packaging capacity restricts the vector from expressing large therapeutic-related genes, which undoubtedly presents a significant challenge for the development of novel gene therapies.REVeRT is an innovative and flexible dual-vector approach based on AAV vectors, designed to overcome this limitation by splitting the gene into two parts and packaging them into separate AAV vectors, followed by mRNA trans-splicing recombination.

According to the research report published in *Nature Communications*, the REVeRT technology has been validated both in vitro and in vivo, demonstrating its ability to efficiently recombine disease-related genes and gene-editing modules larger than 5 kb.REVeRT technology not only achieves high expression of the target gene but also avoids the generation of foreign protein by-products, thereby reducing potential immunogenicity issues.REVeRT can flexibly select cleavage sites and is suitable for various AAV serotypes and different administration routes, efficiently delivering the target gene to various tissues and organs, including the eyes, heart, brain, etc.

In the Stargardt disease mouse model,Researchers further confirmed that the use of REVeRT technology can successfully achieveABCA4High expression of the gene – a gene closely associated with Stargardt disease.Preliminary experimental data supported potential improvements in retinal function.

Dr. Man Xu, co-founder and CEO of ViGeneron, stated in the press release, "The data published in this peer-reviewed paper demonstrate the transformative potential of our REVeRT technology for the efficient delivery of large genes."We have made significant progress in the Stargardt project VG801 using REVeRT technology, which is expected to enter clinical trials next year, alongside the VG901 project targeting retinitis pigmentosa."This once again demonstrates our commitment to advancing cutting-edge gene therapies that have the potential to transform patients' lives."

Dr. Markus Kalousek, Chief Business Officer of ViGeneron, stated: "The REVeRT platform enables us to address some of the most common genetic diseases, which are often associated with large genes. It also allows for transactivation – a CRISPR-driven technology with the potential to regulate multiple genes of any size in both hereditary and non-hereditary diseases. We are actively exploring strategic partnerships across various disease areas to accelerate the delivery of these transformative therapies to patients."

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References:

[1] Nature Communications publication reveals highly efficient AAV delivery of large genes using ViGeneron´s REVeRT technology Retrieved October 25, 2023 from https://vigeneron.com/press/nature-communications-publication-reveals-highly-efficient-aav-delivery-of-large-genes-using-vigenerons-revert-technology/

[2] Riedmayr, L.M., Hinrichsmeyer, K.S., Thalhammer, S.B. et al. mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy. Nat Commun 14, 6578 (2023). https://doi.org/10.1038/s41467-023-42386-0

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