Vesigen Highlights Non-Human Primate Biodistribution of Non-Viral Technology and Functional Delivery of Genome Editors In Vivo at the Cracking the Code: The Dawn of Nucleic Acid Medicines Meeting

Robust uptake observed in photoreceptors and retinal pigment epithelium (RPE) of mice, minipigs, and non-human primates

Additional data demonstrate rescue of acute liver injury disease model by delivery of a gene editor to Kupffer cells

CAMBRIDGE, Mass., October 19, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, presented data further highlighting the potential of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) technology to deliver novel therapeutic payloads to cell and tissue types relevant to multiple therapeutic areas with high unmet need. Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex. The data were presented at the Cracking the Code: The Dawn of Nucleic Acid Medicines meeting, taking place October 17-19, 2023, in Worcester, MA.

“These data further demonstrate the potential of our non-viral ARMM technology to deliver challenging therapeutic payloads, including gene editing complexes, to multiple cell and tissue types impacted in disease,” said Paulash Mohsen, Chief Executive Officer at Vesigen. “We have shown that our ARMM technology can successfully deliver functionally active gene editing complexes to Kupffer cells and ameliorate disease in an acute liver injury model. Additional data in mice, minipigs, and non-human primates demonstrated the efficient delivery of subretinally administered ARMMs to the RPE and photoreceptors, cell types associated with multiple retinal diseases. Together, these data sets underscore the transformative capacity of our technology to overcome fundamental delivery challenges that limit the potential of promising new genetic medicines.”

Details of the poster presentations are as follows.

Wednesday, October 18

Biodistribution of ARMMs as Non-Viral Vehicles for Therapeutic Payloads by Sub-Retinal Administration in Minipigs and NHP

  • Engineered ARMMs were administered to adult minipigs and non-human primates (NHPs) by subretinal injection
  • In both minipigs and NHPs, robust ARMMs uptake was observed in rod and cone photoreceptors as well as the retinal pigment epithelium
  • Histopathological evaluation showed no evidence of immune cell infiltration, cytotoxicity, or cell death following ARMMs administration

ARMMs Enable In Vivo Functional Delivery of Cas9 to Disrupt NLRP3 in Kupffer Cells and Ameliorate Acute Liver Injury

  • ARMMs loaded with Cas9 and a guide RNA targeting NLRP3 – a central regulator of liver disease pathogenesis – were administered intravenously in a mouse model of acute liver injury
  • Mice treated with ARMMs loaded with Cas9/NLRP3 gRNA complexes exhibited ~60% on-target gene editing efficiency in liver Kupffer cells, significant reduction in circulating liver enzymes, and improved liver cell viability compared to vehicle-treated mice

About Vesigen Therapeutics

Vesigen Therapeutics is a biotechnology company developing a novel, non-viral delivery technology for gene editing, RNA, and protein-based therapeutics. Vesigen’s patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types and is committed to developing transformative medicines that address current unmet medical needs. ARMMs were discovered and engineered into a drug delivery system at the Harvard T.H. Chan School of Public Health.

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