Vesigen Highlights Data Demonstrating Directed Tropism of Non-Viral Delivery Platform at the 30th Annual Congress of the European Society of Gene & Cell Therapy

First demonstration of directed tropism of ARMMs for cell type-specific delivery of therapeutic payloads

Dose-dependent gene editing observed across multiple human cell types following delivery of base editors

CAMBRIDGE, Mass., October 25, 2023 /Business Wire/ — Vesigen Therapeutics, Inc., a biotechnology company developing a novel non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, today presented data highlighting the potential of the Company’s non-viral ARMM (ARrestin-domain 1 Mediated Microvesicles) technology to overcome fundamental delivery challenges that limit the clinical application of emerging treatment modalities. Data highlighted the first demonstration of cell type-specific targeting of ARMMs and functional delivery of base editing complexes across diverse human cell types. The data were presented at the 30th Annual Congress of the European Society of Cell and Gene Therapy (ESGCT), taking place October 24-27 in Brussels, Belgium.

“The data we presented at ESGCT represent the first demonstration of directed tropism of our non-viral ARMMs delivery platform. This important result expands upon the natural biodistribution of ARMMs and demonstrates that ARMMs can be engineered to selectively target cell types of interest,” said Paulash Mohsen, Chief Executive Officer at Vesigen. “Together with data showing functional delivery of gene editing payloads by ARMMs across a range of cell types and beyond the liver, these presentations highlight the potential of our delivery platform to dramatically broaden the clinical application of gene editing, RNA, and protein-based therapeutics across therapeutic areas.”

Details of the poster presentations are as follows.

Wednesday, October 25, 18:15-19:30 CEST and Thursday, October 26, 19:30-20:30 CEST

Biodistribution and Re-Targeting of ARRDC1-Mediated Microvesicles (ARMMs) for Non-Viral Delivery of Therapeutic Payloads (Poster Number: P840)

  • ARMMs loaded with an adenine base editor complex and engineered to present an anti-CD8 antibody demonstrated selective uptake and gene editing in CD8+ cells
  • Robust biodistribution of engineered ARMMs observed across multiple cell and tissue types of mice, minipigs, and non-human primates

Wednesday, October 25, 17:00-18:15 CEST and Thursday, October 26, 20:30-21:30 CEST

Engineered ARRDC1-Mediated Microvesicles (ARMMs) as Vehicles for Delivery of Genome Editing Payloads for Immune Modulation Therapeutics (Poster Number: P787)

  • Functional Cas9, adenine base editor, and cytidine base editor/guide RNA complexes were efficiently packaged in ARMMs
  • Robust, dose-dependent functional delivery of ARMMs loaded with an adenine base editor/guide RNA complex demonstrated across diverse cell types, including primary human T cells and macrophages
  • ARMMs loaded with Cas9/NLRP3 guide RNA complexes exhibited ~60% on-target gene editing efficiency in liver Kupffer cells and blunted liver inflammation in a mouse model of acute liver injury

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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