Vesigen Showcases Key In Vivo and Targeted Tropism Data for Non-Viral Delivery at 2024 ASGCT and ARVO Meetings

Vesigen Therapeutics, Inc., a biotech company pioneering a non-viral delivery platform for gene editors, RNA, and protein-based therapeutics, recently highlighted significant advancements in its ARRDC1-Mediated Microvesicles (ARMMs) technology during two major scientific meetings. These presentations were delivered at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting and the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, revealing promising new data related to the delivery of CRISPR/Cas9 and base editing complexes via engineered ARMMs.

Vesigen's ARMMs technology has shown considerable potential in functionally delivering various payloads, including gene editors, to a broad spectrum of disease-relevant cells and tissues. The company’s recent non-human primate (NHP) studies underscored the success of gene editor delivery and the selective targeting capabilities of ARMMs in vivo.

Paulash Mohsen, CEO of Vesigen, expressed excitement over the breakthroughs achieved with the ARMMs platform. He emphasized the therapeutic potential of the engineered ARMMs for in vivo delivery of genome editors to diverse cell types and highlighted the safety benefits of a human cell-derived system. Mohsen also indicated the company’s commitment to advancing this technology and moving toward clinical development of their lead program.

Key findings from the ARVO and ASGCT presentations include:

1. Functional Delivery and Biodistribution:
- New in vivo and in vitro data, including NHP studies, demonstrated the effective delivery and biodistribution of ARMMs to various cell types.
- Studies showed successful subretinal administration of ARMMs in adult minipigs and NHPs, resulting in the transfection of retinal pigment epithelium (RPE) and photoreceptors. In NHP eyes, about 80% of cones, including those in the macular region, were transfected.

2. Ocular Therapeutics:
- ARMMs achieved up to 80% base editing of the ABCA4 gene in targeted regions within in vitro and ex vivo models. This was further validated by in vivo data in mouse and NHP retinas, suggesting potential treatments for genetically-driven ocular diseases like Stargardt disease.

3. Immune Modulation:
- ARMMs successfully delivered payloads to liver sinusoidal endothelial cells (LSECs) and Kupffer cells in mouse and NHP models. Gene editing payloads were able to disrupt inflammatory mediators such as the NLRP3 inflammasome and IRF5, showing reduced inflammation in a mouse acute liver injury (ALI) model.

4. Neurological Applications:
- Another study highlighted ARMMs' capability to deliver genome editors and guide RNAs to neurons derived from Friedreich ataxia (FA) patients and primary mouse neurons, effectively excising pathogenic GAA trinucleotide repeat expansions.

5. Cell-Specific Targeting:
- ARMMs can be engineered to selectively target difficult-to-reach cell types. For instance, ARMMs decorated with anti-CD8 antibodies were shown to specifically target T cell populations, demonstrating selective gene editing in both in vitro and in vivo mouse models.
- Additional presentations showed that different surface engagers could direct payload delivery to multiple target cell types, proving the modular nature of the platform.

6. Optimization for Large-Scale Manufacturing:
- Vesigen's efforts in optimizing scalable production and purification processes for ARMMs were highlighted, showing that the final products possess high purity, consistent size distribution, and payload concentration, ensuring effective functional delivery in both in vitro and in vivo settings.

Vesigen Therapeutics remains dedicated to developing transformative medicines through their innovative ARMMs platform, aiming to address significant unmet medical needs by advancing the non-viral delivery of therapeutic payloads.

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